Archive for the ‘Gene Therapy Research’ Category

Analysis of the Global Hemophilia Gene Therapy Market

The report on the global Hemophilia Gene Therapy market reveals that the market is expected to grow at a CAGR of ~XX% during the considered forecast period (2019-2029) and estimated to reach a value of ~US$XX by the end of 2029. The latest report is a valuable tool for stakeholders, established market players, emerging players, and other entities to devise effective strategies to combat the impact of COVID-19

Further, by leveraging the insights enclosed in the report, market players can devise concise, impactful, and highly effective growth strategies to solidify their position in the Hemophilia Gene Therapy market.

Research on the Hemophilia Gene Therapy Market Addresses the Following Queries

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Competitive Landscape

The competitive landscape section offers valuable insights related to the business prospects of leading market players operating in the Hemophilia Gene Therapy market. The market share, product portfolio, pricing strategy, and growth strategies adopted by each market player is included in the report. The major steps taken by key players to address the business challenges put forward by the novel COVID-19 pandemic is discussed in the report.

Regional Landscape

The regional landscape section provides a deep understanding of the regulatory framework, current market trends, opportunities, and challenges faced by market players in each regional market. The various regions covered in the report include:

End-User Assessment

The report bifurcates the Hemophilia Gene Therapy market based on different end users. The supply-demand ratio and consumption volume of each end-user is accurately depicted in the report.

Competition AnalysisIn the competitive analysis section of the report, leading as well as prominent players of the global Hemophilia Gene Therapy market are broadly studied on the basis of key factors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on price and revenue (global level) by player for the period 2015-2020.On the whole, the report proves to be an effective tool that players can use to gain a competitive edge over their competitors and ensure lasting success in the global Hemophilia Gene Therapy market. All of the findings, data, and information provided in the report are validated and revalidated with the help of trustworthy sources. The analysts who have authored the report took a unique and industry-best research and analysis approach for an in-depth study of the global Hemophilia Gene Therapy market.The following players are covered in this report:Spark TherapeuticsUltragenyxShire PLCSangamo TherapeuticsBioverativBioMarinuniQureFreeline TherapeuticsHemophilia Gene Therapy Breakdown Data by TypeHemophilia AHemophilia BHemophilia Gene Therapy Breakdown Data by ApplicationHemophilia A Gene TherapyHemophilia B Gene Therapy

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Essential Findings of the Hemophilia Gene Therapy Market Report:

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Assessing the Fallout From the Coronavirus Pandemic Hemophilia Gene Therapy to Discern Steadfast Expansion During 2019-2053 - amitnetserver

CAMBRIDGE, Mass., May 1, 2020 /PRNewswire/ --Obsidian Therapeutics, a biotechnology company pioneering controllable cell and gene therapies, today announced it will share the architecture and components of its Safe Workplace Function Tool (SWFT) Productivity Solution. The SWFT Solution was designed in response to the COVID19 pandemic to support a safe work environment while maintaining productivity in the lab.

The SWFT Solution is a web-based application built and integrated into Microsoft 365 that allows scientists to view and schedule lab-based activities including by date, time and lab location. SWFT promotes collaboration and coordination between teams by predicting scheduling and occupancy conflicts, which allows team members to adjust their schedules to promote social distancing in the lab and office.

"The SWFT Solution has enabled Obsidian to continue to generate critical data across our cytoDRiVE development programs, while maximizing the safety of our staff," stated Catherine Stehman-Breen, M.D., Chief Research and Development Officer at Obsidian. "We have already shared this technology with large pharmaceutical and small biotechnology companies who are interested in our SWFT Solution to get their labs back up and running. We believe that it is more important than ever before to leverage one another's expertise in order to overcome challenges as we work tirelessly to deliver meaningful outcomes to patients in need."

To create Obsidian's bespoke application, (1) a capacity analysis was conducted, taking into consideration lab space and occupancy levels, and (2) in collaboration with scientists, workflow recommendations were implemented to determine an appropriate shift schedule. These steps facilitated the development of a solution that enabled scientists to plan their experiments with specific lab locations and shifts, as well as be alerted to and prevent any over-capacity issues. Obsidian implemented four three-hour lab blocks, with thirty-minutes of cleaning time between blocks, over a seven-day work week, to ensure that coronavirus-related safety recommendations were met.

Celeste Richardson, Ph.D., Vice President of Cell Therapy of Obsidian, stated, "We have a commitment to our employees to keep the health and safety of our employees top of mind while they work to bring innovative therapies to patients. The development of the SWFT Solution perfectly demonstrates Obsidian's culture of teamwork, determination and innovation."

Obsidian's IT Partner, TRNDigital, is continuing to iterate the tool to ensure it continues to meet scientists' needs and can be made available to others. The solution has been expanded to include density planning in the Obsidian offices. In addition, the SWFT Solution is scalable and flexible to other laboratory setups.

The SWFT solution was developed in-house by Henry Rogalin, Data Scientist, under the leadership of Nic Betts, Head of IT and Facilities, and in collaboration with a safety and facilities capacity team led by Jillian Giguere, Senior Manager of Laboratory Operations, Facilities, and EHS. For more information on SWFT, submit this inquiry form. Informational sessions and training will be hosted as requested.

About Obsidian TherapeuticsObsidian Therapeutics is a biotechnology company pioneering controllable cell and gene therapies to deliver transformative outcomes for patients with intractable diseases. Obsidian's proprietary cytoDRiVE technology provides a way to control protein degradation using FDA-approved small molecules, permitting precise control of the timing and level of protein expression. The cytoDRiVE platform can be applied to design controllable intracellular, membrane and secreted proteins for cell and gene therapies as well as other applications. The Company's initial applications focus on developing novel cell therapies for the treatment of cancer. Obsidian is headquartered in Cambridge, Mass. For more information, please visit http://www.obsidiantx.com.

Media Contact:Maggie BellerRusso Partners, LLCMaggie.beller@russopartnersllc.com646-942-5631

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SOURCE Obsidian Therapeutics

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Obsidian Therapeutics Develops and Shares Safe Workplace Productivity Solution in Response to COVID19 - P&T Community

Sarepta Therapeutics, Inc.s (NASDAQ:SRPT): Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapy, and other genetic therapeutic modalities approaches for the treatment of rare diseases. The US$9.2b market-cap company announced a latest loss of -US$715.1m on 31 December 2019 for its most recent financial year result. As path to profitability is the topic on SRPTs investors mind, Ive decided to gauge market sentiment. In this article, I will touch on the expectations for SRPTs growth and when analysts expect the company to become profitable.

Check out our latest analysis for Sarepta Therapeutics

According to the 22 industry analysts covering SRPT, the consensus is breakeven is near. They expect the company to post a final loss in 2021, before turning a profit of US$716m in 2022. Therefore, SRPT is expected to breakeven roughly 2 years from today. How fast will SRPT have to grow each year in order to reach the breakeven point by 2022? Working backwards from analyst estimates, it turns out that they expect the company to grow 59% year-on-year, on average, which is rather optimistic! If this rate turns out to be too aggressive, SRPT may become profitable much later than analysts predict.

Im not going to go through company-specific developments for SRPT given that this is a high-level summary, but, take into account that by and large biotechs, depending on the stage of product development, have irregular periods of cash flow. So, a high growth rate is not out of the ordinary, particularly when a company is in a period of investment.

One thing I would like to bring into light with SRPT is its relatively high level of debt. Generally, the rule of thumb is debt shouldnt exceed 40% of your equity, which in SRPTs case is 83%. Note that a higher debt obligation increases the risk in investing in the loss-making company.

There are key fundamentals of SRPT which are not covered in this article, but I must stress again that this is merely a basic overview. For a more comprehensive look at SRPT, take a look at SRPTs company page on Simply Wall St. Ive also compiled a list of key factors you should look at:

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Thank you for reading.

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When Will Sarepta Therapeutics, Inc. (NASDAQ:SRPT) Become Profitable? - Simply Wall St

DetailsCategory: AntibodiesPublished on Friday, 01 May 2020 15:04Hits: 468

Recommendation for approval based on results from pivotal Phase 3 MEDALIST and BELIEVE studies

PRINCETON, NJ & CAMBRIDGE, MA, USA I April 30, 2020 IBristol Myers Squibb (NYSE: BMY) and Acceleron Pharma Inc. (NASDAQ: XLRN) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion, recommending the approval of Reblozyl (luspatercept) for the treatment of:

This CHMP recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). If approved, Reblozyl would be the first erythroid maturation agent approved in the EU, representing a new class of therapy for eligible patients. The safety and efficacy results provided in the application are from the pivotal Phase 3 MEDALIST and BELIEVE studies, evaluating the ability of Reblozyl to effectively address anemia associated with MDS and beta thalassemia, respectively.

"Patients with myelodysplastic syndromes who experience anemia have limited treatment options, and some have been shown to not respond to available erythropoietin-based therapies," said Uwe Platzbecker, M.D., Head of Clinic and Policlinic for Hematology and Cell Therapy, Leipzig University Hospital and lead investigator of the MEDALIST study. If approved, the introduction of a new class of therapy in Reblozyl could provide a promising option to help relieve patients from the burden of regular transfusions to manage their disease.

Todays positive CHMP opinion of Reblozyl is an important milestone for adult beta thalassemia patients in the EU who have limited treatment options to address anemia, a serious consequence of the disease, said Maria Domenica Cappellini, M.D., Professor of Medicine, University of Milan, Fondazione IRCCS Ca Granda and lead investigator of the BELIEVE study. Reblozyl has the potential to significantly decrease the number of red blood cell transfusions patients need.

This decision by the CHMP is an important step towards making this first-in-class therapy an option for eligible patients with anemia due to beta thalassemia or myelodysplastic syndromes, said Diane McDowell, M.D., vice president, Hematology Global Medical Affairs, Bristol Myers Squibb. We, and our partners at Acceleron, look forward to the opportunity to make this treatment option available in the EU and are extremely appreciative of the patients, families and individuals who continue to help us progress important research in a range of serious diseases.

About MEDALIST

MEDALIST is a Phase 3, randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of luspatercept plus best supportive care (BSC) versus placebo plus BSC in adults with IPSS-R-defined very low-, low- or intermediate-risk non-del(5q) myelodysplastic syndromes (MDS). All patients were red blood cell (RBC) transfusion-dependent and were either refractory or intolerant to prior erythropoiesis stimulating agent (ESA) therapy, or were ESA nave and unlikely to respond due to endogenous serum erythropoietin levels of 200 U/L, and had no prior treatment with disease modifying agents. Results of the MEDALIST trial were first presented during the Plenary Session of the 2018 American Society of Hematology (ASH) Annual Meeting and were selected for the Best of ASH. The New England Journal of Medicine published the MEDALIST trial results in January 2020.

About MDS

MDS are a group of closely related blood cancers characterized by ineffective production of healthy red blood cells, white blood cells and platelets, which can lead to anemia and frequent or severe infections. People with MDS who develop anemia often require regular blood transfusions to increase the number of healthy red blood cells in circulation. Frequent transfusions are associated with an increased risk of iron overload, transfusion reactions and infections. There are approximately 50,000 patients with MDS in the EU5 countries.

About BELIEVE

BELIEVE is a Phase 3, randomized, double-blind, placebo-controlled multi-center study comparing luspatercept plus BSC versus placebo plus BSC in adults who require regular RBC transfusions (6-20 RBC units per 24 weeks with no transfusion-free period greater than 35 days during that period) due to beta thalassemia. Results of the BELIEVE trial were first presented at the 2018 ASH Annual Meeting and selected for the Best of ASH. The New England Journal of Medicine published the BELIEVE trial results in March 2020.

About Beta Thalassemia

Beta thalassemia is an inherited blood disorder caused by a genetic defect in hemoglobin. The disease is associated with ineffective erythropoiesis, which results in the production of fewer and less healthy RBCs, often leading to severe anemia a condition that can be debilitating and can lead to more severe complications for patients as well as other serious health issues. Treatment options for anemia associated with beta thalassemia are limited, consisting mainly of frequent RBC transfusions that have the potential to contribute to iron overload, which can cause serious complications such as organ damage. Across the United States, Germany, France, Italy, Spain and the United Kingdom, there are approximately 17,000 patients with beta thalassemia.

About Reblozyl

Reblozyl (luspatercept-aamt), a first-in-class erythroid maturation agent, promotes late-stage red blood cell maturation in animal models. Bristol Myers Squibb and Acceleron are jointly developing Reblozyl as part of a global collaboration. Reblozyl is currently approved in the U.S. for the treatment of:

Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.

Please see full Prescribing Information for REBLOZYL

Bristol Myers Squibb: Advancing Cancer Research

At Bristol Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase patients quality of life, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.

Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.

About Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol-Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as, Celgene, a Bristol-Myers Squibb Company and Juno Therapeutics, a Bristol-Myers Squibb Company.

About Acceleron

Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. The Company's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.

Acceleron focuses its research and development efforts in hematologic and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol Myers Squibb, are co-promoting REBLOZYL (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States and are developing luspatercept for the treatment of chronic anemia in myelofibrosis. Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension, having recently reported positive topline results of the Phase 2 PULSAR trial and actively enrolling patients in the Phase 2 SPECTRA trial.

For more information, please visit http://www.acceleronpharma.com. Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn.

SOURCE: Bristol-Myers Squibb

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Reblozyl (luspatercept) Receives Positive CHMP Opinion for the Treatment of Adults with Anemia in Beta Thalassemia and Myelodysplastic Syndromes |...

In the first article we looked at face masks (when do you need to where one and why?); in the second we looked at disinfection (which disinfectants work best?); and the third topic was social distancing (why is 2 metres considered to be a safe distance?) The subject matter for the fourth article was the lockdown and whether the idea of an 'immunity passport' is a good idea (which is probably isn't). For the firth dive into COVID-19 subjects, we look at where we are with vaccine development, what the most promising antivirals are, and what do the different tests for coronavirus do and how do they vary?In this article, the topic is testing, vaccines and other efforts to help to combat the disease.

A laboratory technician works on coronavirus samples at "Fire Eye" laboratory in Wuhan

STR, STR, AFP

A fluorescent antibody (DFA) stain reveals the presence of rabies viral antigen in a tissue sample harvested from a mandibular salivary gland, suspected of being infected with rabies.

CDC/PHIL

A nurse wearing blue gloves administers a vaccine into a male patient's arm.

Rhoda Baer (National Institutes of Health)

Images of COVID-19 released by US science agencies

John SAEKI, AFP

France has now detected a total of 11 cases of the novel coronavirus

JEFF PACHOUD, AFP/File

This opinion article was written by an independent writer. The opinions and views expressed herein are those of the author and are not necessarily intended to reflect those of DigitalJournal.com

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Op-Ed: Where are we with the coronavirus vaccine? - Digital Journal

Complete study of the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market is carried out by the analysts in this report, taking into consideration key factors like drivers, challenges, recent trends, opportunities, advancements, and competitive landscape. This report offers a clear understanding of the present as well as future scenario of the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing industry. Research techniques like PESTLE and Porters Five Forces analysis have been deployed by the researchers. They have also provided accurate data on Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing production, capacity, price, cost, margin, and revenue to help the players gain a clear understanding into the overall existing and future market situation.

Key companies operating in the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market include , BioReliance, Cobra Biologics, Oxford BioMedica, UniQure, FinVector, MolMed, MassBiologics, Richter-Helm, FUJIFILM Diosynth Biotechnologies, Lonza, Aldevron, Eurogentec, Cell and Gene Therapy Catapult, Biovian, Brammer Bio, VGXI, PlasmidFactory, bluebird bio, Novasep, Spark Therapeutics, Vigene Biosciences

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Segmental Analysis

The report has classified the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing industry into segments including product type and application. Every segment is evaluated based on growth rate and share. Besides, the analysts have studied the potential regions that may prove rewarding for the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing manufcaturers in the coming years. The regional analysis includes reliable predictions on value and volume, thereby helping market players to gain deep insights into the overall Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing industry.

Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Segment By Type:

, BioReliance, Cobra Biologics, Oxford BioMedica, UniQure, FinVector, MolMed, MassBiologics, Richter-Helm, FUJIFILM Diosynth Biotechnologies, Lonza, Aldevron, Eurogentec, Cell and Gene Therapy Catapult, Biovian, Brammer Bio, VGXI, PlasmidFactory, bluebird bio, Novasep, Spark Therapeutics, Vigene Biosciences

Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Segment By Application:

, BioReliance, Cobra Biologics, Oxford BioMedica, UniQure, FinVector, MolMed, MassBiologics, Richter-Helm, FUJIFILM Diosynth Biotechnologies, Lonza, Aldevron, Eurogentec, Cell and Gene Therapy Catapult, Biovian, Brammer Bio, VGXI, PlasmidFactory, bluebird bio, Novasep, Spark Therapeutics, Vigene Biosciences

Competitive Landscape

It is important for every market participant to be familiar with the competitive scenario in the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing industry. In order to fulfil the requirements, the industry analysts have evaluated the strategic activities of the competitors to help the key players strengthen their foothold in the market and increase their competitiveness.

Key companies operating in the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market include , BioReliance, Cobra Biologics, Oxford BioMedica, UniQure, FinVector, MolMed, MassBiologics, Richter-Helm, FUJIFILM Diosynth Biotechnologies, Lonza, Aldevron, Eurogentec, Cell and Gene Therapy Catapult, Biovian, Brammer Bio, VGXI, PlasmidFactory, bluebird bio, Novasep, Spark Therapeutics, Vigene Biosciences

Key questions answered in the report:

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TOC

1 Market Overview of Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing1.1 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Overview1.1.1 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Product Scope1.1.2 Market Status and Outlook1.2 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size Overview by Region 2015 VS 2020 VS 20261.3 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Region (2015-2026)1.4 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Historic Market Size by Region (2015-2020)1.5 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size Forecast by Region (2021-2026)1.6 Key Regions Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size YoY Growth (2015-2026)1.6.1 North America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size YoY Growth (2015-2026)1.6.2 Europe Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size YoY Growth (2015-2026)1.6.3 China Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size YoY Growth (2015-2026)1.6.4 Rest of Asia Pacific Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size YoY Growth (2015-2026)1.6.5 Latin America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size YoY Growth (2015-2026)1.6.6 Middle East & Africa Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size YoY Growth (2015-2026)1.7 Coronavirus Disease 2019 (Covid-19): Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Industry Impact1.7.1 How the Covid-19 is Affecting the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Industry1.7.1.1 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business Impact Assessment Covid-191.7.1.2 Supply Chain Challenges1.7.1.3 COVID-19s Impact On Crude Oil and Refined Products1.7.2 Market Trends and Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Potential Opportunities in the COVID-19 Landscape1.7.3 Measures / Proposal against Covid-191.7.3.1 Government Measures to Combat Covid-19 Impact1.7.3.2 Proposal for Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Players to Combat Covid-19 Impact 2 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Overview by Type2.1 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Type: 2015 VS 2020 VS 20262.2 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Historic Market Size by Type (2015-2020)2.3 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Forecasted Market Size by Type (2021-2026)2.4 AAV2.5 Adenoviral2.6 Lentiviral2.7 Retroviral2.8 Plasmid DNA2.9 Other Vectors 3 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Overview by Type3.1 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application: 2015 VS 2020 VS 20263.2 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Historic Market Size by Application (2015-2020)3.3 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Forecasted Market Size by Application (2021-2026)3.4 Cancers3.5 Inherited Disorders3.6 Viral Infections3.7 Others 4 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Competition Analysis by Players4.1 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size (Million US$) by Players (2015-2020)4.2 Global Top Manufacturers by Company Type (Tier 1, Tier 2 and Tier 3) (based on the Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing as of 2019)4.3 Date of Key Manufacturers Enter into Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market4.4 Global Top Players Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Headquarters and Area Served4.5 Key Players Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Product Solution and Service4.6 Competitive Status4.6.1 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Concentration Rate4.6.2 Mergers & Acquisitions, Expansion Plans 5 Company (Top Players) Profiles and Key Data5.1 BioReliance5.1.1 BioReliance Profile5.1.2 BioReliance Main Business and Companys Total Revenue5.1.3 BioReliance Products, Services and Solutions5.1.4 BioReliance Revenue (US$ Million) (2015-2020)5.1.5 BioReliance Recent Developments5.2 Cobra Biologics5.2.1 Cobra Biologics Profile5.2.2 Cobra Biologics Main Business and Companys Total Revenue5.2.3 Cobra Biologics Products, Services and Solutions5.2.4 Cobra Biologics Revenue (US$ Million) (2015-2020)5.2.5 Cobra Biologics Recent Developments5.3 Oxford BioMedica5.5.1 Oxford BioMedica Profile5.3.2 Oxford BioMedica Main Business and Companys Total Revenue5.3.3 Oxford BioMedica Products, Services and Solutions5.3.4 Oxford BioMedica Revenue (US$ Million) (2015-2020)5.3.5 UniQure Recent Developments5.4 UniQure5.4.1 UniQure Profile5.4.2 UniQure Main Business and Companys Total Revenue5.4.3 UniQure Products, Services and Solutions5.4.4 UniQure Revenue (US$ Million) (2015-2020)5.4.5 UniQure Recent Developments5.5 FinVector5.5.1 FinVector Profile5.5.2 FinVector Main Business and Companys Total Revenue5.5.3 FinVector Products, Services and Solutions5.5.4 FinVector Revenue (US$ Million) (2015-2020)5.5.5 FinVector Recent Developments5.6 MolMed5.6.1 MolMed Profile5.6.2 MolMed Main Business and Companys Total Revenue5.6.3 MolMed Products, Services and Solutions5.6.4 MolMed Revenue (US$ Million) (2015-2020)5.6.5 MolMed Recent Developments5.7 MassBiologics5.7.1 MassBiologics Profile5.7.2 MassBiologics Main Business and Companys Total Revenue5.7.3 MassBiologics Products, Services and Solutions5.7.4 MassBiologics Revenue (US$ Million) (2015-2020)5.7.5 MassBiologics Recent Developments5.8 Richter-Helm5.8.1 Richter-Helm Profile5.8.2 Richter-Helm Main Business and Companys Total Revenue5.8.3 Richter-Helm Products, Services and Solutions5.8.4 Richter-Helm Revenue (US$ Million) (2015-2020)5.8.5 Richter-Helm Recent Developments5.9 FUJIFILM Diosynth Biotechnologies5.9.1 FUJIFILM Diosynth Biotechnologies Profile5.9.2 FUJIFILM Diosynth Biotechnologies Main Business and Companys Total Revenue5.9.3 FUJIFILM Diosynth Biotechnologies Products, Services and Solutions5.9.4 FUJIFILM Diosynth Biotechnologies Revenue (US$ Million) (2015-2020)5.9.5 FUJIFILM Diosynth Biotechnologies Recent Developments5.10 Lonza5.10.1 Lonza Profile5.10.2 Lonza Main Business and Companys Total Revenue5.10.3 Lonza Products, Services and Solutions5.10.4 Lonza Revenue (US$ Million) (2015-2020)5.10.5 Lonza Recent Developments5.11 Aldevron5.11.1 Aldevron Profile5.11.2 Aldevron Main Business and Companys Total Revenue5.11.3 Aldevron Products, Services and Solutions5.11.4 Aldevron Revenue (US$ Million) (2015-2020)5.11.5 Aldevron Recent Developments5.12 Eurogentec5.12.1 Eurogentec Profile5.12.2 Eurogentec Main Business and Companys Total Revenue5.12.3 Eurogentec Products, Services and Solutions5.12.4 Eurogentec Revenue (US$ Million) (2015-2020)5.12.5 Eurogentec Recent Developments5.13 Cell and Gene Therapy Catapult5.13.1 Cell and Gene Therapy Catapult Profile5.13.2 Cell and Gene Therapy Catapult Main Business and Companys Total Revenue5.13.3 Cell and Gene Therapy Catapult Products, Services and Solutions5.13.4 Cell and Gene Therapy Catapult Revenue (US$ Million) (2015-2020)5.13.5 Cell and Gene Therapy Catapult Recent Developments5.14 Biovian5.14.1 Biovian Profile5.14.2 Biovian Main Business and Companys Total Revenue5.14.3 Biovian Products, Services and Solutions5.14.4 Biovian Revenue (US$ Million) (2015-2020)5.14.5 Biovian Recent Developments5.15 Brammer Bio5.15.1 Brammer Bio Profile5.15.2 Brammer Bio Main Business and Companys Total Revenue5.15.3 Brammer Bio Products, Services and Solutions5.15.4 Brammer Bio Revenue (US$ Million) (2015-2020)5.15.5 Brammer Bio Recent Developments5.16 VGXI5.16.1 VGXI Profile5.16.2 VGXI Main Business and Companys Total Revenue5.16.3 VGXI Products, Services and Solutions5.16.4 VGXI Revenue (US$ Million) (2015-2020)5.16.5 VGXI Recent Developments5.17 PlasmidFactory5.17.1 PlasmidFactory Profile5.17.2 PlasmidFactory Main Business and Companys Total Revenue5.17.3 PlasmidFactory Products, Services and Solutions5.17.4 PlasmidFactory Revenue (US$ Million) (2015-2020)5.17.5 PlasmidFactory Recent Developments5.18 bluebird bio5.18.1 bluebird bio Profile5.18.2 bluebird bio Main Business and Companys Total Revenue5.18.3 bluebird bio Products, Services and Solutions5.18.4 bluebird bio Revenue (US$ Million) (2015-2020)5.18.5 bluebird bio Recent Developments5.19 Novasep5.19.1 Novasep Profile5.19.2 Novasep Main Business and Companys Total Revenue5.19.3 Novasep Products, Services and Solutions5.19.4 Novasep Revenue (US$ Million) (2015-2020)5.19.5 Novasep Recent Developments5.20 Spark Therapeutics5.20.1 Spark Therapeutics Profile5.20.2 Spark Therapeutics Main Business and Companys Total Revenue5.20.3 Spark Therapeutics Products, Services and Solutions5.20.4 Spark Therapeutics Revenue (US$ Million) (2015-2020)5.20.5 Spark Therapeutics Recent Developments5.21 Vigene Biosciences5.21.1 Vigene Biosciences Profile5.21.2 Vigene Biosciences Main Business and Companys Total Revenue5.21.3 Vigene Biosciences Products, Services and Solutions5.21.4 Vigene Biosciences Revenue (US$ Million) (2015-2020)5.21.5 Vigene Biosciences Recent Developments 6 North America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing by Players and by Application6.1 North America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size and Market Share by Players (2015-2020)6.2 North America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020) 7 Europe Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing by Players and by Application7.1 Europe Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size and Market Share by Players (2015-2020)7.2 Europe Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020) 8 China Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing by Players and by Application8.1 China Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size and Market Share by Players (2015-2020)8.2 China Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020) 9 Rest of Asia Pacific Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing by Players and by Application9.1 Rest of Asia Pacific Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size and Market Share by Players (2015-2020)9.2 Rest of Asia Pacific Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020) 10 Latin America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing by Players and by Application10.1 Latin America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size and Market Share by Players (2015-2020)10.2 Latin America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020) 11 Middle East & Africa Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing by Players and by Application11.1 Middle East & Africa Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size and Market Share by Players (2015-2020)11.2 Middle East & Africa Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020) 12 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Dynamics12.1 Industry Trends12.2 Market Drivers12.3 Market Challenges12.4 Porters Five Forces Analysis 13 Research Finding /Conclusion 14 Methodology and Data Source 14.1 Methodology/Research Approach14.1.1 Research Programs/Design14.1.2 Market Size Estimation14.1.3 Market Breakdown and Data Triangulation14.2 Data Source14.2.1 Secondary Sources14.2.2 Primary Sources14.3 Disclaimer14.4 Author List

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Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020: Analysis By Regional Outlook, Competitive Landscape, Strategies And...

CHELMSFORD May 1, 2020 (Thomson StreetEvents) -- Edited Transcript of Brooks Automation Inc earnings conference call or presentation Thursday, April 30, 2020 at 8:30:00pm GMT

* Lindon G. Robertson

Brooks Automation, Inc. - Executive VP & CFO

Brooks Automation, Inc. - Director of IR

* Stephen S. Schwartz

* J. Ho

Stifel, Nicolaus & Company, Incorporated, Research Division - MD of Technology Sector

* Jacob K. Johnson

Crdit Suisse AG, Research Division - MD, Global Technology Strategist and Global Technology Sector Head

Janney Montgomery Scott LLC, Research Division - MD, Head of Healthcare Research & Senior Equity Research Analyst

Greetings, and welcome to the Brooks Automation Q2 2020 Financial Results. (Operator Instructions) As a reminder, this conference is being recorded Thursday, April 30, 2020. I would now like to turn the conference over to Mark Namaroff. Please go ahead.

Mark Namaroff, Brooks Automation, Inc. - Director of IR [2]

Thank you, Malaika. Good afternoon everyone on the line today. We hope everyone is staying healthy in this environment. We'd like to welcome you to our earnings conference call for the second quarter of fiscal 2020. Our Q2 earnings press release was issued after the close of the market today and it is available on our Investor Relations website located at brooks.investorroom.com, as are the supplementary PowerPoint slides that we'll be using during the prepared remarks.

I would like to remind everyone that during the course of the call today, we will be making a number of forward-looking statements within the meaning of the Private Litigation and Securities Act of 1995. There are many factors that may cause actual financial results or other events to differ from those identified in such forward-looking statements. I would refer you to the section of our earnings release titled Safe Harbor Statement, the safe harbor slide on our aforementioned PowerPoint presentation on our website, and our various filings with the SEC, including our annual reports on Form 10-K and our quarterly reports on Form 10-Q. We make no obligation to update these statements should future financial data or events occur that differ from the forward-looking statements presented today.

We would also refer to a number of non-GAAP financial measures, which are used in addition to and in conjunction with results presented in accordance with GAAP. We believe non-GAAP measures provide an additional way of viewing aspects of our operations and performance. But when considered with GAAP financial results, the reconciliation of GAAP measures, and a reconciliation of GAAP measures, they provide an even more complete understanding of the Brooks business. Non-GAAP measures should not be relied upon to the exclusion of GAAP measures themselves.

On the call with me today is our President and Chief Executive Officer, Steve Schwartz, and Executive Vice President and Chief Financial Officer, Lindon Robertson. We will open the call up with remarks from Steve on the highlights of the second quarter, and then Lindon will provide a more detailed look into our financial results for the quarter and our outlook for the third fiscal quarter of 2020. We will then have time to take your questions at the end of our prepared remarks.

And with that, now I'd like to turn the call over to our CEO, Steve Schwartz.

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Stephen S. Schwartz, Brooks Automation, Inc. - CEO, President, Interim GM of Life Sciences & Director [3]

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Thank you, Mark, and good afternoon, everyone. We're glad to have you with us today. As we've already published preliminary results in a press release we issued on April 13th, I'll recap those results briefly and focus my remarks on some extra color at the segment level to give you some indication about how we see the current and near term business environment.

I'd like to start by discussing our priorities during this pandemic. First is the safety and wellbeing of our employees and their families as well as our partners including our suppliers and customers. Second, our ability to serve our customers that are deemed essential businesses with specific focus on any and all COVID-19 requests. And there have been many. And finally, the ongoing health of the company for the long term and actions we're taking to ensure that we emerge from this crisis stronger than ever.

With the exceptions of only a few brief work interruptions, all of our 20 plus factories, laboratories and repair centers were up and running throughout Q2 and all are currently operating. Of our approximately 3,300 global employees, about 1,800 have jobs that require them to be onsite. The rest of our employees are working from home. We are particularly proud of the way that each and every employee has embraced the new work practices that include social distancing, the diligent use of PPE, temperature screenings, staggered shifts and other practices which have allowed us to continue serving customers. All have adapted to this new work environment and we could not be more pleased with the resiliency of our operations during these turbulent times.

We consistently met our delivery commitments to customers as they struggled with their supply chains. And in many instances on the Life Sciences side, we stepped up to provide GENEWIZ, Sample Management and Informatic Services to customers who had either lost their regular services providers or who needed new services from us that came about because of the COVID-19 crisis. It's been energizing to bring even more capability to our customers and their need for our services underscores our purpose and mission.

Finally, we're building this company for the long term and our quarter to quarter and year to year revenue and earnings growth speaks to the momentum that we've generated. We've amassed an incredible technology and scientific portfolio, a global presence that allows us to deliver our world class capability to customers around the world, and we possess market positions in trusted brands that are the envy of our competitors. We've accomplished this through the energy and commitment of our global workforce. They know just how to ensure that we deliver on our long-term goals.

There is so much opportunity in front of us and it would be shortsighted of us to slow down now, especially since the capability we're building will be even more valuable in the future. For that reason, we intend to retain our team and fund their activities for technology development, new discovery, market capture, and customer support.

With each day that goes by, we're taking actions and making investments to emerge from the pandemic even stronger and more necessary to our customers. Our decision to continue these investments will have some impact on our earnings performance through the rest of this year, but we believe that it's absolutely the right action to take.

As companies and countries are contemplating how to reemerge from the pandemic, we have a definitive plan in place and a dedicated team that will continue to drive the momentum that we've established.

Now I'll report on Q2. Second quarter performance was strong with revenue of $220 million, up 11% year-over-year with the same 11% growth for each of our Semiconductor and Life Sciences segments. As I mentioned, for the most part our factories and laboratories were running throughout the quarter as we worked to meet customer demand, albeit it with many adjustments to mix because of both supply and demand changes throughout the quarter.

I'll begin with Life Sciences which delivered strong results with $95 million in revenue, up 4% sequentially with growth coming from both GENEWIZ and Sample Management. As each of these subsegments witnessed its own dynamics, I'll cover them separately starting with GENEWIZ.

Once again, GENEWIZ outperformed even our expectations, delivering a 25% year-over-year increase in revenue to $41 million. We were advantaged by our unique capability as both readers and writers of genes which allowed us to overcome some of the curveballs thrown our way. First, in early February when we announced our Q1 results, we told you that except for a team of scientists who remained onsite in support of urgent COVID-19 research, our Suzhou, China operations were basically shut down. It's in our Suzhou site where we perform the vast majority of our gene synthesis. Although we were bracing for what could have been a prolonged slowdown, China operations came back much faster than we'd imagined. By the end of February, more than 90% of our employees were back at work. And even without recovery in academia in China, demand from global customers was strong. In fact, despite the speedbump in February, the March quarter was a record for synthesis at just over $10 million.

Similarly, in next generation sequencing, we also had a record quarter with year-over-year growth of 46% and consistent 7% sequential growth, but with more volatility as the quarter progressed. We saw a normal pattern for NGS demand in the first two months of the quarter and then something of a spike in orders in the first half of March followed by two weeks of lower order volume. As we assess the NGS and synthesis businesses, we believe that much of the Q2 increase was the result of our normal customer capture. At the same time, it appears that some of the March burst came from researchers who no longer had capacity from their core laboratories or were facing reduced productivity from their existing suppliers while GENEWIZ remained at full capability and was eager to serve.

That said, order patterns for April have been steady but slightly lower in both synthesis and NGS but we have mixed signals as customer engagement for both synthesis and NGS projects remains quite active.

In Sanger sequencing, which is a high volume, overnight turns business where we perform millions of measurements for thousands of customers each quarter, revenue was steady for the first 10 weeks of the quarter. But when shelter in place orders closed many academic labs and caused a shutdown of nonessential research, we saw an abrupt reduction of more than 50% of our average daily volumes for the last two weeks of the quarter. And that level has persisted throughout most of April.

Though total Sanger revenue actually decreased slightly quarter-over-quarter, it was still up low single digits from Q2, one year ago. In Q3, we anticipate lower Sanger revenue until academic labs come back online and industry reaccelerates. And although we've seen average daily volumes increase during each of the past 4 weeks, we still remain below 50% of average. So at this point in the quarter, it would be too early to say that we have any concrete signs of a meaningful return to work.

In terms of overall GENEWIZ outlook, we expect business to be slightly down until academic researchers return to their benches and the activities that support clinical trials are back on the upswing. We don't have a prediction for when that will be, but we modeled a gradual return toward more normal volumes as we make our way through the rest of this quarter.

A difficult environment like the one we're in provides opportunities for companies that are prepared. In Q2, customers challenged us with hundreds of COVID-19 projects that gave us opportunities to demonstrate our incredible scientific acumen, round-the-clock collaboration, and fast turn, high quality results in support of their essential research. In addition, we took on many new urgent projects for first time customers. We believe that over the long run, it's this attention to solving customer issues that cement them to us and enabled GENEWIZ to add more than 200 new customers in the quarter.

Now I'll turn to Sample Management which even with all the disruption from coronavirus came in right on forecast I remind you that this year in Sample Management we have two areas of focus. A return to double digit revenue growth and sustainable gross margin improvement. And we're pleased with our progress against these goals. Even in this environment, Q2 was a very solid quarter. Revenue came in at $54 million, up 5% from Q1 and 3% year-over-year. Moreover, gross margin was up another 290 basis points from Q1. We're very encouraged by these results and it gives us confidence that this business is solidly on a path to be able to deliver on our expectations.

At the time we announced Q1 results, we knew two things. That the changes we had made to the organization structure and our focus on performance were set to deliver more growth and keep us on our trajectory for reacceleration of revenue in 2020, and that we were on track to deliver more profitability improvements.

That said, we also mentioned to you that we could foresee approximately $2 million of potential headwind from the coronavirus because of the inability of our field teams to complete system startups and perform revenue generating service on our equipment. Unfortunately, our customers obviously did not open for our employees and we did incur the headwinds that we had built into our forecast.

That said, there were many accomplishments in the quarter and I do want to highlight a few as they relate to trends in the business. Our cryogenics cold-chain products continue to make strong progress. Revenue topped $3 million in the quarter and we shipped B3C Cryo systems to 9 different customers. Six of those were repeat buyers. To date, 3/4 of our systems have been sold into cell and gene therapy applications and the momentum for these automated solutions continues to build.

In spite of the significant slowdown in clinical trial activity in March, Q2 Bio Storage activity generated our largest sample intake quarter since 2017, and we're encouraged by the reinvigoration of our customer capture activities. And in our consumables and instruments business lines, we made fast turn capital addition to respond to increased volume of consumables that will be used for COVID-19 tests as part of packages that we sell to diagnostics companies. This added capacity should allow us to increase revenue in Q3.

All-in, it was a very good quarter for Sample Management. Even with some COVID-19 related delays, revenue growth was right on track, profitability was ahead of schedule, and the result of our deliberate action is something that we'll build upon. The team is energized and active and we plan to come out of the COVID-19 days stronger than ever. What energizes us even more is the amount and level of large customer, large deal activity that's underway. During these days of no travel, our customer engagement activity has moved to the internet and deals that we've been working on for months are still being advanced. We are currently negotiating the final Ts and Cs on two large contracts and we have more multimillion dollar contracts in the pipeline. There opportunities speak to our capabilities and confidence to resume growth and profitability in this business.

On the semiconductor side, our performance in Q2 demonstrates the value of our product portfolio, and because of our close working relationship with our supply chain, we experienced only a small COVID-19 related revenue impact in the quarter which caused no impact to our customers. At $125 million in Q2, we established a new record high for quarterly semiconductor revenue and that's all the more remarkable because as an industry, we are still not back to our highs for semiconductor capital equipment spending. We attribute this outperformance to two factors. The strong high market share position of our Contamination Control Solutions business which satisfies a rapidly growing technology need, and continued design win and market share capture in our equipment automation products for OEM process equipment along with the steadily evolving advanced packaging market.

I'll give some specific color from our major Semiconductor business drivers, tool automation, advanced packaging and Contamination Control Solutions. Our automation products remained strong in the quarter with systems up approximately 10% and robots similar to last quarter's results. We had record bookings for our vacuum robots and vacuum systems, indicating demand remains very strong.

Sales to Chinese equipment manufacturers is picking up and this is a good indicator for both China fab activity and advanced packaging capability. We saw an uptick in advanced packaging in the quarter to just over $13 million or up 30% from Q1. This is still lower than one year ago, but it's the first sequential uptick in the last three quarters and in general a positive indicator. Based on order activity, we do have some indication that advanced packaging opportunities may be staring to percolate, especially in China.

Finally, I'll give a brief update on Contamination Control business which has been extremely strong as we delivered a record $45 million in revenue. The $45 million number is extremely significant for several reasons. First, we truly tested our supply chain and determined it to be very capable. In the first 2 quarters of 2020, we shipped as much revenue as in the last 3 quarters of 2019. And those were already healthy business levels.

Second, we met the demand for the expansion of the 5 Nanometer Foundry ramp on schedule and with very high quality. And finally, we continue to win additional business across a broad range of customers, device technologies that will serve us well as tier one foundry spending subsides.

Revenue in CCS will necessarily be lower in Q3 after two very strong back to back quarters, but still we expect healthy levels of more than $30 million in revenue which will be largely made up of a broader base of customers across different geographies and technology applications.

All-in, we're prepared for another strong quarter in our Semiconductor business. That said, we're also aware of some COVID-19 related supply chain issues that are slowing delivery of some parts. So far, we've been able to navigate through most of these issues, but it remains to be seen what the impact might be on actual demand from our customers. There's currently a lot of speculation as to the outlook for semiconductor processing equipment in 2020. Already our March quarter Semiconductor revenue was up 11% from one year ago and our Q2 Semiconductor book to bill was 1.2 for the quarter on a record revenue quarter. Depending upon June quarter capability of the global supply chain, our backlog and customer demand expectations would allow us to deliver yet another record quarter in June if we do not see any meaningful interruptions.

All-in, we had a very solid second quarter. We adjusted well as changes hit us and all companies. And we know that we'll need to rely on these same adaptive skills in the June quarter which may be even more uncertain. But already, we're well positioned and we're extremely confident in the long-term opportunities that we're diligently winning with all that we're investing today.

That concludes my formal remarks, and I'll turn the call now over to Lindon.

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Lindon G. Robertson, Brooks Automation, Inc. - Executive VP & CFO [4]

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Thank you, Steve. I'd like to refer your attention to the slides on our website, starting with Slide 3. Since Steve hit on these messages in his remarks, I will also be brief on what we see as the headlines of our performance. First, we had excellent momentum that has only modestly slowed by COVID-19 disruptions.

We have performed remarkably well with double digit year-over-year revenue growth in both segments and have established ourselves truly as a top reliable supplier in every market we serve.

Next, we expanded earnings substantially with continued performance enhancements in the Life Science segment. To put a finer point on it, gross margins in the Sample Management business are up nearly 600 basis points from a year ago. And third, we are well equipped for what lies ahead. Our liquidity is strong with $198 million of net cash on the balance sheet and healthy cash generation. To this point, our year-to-date cash flow from operations when you exclude the taxes paid on the sale of the semi cryo business, are $51 million. This a year-to-date improvement of $29 million over the prior year. The business is healthy and both sides are contributing growth, profit and cash flow.

Let's move onto Slide 4 to review the overall P&L. Let me first point you to the GAAP earnings and highlight what is different in that comparison. The growth of revenue and operating income is similar to that shown in the Non-GAAP profile on the right side. But what is different on the left side is in the tax line. If the first fiscal quarter, as guided under GAAP, we recognized a windfall tax benefit of approximately $6 million which is related to long term incentive stock units divested in the first quarter. That allowed deduction resulted in a net tax benefit of $3 million for the first fiscal quarter that in the second quarter is back to normal with the $3 million tax expense. The $6 million swing offsets the positive operating performance picture in the GAAP Profile.

In the Non-GAAP results, we apply this windfall to the projected tax rate and take it across the quarters of the fiscal year so that you can use the financials for a level performance indicator.

Continuing with the Non-GAAP performance on the right side, you can see the performance supported 48% year-to-year growth in earnings per share. Starting at the top, the revenue growth came similarly from each business with each growing 11% year to year. We saw a nice uptick in gross margins of 60 basis points sequentially, but Semiconductor gross margins remained approximately level from Q1 as expected. And the uptick was driven by Life Sciences improvement. On a year-over-year basis, the dynamics are also similar with improvement in Life Sciences and a bit softer margins in semiconductor. But all-in, gross margins continue to make progress and contribute to the bottom line.

Operating expenses during the quarter were up. On a sequential basis, the increase was in R&D for our Semiconductor business. Our engineering team has been kept busy throughout the COVID-19 environment, committed to advancing the projects with customers. On a year-over-year basis, operating expense was $7 million higher, driven by both R&D and SG&A. In the SG&A line, the growth was driven primarily around investments in Life Sciences including our IT transition to a single cloud based ERP platform, GENEWIZ growth, and some additional structure from our recent acquisition of RURO Software.

While we are spending some extra to keep our team in a safe environment and onboard with us through the COVID crisis, we anticipate reductions in travel expenses and G&A which will result in lower operating expense by approximately $1 million to $2 million in the third quarter. You can see operating margin expansion. Combining the growth with the margin expansion, you can also see Non-GAAP net income grew 51% year-over-year and expanded 11% sequentially.

Moving to below the operating income line, net interest expense was $600,000, lower by about $7 million compared with last year when we were carrying debt associated with the GENEWIZ acquisition. The non-GAAP tax rate for the quarter came in at about 23%, very consistent with our expectation of 21% to 25% for the year.

Let's turn now over to Slide 5 to discuss the segment results, starting with Life Sciences. In the second quarter, Life Sciences revenue grew 11% to $95 million compared with the second quarter last year. On an organic basis, Life Sciences grew 11% as well. This quarter was the first full year-over-year comparison which includes a full quarter of GENEWIZ revenue since the acquisition of the business in November of 2018. GENEWIZ had a strong second quarter with a revenue of $41 million, growing 25% from last year despite the impacts of COVID-19 as Steve addressed. As a reminder, our GENEWIZ China operations were largely closed down and observed strict quarantine restrictions from that Chinese New Year through February 10th. The exception to that were the labs from which GENEWIZ provided gene synthesis support for COVID-19 research customers. We estimated that the shutdown had an impact of approximately $2 million in the quarter. We are currently fully operational and demand from the China market has largely returned.

Sample Management provided 3% organic growth during the quarter in line with our expectation when we started the quarter. We had described and we did see about $2 million headwind from COVID-19 in the large store systems and post warranty services. This was due to some delays in an onsite installation and general lack of access to customer sites. But there were bright spots in Sample Management as well. Year-over-year growth was driven by cryo which more than doubled, and more substantively, Bio Storage services grew 7%. We have been very encouraged by the continued significant engagements and wins by the teams in the large storage business and in Bio Storage during this environment, fueling the path to return to double digit growth.

In addition, during the second quarter we acquired the Life Science Informatics Software firm RURO. RURO provides cloud-based software solutions to manage the laboratory workflow and bio sample data for a broad range of customers in the biotech, healthcare and pharmaceutical sectors. The business contributed approximately half a million of revenue in the time that we've owned them since mid-February.

As we announced in a March press release effective April 1st, we reorganized our Life Sciences business. We've combined the Bio Storage services with GENEWIZ, leveraging the common lab services and sample handling capabilities. This business combined is now $64 million of this quarter's $95 million business and showed year-to-year growth on that basis of 17%.

The remaining portion of the segment of Life Sciences is the Life Sciences products which was about $31 million and was up 1% year-over-year. If we had avoided the negative impact of the COVID-19 constraints, we estimate that the Life Science Products business would have grown about 4%.

Another bright spot in the quarter was on the gross margin line. The segment improved nicely in the second quarter up to 45.8%, up 330 basis points compared with last year. The year-over-year improvement was driven by the performance in Sample Management. In fact, we had improvement on every area of the business including our large TwinBank's store systems and services, the cryo systems, the consumables and instruments, and the Bio Storage services. This is driven by the performance improvements and cost reductions and the improved price management that's been implemented.

The growth of the segment and gross margin expansion covered the increased operating expense and drove 260 basis points of incremental operating margin year-over-year. The leverage of this business model and growth is fully evident on this page.

As we look into the third quarter, we are more cautious due to the continued and varied impacts of COVID-19. As Steve described, we've seen inside GENEWIZ sustained demand in synthesis, some volatility in the next generation sequencing, and a lower run rate from Sanger sequencing. Most notable to use is that continued absence of the academic research institutions and we have seen only a portion of the commercial teams return to full active status. And in Sample Management, it is unclear when customer sites will fully open around the world, limiting our access for installations and services. So at this point, we're expecting Life Sciences to deliver revenue in the range of $85 million to $91 million which is about 5% to 10% lower quarter-to-quarter.

Let's turn over to Slide 6 to review our Semiconductor business. Semiconductor Solutions revenue was $125 million for the second quarter, an increase of 5% sequentially and 11% year-over-year. If you were to look into the details of the quarter-to-quarter, you would see an uptick in the systems business which we ship primarily to tier two OEMs. I would highlight that the strength in that line in is the atmospheric systems as we expected. We do expect to see more vacuum systems coming in the second half which should assist gross margin.

The real story in the revenue line however is the strength of the Contamination Control Solutions which indeed ships to end user fabs. As referenced, these are record quarters for us in CCS and we continue to see expansion of qualifications and orders across many fabs. The rest of the Semiconductor story is in robots. While it is stable quarter-to-quarter, it is significantly higher year-over-year and our tier one OEM customers continue to call for additional output as we head into our second half.

It may appear we face no challenges in the second quarter, but in fact, the accomplishment reflects much diligence to coordinate supply availability, factory production and of course customer needs. We estimate that the negative impact to COVID-19 on revenue during the quarter for Semiconductor was approximately $5 million due to supply chain and the services challenges. On the positive side, these are delayed, not lost opportunities and due to the coordination efforts, our customers were not surprised nor disappointed by us.

Gross margins in Semi were similar to the prior quarter at about 40% and operating margins improved by 70 basis points. As mix improves in the second half, we expect gross margins to strengthen and if the market holds, growth will bring operating margins back upward on the leverage of the model.

Similar to the Life Science business, we're cautious about the outlook for the third quarter. We expect our supply chain will continue to face some issues. We also are sensitive that the broader supply chain of our customers aside from us affects the end user's timing for when they need our products. Finally, we know the impact of COVID-19 on the end markets can change rapidly. We expect our semiconductor business to deliver revenue in the range of $115 million to $124 million. This is approximately flat to lower -- I'm sorry, flat to down 8% quarter-to-quarter.

Let's turn now over to Slide 7 for a summary of our cash flow over the quarter. We generated $26 million of adjusted operating cash flow during the quarter. This excludes $92 million of income tax payments associated with a gain of the 2019 sale of our semiconductor cryo business. We also used approximately $16 million of cash for the acquisition of RURO as mentioned previously.

Our CapEx amounted to $12 million for the quarter, driven primarily by investments in operations and it includes approximately $1 million for the new GENEWIZ operation facility in Suzhou, China. Total uses of cash during the quarter totaled $105 million, and the change in net cash and equivalents during the quarter brings our cash, restricted cash and cash equivalents balance down to $249 million. The highlight here is the cash capability of the business we have built with GENEWIZ and subsequent to the divestiture of the semi cryo business. Year-to-date, as I said before, our operating cash flow when you exclude the tax on the gain in the sale, was $51 million or an increase of $29 million year-over-year.

On Slide 8, you will see a summary of the balance sheet. You can see at the top of the March 31st column we now carry $249 million in cash and marketable securities. With $51 million of debt, we have $198 million of net cash for operations and investments. This strong position provides us the fuel to invest for growth and gives us the confidence we will weather these uncertain times, and customers and our employees the confidence that we will sustain our investments.

Let's turn now to the summary on Slide 9. Fundamentals of the business are intact and we've gained customers and market share during these times. We have extended our leadership and continue preparing ourselves to be ready to accelerate when the recovery of the market. The balance sheet is strong, provides us the fuel for the journey and the strategic flexibility. We have seen growth momentum in each business. In Semiconductor we saw strong orders and have healthy backlog. But we also know the WFE CapEx environment comes down to the path which the chip makers decide to take. And in this time in particular, the OEM customers depend on a broad supply chain that is vulnerable.

Our Life Sciences business continues to win new business. The active markets of pharma, biotech and clinical continue to engage on improving their infrastructure with our capabilities and to request our support for the COVID research that they have stepped up to do themselves. So meanwhile, the academic research institutions have not returned to work yet and the timing of that return is uncertain and varied across the regions. So we find ourselves with unusually mixed signals. Good customer demand momentum, but uncertain market continuity in the near term. We have solid backlog but uncertain access to some customer locations. But there is one aspect that is very clear to us. We have the momentum with customers and the opportunity to delight them. If we keep that momentum with us, we will maintain our resources and be ready for the ramp as markets do fully return. In the short term, we are carrying some costs. We have some elements of our business less active during this time, but we're going to keep the team employed. We are providing premium pay for onsite labor, ensuring the PPE and clean facilities make a safe environment and expediting shipments to satisfy stressed delivery schedules. We will carry some so this cost in the short term and we will win additional customers as we are there for them throughout and we will be prepared for the full recovery ramp as the market returns to working order.

Let's cover that guidance for the third fiscal quarter of 2020. Revenue is expected to be in the range of $200 million to $215 million. Adjusted EBITDA is anticipated to be $26 million to $34 million and non-GAAP diluted earnings per share to be $0.16 to $0.24 per share. GAAP will be in the range of $0.04 to $0.12. For Life Sciences we factored in a return of the markets in the June month. This now concludes our prepared remarks, and I'll turn the call back over to Malaika, the Operator, to take questions from the line.

================================================================================

Questions and Answers

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Operator [1]

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(Operator Instructions) Our first phone question is from the line of Patrick Ho with Stifel. Please go ahead, your line is open.

--------------------------------------------------------------------------------

J. Ho, Stifel, Nicolaus & Company, Incorporated, Research Division - MD of Technology Sector [2]

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Thank you very much and hope everyone is well. And congrats on a really nice quarter in these challenging circumstances. Steve, maybe first off, in terms of the disruptions and the volatilities you've been seeing, can you give a little bit of color on your end how you were able to manage a very fluid environment and ensure that your top tier customers got what they needed? What were some of the steps you took? How did the business continuity plan work? If you could just give a little bit of color on that first.

--------------------------------------------------------------------------------

Stephen S. Schwartz, Brooks Automation, Inc. - CEO, President, Interim GM of Life Sciences & Director [3]

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Sure, Patrick, and thanks for the question on this. Specifically, on the Semi side, we give a lot of credit to the large OEM customers. They give us a good look at the business. And they adjust it quite frequently, so we're able to get out in front of the supply chain. And then our team managed the supply base extremely well. I think this is something over the past years that's become really fluid. When we get a signal from our tier one OEMs, we're able to transmit that quickly to our suppliers to get them prepared and I think the team managed it really well. It wasn't without problems for sure, but everybody knew how to accelerate and to get the job done. And Patrick, inside the company, everybody knows that deliveries to the customers are top priority, so if we go overtime, we spend. If we do something to get shipments there, we do it. And so I think it's a successful model that played out through the COVID period. And I think the business continuity plans that we have in place, we kicked them into action and I think they played out extremely well. I don't know that anybody could have anticipated the magnitude of what everybody was going to be faced with. But I think the team responded well. We're functioning completely differently just even two weeks into the shutdown period. Two weeks into the shutdown period we were operating with the same fluidity, but in a really different mode. So in general, the things that should have happened did. And we continue to manage going forward the supply base. There are air pockets we're finding coming from India, from Malaysia that we've been trying to work through now for the past 5 weeks. I think we're doing very well, but when you hear uncertainty from everybody in and around the supply chain, I think we're all dealing with the same kinds of things. But I do believe the team is in front of it and we seem to be weathering it pretty well.

--------------------------------------------------------------------------------

J. Ho, Stifel, Nicolaus & Company, Incorporated, Research Division - MD of Technology Sector [4]

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Edited Transcript of BRKS earnings conference call or presentation 30-Apr-20 8:30pm GMT - Yahoo Finance

In this report, the global Cancer Gene Therapy market is valued at USD XX million in 2019 and is projected to reach USD XX million by the end of 2025, growing at a CAGR of XX% during the period 2019 to 2025.

Persistence Market Research recently published a market study that sheds light on the growth prospects of the global Cancer Gene Therapy market during the forecast period (20XX-20XX). In addition, the report also includes a detailed analysis of the impact of the novel COVID-19 pandemic on the future prospects of the Cancer Gene Therapy market. The report provides a thorough evaluation of the latest trends, market drivers, opportunities, and challenges within the global Cancer Gene Therapy market to assist our clients arrive at beneficial business decisions.

The Cancer Gene Therapy market report firstly introduced the basics: definitions, classifications, applications and market overview; product specifications; manufacturing processes; cost structures, raw materials and so on. Then it analyzed the worlds main region market conditions, including the product price, profit, capacity, production, supply, demand and market growth rate and forecast etc. In the end, the Cancer Gene Therapy market report introduced new project SWOT analysis, investment feasibility analysis, and investment return analysis.

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Resourceful insights enclosed in the report:

The major players profiled in this Cancer Gene Therapy market report include:

Some of the major companies operating in the global cancer gene therapy market are Cell Genesys, Advantagene, GenVec, BioCancell, Celgene and Epeius Biotechnologies. Other leading players in cancer gene therapy market include Introgen Therapeutics, ZIOPHARM Oncology, MultiVir and Shenzhen SiBiono GeneTech

Key points covered in the report

For any queries get in touch with Industry Expert @ https://www.persistencemarketresearch.co/ask-an-expert/2988

The market report addresses the following queries related to the Cancer Gene Therapy market:

The study objectives of Cancer Gene Therapy Market Report are:

To analyze and research the Cancer Gene Therapy market status and future forecast in United States, European Union and China, involving sales, value (revenue), growth rate (CAGR), market share, historical and forecast.

To present the Cancer Gene Therapy manufacturers, presenting the sales, revenue, market share, and recent development for key players.

To split the breakdown data by regions, type, companies and applications

To analyze the global and key regions Cancer Gene Therapy market potential and advantage, opportunity and challenge, restraints and risks.

To identify significant trends, drivers, influence factors in global and regions

To analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the Cancer Gene Therapy market.

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Coronavirus (COVID-19) Business Impact Mounting Prices of Coupled With Onset of Cost-effective Alternatives to Fuel the Growth of the Cancer Gene...

MILAN, Italy--(BUSINESS WIRE)--Genespire, a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, announced today the successful close of a 16M Series A financing from Sofinnova Partners, a leading European life sciences venture capital firm based in Paris, London and Milan. The company also announced the appointment of Julia Berretta, Ph.D., as Chief Executive Officer and member of the Board of Directors. Graziano Seghezzi, Managing Partner at Sofinnova Partners, and Lucia Faccio, Ph.D., Partner at Sofinnova Partners, will also join the Board.

Genespire was founded in March 2020 as a spin-off of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), one of the worlds leading cell and gene therapy research institutes spearheaded by gene therapy pioneer Prof. Luigi Naldini. The Company was co-founded by Fondazione Telethon and the San Raffaele Hospital, along with Prof. Naldini and Dr. Alessio Cantore.

The funds will be used to advance Genespires leading-edge platform technologies towards the development of novel gene therapies in two main areas: primary immunodeficiencies and metabolic genetic diseases.

Our mission has always been to develop breakthrough solutions for genetic diseases, said Prof. Naldini, Genespires co-founder and Director of SR-Tiget. This financing enables the company to translate our innovative science and early stage programs into clinical development. The appointment of Dr. Berretta as CEO is a major reinforcement of our team.

Sofinnova Partners Dr. Faccio added, Genespire is an exciting investment with all the key ingredients for success: Outstanding scientists that developed the first ex-vivo gene therapy to market, experienced executives brought in through Sofinnova Partners network and game changing technologies that have the potential to impact the lives of patients with genetic diseases.

I am thrilled to be joining Genespire and such exceptional scientific founders, said Dr. Berretta. Genespire was born of decades of experience in the gene therapy field, and is optimally positioned to advance transformative therapies for patients affected by severe inherited diseases.

Dr. Berretta was part of the Executive Committee of Cellectis S.A., a Nasdaq-listed clinical stage gene editing company developing CAR-T cell therapies for cancer, where she led business development as well as strategic planning. She is also an independent Board member of Treefrog Therapeutics, an innovative stem cell company.

About Genespire

Genespire is a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, particularly primary immunodeficiencies and inherited metabolic diseases. Based in Milan, Italy, Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini, Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. It is a spin-off of SR-Tiget, a world leading cell and gene therapy research institute and is backed by Sofinnova Partners. http://www.genespire.com

About Sofinnova Partners

Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Based in Paris, France, with offices in London and Milan, the firm brings together a team of 40 professionals from all over Europe, the U.S. and Asia. The firm focuses on paradigm-shifting technologies alongside visionary entrepreneurs. Sofinnova Partners invests across the Life Sciences value chain as a lead or cornerstone investor, from very early-stage opportunities to late-stage/public companies. It has backed nearly 500 companies over more than 48 years, creating market leaders around the globe. Today, Sofinnova Partners has over 2 billion under management.

For more information, please visit: http://www.sofinnovapartners.com

About Fondazione Telethon

Fondazione Telethon is a non-profit organisation created in 1990 as a response to the appeals of a patient association group of stakeholders, who saw scientific research as the only real opportunity to effectively fight genetic diseases. Thanks to the funds raised through the television marathon, along with other initiatives and a network of partners and volunteers, Telethon finances the best scientific research on rare genetic diseases, evaluated and selected by independent internationally renowned experts, with the ultimate objective of making the treatments developed available to everyone who needs them. Throughout its 30 years of activity, Fondazione Telethon has invested more than 528 million in funding more than 2.630 projects to study more than 570 diseases, involving over 1.600 scientists. Fondazione Telethon has made a significant contribution to the worldwide advancement of knowledge regarding rare genetic diseases and of academic research and drug development with a view to developing treatments. For more information, please visit: http://www.telethon.it

About Ospedale San Raffaele

Ospedale San Raffaele (OSR) is a clinical-research-university hospital established in 1971 to provide international-level specialised care for the most complex and difficult health conditions. OSR is part of Gruppo San Donato, the leading hospital group in Italy. The hospital is a multi-specialty center with over 60 clinical specialties; it is accredited by the Italian National Health System to provide care to both public and private, national and international patients. Research at OSR focuses on integrating basic, translational and clinical activities to provide the most advanced care to our patients. The institute is recognized as a global authority in molecular medicine and gene therapy, and is at the forefront of research in many other fields. Ospedale San Raffaele is a first-class institute which treats many diseases and stands out for the deep interaction between clinical and scientific area. This makes the transfer of scientific results from the laboratories to the patients bed easier. Its mission is to improve knowledge of diseases, identify new therapies and encourage young scientists and doctor to grow professionally. For more information, please visit: http://www.hsr.it

About the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele and Fondazione Telethon. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases.

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Genespire Secures 16 Million Series A Financing from Sofinnova Partners to Advance Transformative Gene Therapies - Business Wire

The Gene Therapy for Ovarian Cancer report provides independent information about the Gene Therapy for Ovarian Cancer industry supported by extensive research on factors such as industry segments size & trends, inhibitors, dynamics, drivers, opportunities & challenges, environment & policy, cost overview, porters five force analysis, and key companies profiles including business overview and recent development.

Gene Therapy for Ovarian Cancer MarketLatest Research Report 2020:

Download Premium Sample Copy Of This Report: Download FREE Sample PDF!

In this report, our team offers a thorough investigation of Gene Therapy for Ovarian Cancer Market, SWOT examination of the most prominent players right now. Alongside an industrial chain, market measurements regarding revenue, sales, value, capacity, regional market examination, section insightful information, and market forecast are offered in the full investigation, and so forth.

Scope of Gene Therapy for Ovarian Cancer Market: Products in the Gene Therapy for Ovarian Cancer classification furnish clients with assets to get ready for tests, tests, and evaluations.

Major Company Profiles Covered in This Report

Takara Bio, VBL Therapeutics, CELSION, Targovax

Gene Therapy for Ovarian Cancer Market Report Covers the Following Segments:

Segment by Type:

IntravenousIntratumoralIntraperitoneal

Segment by Application:

Ovarian Cancer (unspecified)Recurrent Ovarian Epithelial CancerPlatinum-Resistant Ovarian Cancer

North America

Europe

Asia-Pacific

South America

Center East and Africa

United States, Canada and Mexico

Germany, France, UK, Russia and Italy

China, Japan, Korea, India and Southeast Asia

Brazil, Argentina, Colombia

Saudi Arabia, UAE, Egypt, Nigeria and South Africa

Market Overview:The report begins with this section where product overview and highlights of product and application segments of the global Gene Therapy for Ovarian Cancer Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company:Here, the competition in the Worldwide Gene Therapy for Ovarian Cancer Market is analyzed, By price, revenue, sales, and market share by company, market rate, competitive situations Landscape, and latest trends, merger, expansion, acquisition, and market shares of top companies.

Company Profiles and Sales Data:As the name suggests, this section gives the sales data of key players of the global Gene Therapy for Ovarian Cancer Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the global Gene Therapy for Ovarian Cancer Market.

Market Status and Outlook by Region:In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the global Gene Therapy for Ovarian Cancer Market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User:This section of the research study shows how different end-user/application segments contribute to the global Gene Therapy for Ovarian Cancer Market.

Market Forecast:Here, the report offers a complete forecast of the global Gene Therapy for Ovarian Cancer Market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Research Findings and Conclusion:This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

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Cover Corona Outbreak: Gene Therapy for Ovarian Cancer Market Size Analysis and Growth (2020-2025) - Cole of Duty

DUBLIN, April 30, 2020 /PRNewswire/ -- The "SRP-9001 - Emerging Insight and Market Forecast - 2030" report has been added to ResearchAndMarkets.com's offering.

The report provides comprehensive insights about an investigational product for Duchene muscular dystrophy (DMD) in 7 Major Markets.

A detailed picture of the SRP-9001 in Seven Major Markets, i.e., United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan, for the study period 2020-2030 is provided in this report along with a detailed description of the product.

The product details cover mechanism of action, dosage and administration, route of synthesis, and research and development activity including regulatory milestones, and other development activities. Further, it also consists of future market assessments inclusive of the market forecast, SWOT analysis, market competitors, and other emerging therapies.

Overview

SRP-9001 is being developed by Sarepta Therapeutics which is currently in Phase II clinical development for the treatment of Duchenne muscular dystrophy. It is designed to deliver the micro-dystrophin-encoding gene directly to the muscle tissue for the targeted production of the micro-dystrophin protein. Roche has acquired exclusive commercial rights outside the United States to Sarepta Therapeutics' lead gene therapy pipeline candidate SRP-9001 for Duchenne muscular dystrophy (DMD).

The report provides insights into:

Report Highlights

Key Questions Answered

Key Topics Covered

1. Drug Overview1.1. Product Detail1.2. Mechanism of Action1.3. Dosage and Administration1.4. Research and Development Activity1.4.1. Clinical Development1.4.2. Safety and Efficacy1.5. Other Development Activities

2. Market Assessment2.1. 7MM Market Analysis2.2. The United States Market2.3. Germany Market2.4. France Market2.5. Italy Market2.6. Spain Market2.7. United Kingdom Market2.8. Japan Market

3. SWOT Analysis

4. Analyst Views

5. Market Competitors

6. Other Emerging Therapies

List of TablesTable 1 SRP-9001, DescriptionTable 2 SRP-9001, Clinical Trial DescriptionTable 3 SRP-9001, 7MM Market Size from 2020 to 2030 (in Million USD)Table 4 Market CompetitorsTable 5 Other Emerging Therapies

List of FiguresFigure 1 The Development Timeline of SRP-9001 Figure 2 Patent Details, SRP-9001 Figure 3 SRP-9001, 7MM Market Size from 2020 to 2030 (in Million USD)Figure 4 SRP-9001, US Market Size from 2020 to 2030 (in Millions USD)Figure 5 SRP-9001, EU5 Market Size from 2020 to 2030 (in Millions USD)Figure 6 SRP-9001, Japan Market Size from 2020 to 2030 (in Millions USD)

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SRP-9001 [Sarepta Therapeutics] Emerging Insights and Market Forecast to 2030 - P&T Community

A cancer researcher at the University of Alberta and his team are breathing new life into a promising cancer therapy by improving how the treatment is delivered.

Hasan Uludag, a professor in chemical and materials engineering, and his team have developed a chemical compound that allows them to effectively inject genetic material into cells.

The compound, a lipopolymer, prompts the cells to begin producing a proteintumour necrosis factor-related apoptosis-inducing ligand (TRAIL)that has been proven to kill tumour cells effectively.

The TRAIL protein has been used in treatment but led to many side-effects. The addition of the compound, which uses the positive cancer-killing aspect of the TRAIL protein, eliminates the side-effects.

When you have this combination (of the protein and the compound), the cells are ready, they are primed for the action of this protein, so when they see TRAIL they display this high potency and they die much more easily, said Uludag.

With the new delivery method, Uludag, a member of the Cancer Research Institute of Northern Alberta, also addressed the issue of cell resistance in TRAIL therapy.

Though the treatment was somewhat effective with previous delivery methods, about 10 per cent of cancerous cells survived and eventually developed resistance to the treatment. With the new lipopolymer delivery method, researchers can find alternate targets within the cell and break that resistance, killing the remainder of tumour cells.

The new approach also holds possibilities for individualized therapeutics. Common therapeutics used today are not customizable, and though things like dosage can be changed as appropriate, ultimately, if the cells resist a drug or it proves ineffective, there is no option but to completely switch the drug and try another treatment. With an approach that uses genetic materials, alterations can be made to the order of materials in the DNA strand, known as the nucleotide sequence, to target different proteins.

The nature of the drug stays the same, the delivery, the application stays the same, you just change the siRNA, said Uludag. In terms of tailoring the therapy for patients, what we are proposing is much more possible to personalize compared to conventional ways for treating cancer patients.

The next steps in this co-delivery approach are to reproduce the studies on a larger scale and do a parallel set of studies to specifically examine whether the new therapeutic is safe, not just whether it works.

Different genetic material combinations could be used to target different types of breast cancer cells as well as other types of cancer.

Some of the old drugs we gave up, I think, are promising and might provide us with new ways to combat cancer, Uludag said.

Uludags spinoff company, RJH Biosciences, recently received a $100,000 grant from the Bill and Melinda Gates Foundation for a related project exploring how to better deliver therapeutics to target blood cancer by developing particular delivery systems.

The study, Breathing New Life into TRAIL for Breast Cancer Therapy: Co-Delivery of pTRAIL and Complementary siRNAs Using Lipopolymers, was published in Human Gene Therapy.

Originally posted here:
Cancer researchers find more effective way to deliver promising breast cancer treatment - Folio - University of Alberta

The research report on Gene Therapy Market provides comprehensive analysis on market status and development pattern, including types, applications, rising technology and region. Gene Therapy Market report covers the present and past market scenarios, market development patterns, and is likely to proceed with a continuing development over the forecast period. The report covers all information on the global and regional markets including historic and future trends for market demand, size, trading, supply, competitors, and prices as well as global predominant vendors information.

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This market research report on the Gene Therapy Market is an all-inclusive study of the business sectors up-to-date outlines, industry enhancement drivers, and manacles. It provides market projections for the coming years. It contains an analysis of late augmentations in innovation, Porters five force model analysis and progressive profiles of hand-picked industry competitors. The report additionally formulates a survey of minor and full-scale factors charging for the new applicants in the market and the ones as of now in the market along with a systematic value chain exploration.

An outline of the manufacturers active within the Gene Therapy Market, consisting of

Sibiono GeneTech,Advantagene,Spark Therapeutics,Shanghai Sunway Biotech Co. LtdBluebird Bio,UniQure NVAvalanche Bio,Celladon,Sangamo,Dimension Therapeutics

The Gene Therapy Market Segmentation by Type:

Viral vectorNon-viral vector

The Gene Therapy Market Segmentation by Application:

Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

Market Segment by Regions, regional analysis covers

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The competitive landscape of the Gene Therapy Market is discussed in the report, including the market share and new orders market share by company. The report profiles some of the leading players in the global market for the purpose of an in-depth study of the challenges faced by the industry as well as the growth opportunities in the market. The report also discusses the strategies implemented by the key companies to maintain their hold on the industry. The business overview and financial overview of each of the companies have been analyzed.

This report provide wide-ranging analysis of the impact of these advancements on the markets future growth, wide-ranging analysis of these extensions on the markets future growth. The research report studies the market in a detailed manner by explaining the key facets of the market that are foreseeable to have a countable stimulus on its developing extrapolations over the forecast period.

Key questions answered in this research report:

Table of Contents:

Global Gene Therapy Market Research Report

Chapter 1 Gene Therapy Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

.CONTINUED FOR TOC

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Global Gene Therapy Market Size, Share, Growth, Revenue, Global Industry Analysis and Future Demand And Forecast To 2020-2026 Cole Reports - Cole of...

R.A. Session II, president, CEO, and founder of Taysha Gene Therapies [Background image: Olena Yepifanova via iStock]

Dallas-based biotech Taysha Gene Therapies has emerged out of stealth with a $30 million seed round and a new strategic partnership with UT Southwestern. The company aims to end severe and life-threatening diseases of the central nervous system caused by variation in a single gene, also known as monogenic CNS diseases.

Hitting the ground running, the company launches with a pipeline of 15 adeno-associated virus gene therapy programs, along with options to four others.

Taysha plans human testing this year and expects to file four Initial New Drug applications in 2021. In addition, the biotech is developing an improved treatment delivery platform that uses machine learning, DNA shuffling, and something it calls directed evolution.

The gene therapy startup closed its round of seed financing in early March just as the Dow dropped a couple of thousand points, according to a report in EndpointsNews.

If you had to ask me, was I worried? Absolutely. I think I wouldnt be human if I wasnt, RA Session II, president, CEO, and founder of Taysha, told the healthcare publication.

Session had a lot of certainty to balance out all the uncertainty of launching a new biotech during a pandemic, noted Endpoints Amber Tong.

The experience of a proven management team, including some who previously led the development and commercialization of the first FDA-approved gene therapy for CNS disease, Zolgensma, is part of the equation.

The startup intends to combine the speed, scale, and expertise of its partners UT Southwestern Gene Therapy Program with the experience of the Taysha team.

Session says the launch reunites former investors and executives from AveXis. Tayshas funding round was co-led by PBM Capital, the first institutional investor in AveXis, and Nolan Capital, the investment fund of former AveXis CEO Sean Nolan.

Tayshas Board of Directors played a key role in the formation of the company, it said. The board is comprised of Sean Nolan, Paul Manning of PBM Capital, Claire Aldridge, Ph.D., of UT Southwestern, and RA Session II, President, CEO, and Founder of Taysha.

Nolan, who serves as Chairman of the Board, sees promise in Tayshas approach for patients suffering from many devastating CNS diseases.

This is an exciting time for gene therapy, he said.

The strategic partnership with UT Southwestern could help Taysha to rapidly and efficiently translate novel AAV gene therapies from bench to bedside, the company said.

UTSW is home to some of the brightest minds in gene therapy, Sessions says. By joining forces with UT Southwestern, Taysha can advance its potential cures with both speed and scale.

The collaboration is ultimately creating an engine for new cures, said Claire Aldridge, Ph.D., a Taysha board member and associate vice president of commercialization and business development at UT Southwestern Medical Center.

Together, Taysha and UTSW will merge cutting-edge translational research, hands-on clinical care, and expertiseboth regulatory and commercial, she said.

Aldridge has already witnessed how quickly and efficiently the partners can leverage their collective expertise and resources. That means getting new gene therapies to the patients who so desperately need them, she said.

The UT Southwestern Gene Therapy Program, led by Steven Gray, Ph.D., Director of the Viral Vector Core and Assistant Professor in the Department of Pediatrics, and Berge Minassian, M.D., Division Chief of Child Neurology, has the capacity to support Tayshas wide range of preclinical and clinical development programs, the company said.

Under the partnership, UT Southwestern, which has developed a state-of-the-art viral vector manufacturing facility, will run discovery and preclinical research, as well as lead studies, provide manufacturing, and execute natural history studies.

For its part, Taysha will lead all clinical development, regulatory strategy, commercial manufacturing, and commercialization activities, according to the company.

A joint steering committee of key leadership members from Taysha and UT Southwestern will govern the collaboration.

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Dallas Gene Therapy Startup Launches with $30M Seed Round and a UT Southwestern Partnership - dallasinnovates.com

Documenting the Industry Development of Gene Therapy Market concentrating on the industry that holds a massive market share 2020 both concerning volume and value With top countries data, Manufacturers, Suppliers, In-depth research on market dynamics, export research report and forecast to 2029

As per the report, the Gene Therapy Market is anticipated to gain substantial returns while registering a profitable annual growth rate during the predicted time period.The global gene therapy market research report takes a chapter-wise approach in explaining the dynamics and trends in the gene therapy industry.The report also provides the industry growth with CAGR in the forecast to 2029.

A deep analysis of microeconomic and macroeconomic factors affecting the growth of the market are also discussed in this report. The report includes information related to On-going demand and supply forecast. It gives a wide stage offering numerous open doors for different businesses, firms, associations, and start-ups and also contains authenticate estimations to grow universally by contending among themselves and giving better and agreeable administrations to the clients. In-depth future innovations of gene therapy Market with SWOT analysis on the basis Of type, application, region to understand the Strength, Weaknesses, Opportunities, and threats in front of the businesses.

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***[Note: Our Complimentary Sample Report Accommodate a Brief Introduction To The Synopsis, TOC, List of Tables and Figures, Competitive Landscape and Geographic Segmentation, Innovation and Future Developments Based on Research Methodology are also Included]

An Evaluation of the Gene Therapy Market:

The report is a detailed competitive outlook including the Gene Therapy Market updates, future growth, business prospects, forthcoming developments and future investments by forecast to 2029. The region-wise analysis of gene therapy market is done in the report that covers revenue, volume, size, value, and such valuable data. The report mentions a brief overview of the manufacturer base of this industry, which is comprised of companies such as- Bluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical Inc, Advantagene.

Segmentation Overview:

Product Type Segmentation :

Ex vivo, In Vivo

Application Segmentation :

Cancer, Monogenic, Infectious disease, Cardiovascular disease

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Key Highlights of the Gene Therapy Market:

The fundamental details related to Gene Therapy industry like the product definition, product segmentation, price, a variety of statements, demand and supply statistics are covered in this article.

The comprehensive study of gene therapy market based on development opportunities, growth restraining factors and the probability of investment will anticipate the market growth.

The study of emerging Gene Therapy market segments and the existing market segments will help the readers in preparing the marketing strategies.

The study presents major market drivers that will augment the gene therapy market commercialization landscape.

The study performs a complete analysis of these propellers that will impact the profit matrix of this industry positively.

The study exhibits information about the pivotal challenges restraining market expansion

The market review for the global market is done in context to region, share, and size.

The important tactics of top players in the market.

Other points comprised in the Gene Therapy report are driving factors, limiting factors, new upcoming opportunities, encountered challenges, technological advancements, flourishing segments, and major trends of the market.

Check Table of Contents of This Report @https://market.us/report/gene-therapy-market//#toc

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Gene Therapy Market | on(impact of COVID-19) 2020-2029 Analysis on Growth, Future Demand 2020 - Jewish Life News

Complete study of the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market is carried out by the analysts in this report, taking into consideration key factors like drivers, challenges, recent trends, opportunities, advancements, and competitive landscape. This report offers a clear understanding of the present as well as future scenario of the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing industry. Research techniques like PESTLE and Porters Five Forces analysis have been deployed by the researchers. They have also provided accurate data on Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing production, capacity, price, cost, margin, and revenue to help the players gain a clear understanding into the overall existing and future market situation.

Key companies operating in the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market include , BioReliance, Cobra Biologics, Oxford BioMedica, UniQure, FinVector, MolMed, MassBiologics, Richter-Helm, FUJIFILM Diosynth Biotechnologies, Lonza, Aldevron, Eurogentec, Cell and Gene Therapy Catapult, Biovian, Brammer Bio, VGXI, PlasmidFactory, bluebird bio, Novasep, Spark Therapeutics, Vigene Biosciences Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing

Get PDF Sample Copy of the Report to understand the structure of the complete report: (Including Full TOC, List of Tables & Figures, Chart) :

https://www.qyresearch.com/sample-form/form/1688742/covid-19-impact-on-global-viral-vectors-non-viral-vectors-and-gene-therapy-manufacturing-market

Segmental Analysis

The report has classified the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing industry into segments including product type and application. Every segment is evaluated based on growth rate and share. Besides, the analysts have studied the potential regions that may prove rewarding for the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing manufcaturers in the coming years. The regional analysis includes reliable predictions on value and volume, thereby helping market players to gain deep insights into the overall Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing industry.

Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Segment By Type:

, AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA, Other Vectors Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing

Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Segment By Application:

, Cancers, Inherited Disorders, Viral Infections, Others

Competitive Landscape

It is important for every market participant to be familiar with the competitive scenario in the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing industry. In order to fulfil the requirements, the industry analysts have evaluated the strategic activities of the competitors to help the key players strengthen their foothold in the market and increase their competitiveness.

Key companies operating in the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market include , BioReliance, Cobra Biologics, Oxford BioMedica, UniQure, FinVector, MolMed, MassBiologics, Richter-Helm, FUJIFILM Diosynth Biotechnologies, Lonza, Aldevron, Eurogentec, Cell and Gene Therapy Catapult, Biovian, Brammer Bio, VGXI, PlasmidFactory, bluebird bio, Novasep, Spark Therapeutics, Vigene Biosciences Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing

Key questions answered in the report:

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TOC

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered: Ranking by Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Revenue1.4 Covid-19 Implications on Market by Type1.4.1 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size Growth Rate by Type: 2020 VS 20261.4.2 AAV1.4.3 Adenoviral1.4.4 Lentiviral1.4.5 Retroviral1.4.6 Plasmid DNA1.4.7 Other Vectors1.5 Covid-19 Implications on Market by Application1.5.1 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Share by Application: 2020 VS 20261.5.2 Cancers1.5.3 Inherited Disorders1.5.4 Viral Infections1.5.5 Others1.6 Coronavirus Disease 2019 (Covid-19): Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Industry Impact1.6.1 How the Covid-19 is Affecting the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Industry1.6.1.1 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business Impact Assessment Covid-191.6.1.2 Supply Chain Challenges1.6.1.3 COVID-19s Impact On Crude Oil and Refined Products1.6.2 Market Trends and Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Potential Opportunities in the COVID-19 Landscape1.6.3 Measures / Proposal against Covid-191.6.3.1 Government Measures to Combat Covid-19 Impact1.6.3.2 Proposal for Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Players to Combat Covid-19 Impact 1.7 Study Objectives 1.8 Years Considered2 Global Growth Trends by Regions2.1 Covid-19 Implications on Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Perspective (2015-2026)2.2 Covid-19 Implications on Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Growth Trends by Regions2.2.1 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Regions: 2015 VS 2020 VS 20262.2.2 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Historic Market Share by Regions (2015-2020)2.2.3 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Forecasted Market Size by Regions (2021-2026) 2.3 Industry Trends and Growth Strategy 2.3.1 Market Top Trends 2.3.2 Market Drivers2.3.3 Market Challenges2.3.4 Porters Five Forces Analysis2.3.5 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Growth Strategy2.3.6 Primary Interviews with Key Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Players (Opinion Leaders)3 Covid-19 Implications on Competition Landscape by Key Players3.1 Global Top Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Players by Market Size3.1.1 Global Top Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Players by Revenue (2015-2020)3.1.2 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Revenue Market Share by Players (2015-2020)3.1.3 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.2 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Concentration Ratio3.2.1 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Concentration Ratio (CR5 and HHI)3.2.2 Global Top 10 and Top 5 Companies by Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Revenue in 20193.3 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Key Players Head office and Area Served3.4 Key Players Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Product Solution and Service3.5 Date of Enter into Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market3.6 Mergers & Acquisitions, Expansion Plans4 Covid-19 Implications on Market Size by Type (2015-2026)4.1 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Historic Market Size by Type (2015-2020)4.2 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Forecasted Market Size by Type (2021-2026)5 Covid-19 Implications on Market Size by Application (2015-2026)5.1 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020)5.2 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Forecasted Market Size by Application (2021-2026)6 North America6.1 North America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size (2015-2020)6.2 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Key Players in North America (2019-2020)6.3 North America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Type (2015-2020)6.4 North America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020)7 Europe7.1 Europe Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size (2015-2020)7.2 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Key Players in Europe (2019-2020)7.3 Europe Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Type (2015-2020)7.4 Europe Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020)8 China8.1 China Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size (2015-2020)8.2 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Key Players in China (2019-2020)8.3 China Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Type (2015-2020)8.4 China Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020)9 Japan9.1 Japan Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size (2015-2020)9.2 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Key Players in Japan (2019-2020)9.3 Japan Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Type (2015-2020)9.4 Japan Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020)10Key Players Profiles10.1 BioReliance10.1.1 BioReliance Company Details10.1.2 BioReliance Business Overview and Its Total Revenue10.1.3 BioReliance Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.1.4 BioReliance Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020))10.1.5 BioReliance Recent Development10.2 Cobra Biologics10.2.1 Cobra Biologics Company Details10.2.2 Cobra Biologics Business Overview and Its Total Revenue10.2.3 Cobra Biologics Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.2.4 Cobra Biologics Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.2.5 Cobra Biologics Recent Development10.3 Oxford BioMedica10.3.1 Oxford BioMedica Company Details10.3.2 Oxford BioMedica Business Overview and Its Total Revenue10.3.3 Oxford BioMedica Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.3.4 Oxford BioMedica Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.3.5 Oxford BioMedica Recent Development10.4 UniQure10.4.1 UniQure Company Details10.4.2 UniQure Business Overview and Its Total Revenue10.4.3 UniQure Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.4.4 UniQure Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.4.5 UniQure Recent Development10.5 FinVector10.5.1 FinVector Company Details10.5.2 FinVector Business Overview and Its Total Revenue10.5.3 FinVector Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.5.4 FinVector Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.5.5 FinVector Recent Development10.6 MolMed10.6.1 MolMed Company Details10.6.2 MolMed Business Overview and Its Total Revenue10.6.3 MolMed Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.6.4 MolMed Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.6.5 MolMed Recent Development10.7 MassBiologics10.7.1 MassBiologics Company Details10.7.2 MassBiologics Business Overview and Its Total Revenue10.7.3 MassBiologics Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.7.4 MassBiologics Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.7.5 MassBiologics Recent Development10.8 Richter-Helm10.8.1 Richter-Helm Company Details10.8.2 Richter-Helm Business Overview and Its Total Revenue10.8.3 Richter-Helm Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.8.4 Richter-Helm Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.8.5 Richter-Helm Recent Development10.9 FUJIFILM Diosynth Biotechnologies10.9.1 FUJIFILM Diosynth Biotechnologies Company Details10.9.2 FUJIFILM Diosynth Biotechnologies Business Overview and Its Total Revenue10.9.3 FUJIFILM Diosynth Biotechnologies Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.9.4 FUJIFILM Diosynth Biotechnologies Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.9.5 FUJIFILM Diosynth Biotechnologies Recent Development10.10 Lonza10.10.1 Lonza Company Details10.10.2 Lonza Business Overview and Its Total Revenue10.10.3 Lonza Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.10.4 Lonza Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.10.5 Lonza Recent Development10.11 Aldevron10.11.1 Aldevron Company Details10.11.2 Aldevron Business Overview and Its Total Revenue10.11.3 Aldevron Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.11.4 Aldevron Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.11.5 Aldevron Recent Development10.12 Eurogentec10.12.1 Eurogentec Company Details10.12.2 Eurogentec Business Overview and Its Total Revenue10.12.3 Eurogentec Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.12.4 Eurogentec Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.12.5 Eurogentec Recent Development10.13 Cell and Gene Therapy Catapult10.13.1 Cell and Gene Therapy Catapult Company Details10.13.2 Cell and Gene Therapy Catapult Business Overview and Its Total Revenue10.13.3 Cell and Gene Therapy Catapult Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.13.4 Cell and Gene Therapy Catapult Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.13.5 Cell and Gene Therapy Catapult Recent Development10.14 Biovian10.14.1 Biovian Company Details10.14.2 Biovian Business Overview and Its Total Revenue10.14.3 Biovian Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.14.4 Biovian Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.14.5 Biovian Recent Development10.15 Brammer Bio10.15.1 Brammer Bio Company Details10.15.2 Brammer Bio Business Overview and Its Total Revenue10.15.3 Brammer Bio Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.15.4 Brammer Bio Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.15.5 Brammer Bio Recent Development10.16 VGXI10.16.1 VGXI Company Details10.16.2 VGXI Business Overview and Its Total Revenue10.16.3 VGXI Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.16.4 VGXI Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.16.5 VGXI Recent Development10.17 PlasmidFactory10.17.1 PlasmidFactory Company Details10.17.2 PlasmidFactory Business Overview and Its Total Revenue10.17.3 PlasmidFactory Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.17.4 PlasmidFactory Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.17.5 PlasmidFactory Recent Development10.18 bluebird bio10.18.1 bluebird bio Company Details10.18.2 bluebird bio Business Overview and Its Total Revenue10.18.3 bluebird bio Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.18.4 bluebird bio Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.18.5 bluebird bio Recent Development10.19 Novasep10.19.1 Novasep Company Details10.19.2 Novasep Business Overview and Its Total Revenue10.19.3 Novasep Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.19.4 Novasep Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.19.5 Novasep Recent Development10.20 Spark Therapeutics10.20.1 Spark Therapeutics Company Details10.20.2 Spark Therapeutics Business Overview and Its Total Revenue10.20.3 Spark Therapeutics Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.20.4 Spark Therapeutics Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.20.5 Spark Therapeutics Recent Development10.21 Vigene Biosciences10.21.1 Vigene Biosciences Company Details10.21.2 Vigene Biosciences Business Overview and Its Total Revenue10.21.3 Vigene Biosciences Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Introduction10.21.4 Vigene Biosciences Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business (2015-2020)10.21.5 Vigene Biosciences Recent Development11Analysts Viewpoints/Conclusions12Appendix12.1 Research Methodology12.1.1 Methodology/Research Approach12.1.2 Data Source12.2 Disclaimer12.3 Author Details

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NOVATO Apr 30, 2020 (Thomson StreetEvents) -- Edited Transcript of Biomarin Pharmaceutical Inc earnings conference call or presentation Wednesday, April 29, 2020 at 8:15:00pm GMT

* Brian R. Mueller

BioMarin Pharmaceutical Inc. - Senior VP of Finance, Acting CFO & CAO

* Henry J. Fuchs

BioMarin Pharmaceutical Inc. - President of Worldwide Research & Development

BioMarin Pharmaceutical Inc. - Chairman & CEO

BioMarin Pharmaceutical Inc. - Executive VP & Chief Commercial Officer

* Robert A. Baffi

BioMarin Pharmaceutical Inc. - President of Global Manufacturing & Technical Operations

BioMarin Pharmaceutical Inc. - VP of IR

Evercore ISI Institutional Equities, Research Division - Senior MD & Equity Analyst

* Kennen B. MacKay

RBC Capital Markets, Research Division - MD & Co-Head of US Biotechnology Research

* Peter B. Kim

* Philip M. Nadeau

William Blair & Company L.L.C., Research Division - Co-Group Head of Biopharma Equity Research

Sanford C. Bernstein & Co., LLC., Research Division - VP

Welcome to the BioMarin First Quarter 2020 Financial Results Conference Call. Hosting the conference call today from BioMarin is Traci McCarty, Vice President of Investor Relations. Please go ahead, Traci.

Traci McCarty, BioMarin Pharmaceutical Inc. - VP of IR [2]

Thank you, May, and thank you, everyone, for joining us today. To remind you, this nonconfidential presentation contains forward-looking statements about the business prospects of BioMarin, including expectations regarding BioMarin's financial performance, commercial products and potential future products in different areas of therapeutic research and development.

Results may differ materially depending on the progress of BioMarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market and developments by competitors, and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, such as 10-Q, 10-K and 8-K reports.

On the call remotely from BioMarin management today are J.J. Bienaim, Chairman and Chief Executive Officer; Jeff Ajer, Executive Vice President, Chief Commercial Officer; Robert Baffi, President, Global Manufacturing and Technical Operations; Hank Fuchs, President, Worldwide Research and Development; and Brian Mueller, acting Chief Financial Officer. We hope to keep this call to 1 hour and also give everyone the opportunity to ask a question today, so we request that you limit yourself to one during the Q&A portion of the call. Thank you for your understanding. I will now turn the call over to our Chairman and CEO, J.J. Bienaim.

--------------------------------------------------------------------------------

Jean-Jacques Bienaim, BioMarin Pharmaceutical Inc. - Chairman & CEO [3]

--------------------------------------------------------------------------------

Thank you, Traci. Good afternoon, and thank you for joining us on today's call. We hope you and your families are healthy and managing through these unusual circumstances brought about by the COVID-19 virus. So these are unprecedented times, but the essential nature of our medicines to the patients who need them has enabled BioMarin to weather the challenge of COVID-19 quite well. Equally as important, I want to underscore the extraordinary dedication of our employees who have kept operations running smoothly in order to maintain access to our therapies around the world.

Our first quarter record results of $502 million of total revenues or 25% growth over last year, the testament to the importance of our therapies and our diversified product base and commercial footprint. Due in part to the sale of Firdapse, GAAP net income in the first quarter was $81.4 million, exceeding our current full year guidance range of $20 million to $80 million.

In the first quarter, we experienced minimal interruptions due to COVID-19, but we do anticipate the potential for more meaningful business disruptions for the remainder of 2020 due to the pandemic. As a result, we have chosen to reduce our full year total revenue guidance by around 5% or a total of $100 million, while maintaining both GAAP and non-GAAP income fully estimated provided earlier this year. Despite potential near-term impacts to our commercial business on COVID-19, our next blockbusters, BMN 270, valoctocogene roxaparvovec, (inaudible) valrox for hemophilia A and vosoritide for achondroplasia continuing to advance, and Jeff will reveal our recently approved brand name for BMN 270 in a moment.

Briefly on vosoritide for achondroplasia. In the quarter, we announced that based on recent meetings, a successful meeting with health authorities in the U.S. and Europe, we plan to submit marketing applications to the FDA and EMA in the third quarter of this year. If approved, vosoritide will be the first medicine for the treatment of achondroplasia in the U.S. and Europe. So we are delighted that this potential therapy proceeds a step closer to regulatory.

In conclusion, BioMarin employees have risen to the evolving challenges of the COVID-19 pandemic, demonstrating a high level of commitment and dedication to the patients we serve. The underlying fundamentals of our business remains strong and our manufacturing and supply chain resilience. We have built a durable base business with essential medicines transition the pipeline to address larger rare indications, diversified risk and positions ourselves for substantial success in both the near term and the long term. We are confident in our ability to manage through this ongoing global health crisis, while staying grounded in our long-term strategy for success.

I'd like to say a few words about Robert Baffi, who has made tremendous contributions to the organization over the last 20 years. During this time at BioMarin, he has manufactured the most complex biological products in the world and visits the most advanced commercial scale gene therapy manufacturing capability. His leadership, technical expertise, foresight and dedication has played a key role in where we stand today, and we want to acknowledge his many contributions. Thank you, Robert. And we are pleased that he will remain with BioMarin through the review of BMN 270 valrox and the vosoritide marketing applications to ensure manufacturing continuity as the successor, Greg Guyer, begins his journey with us in May, coming from Bristol-Myers Squibb.

Thank you all for your continued support. And now I would like to turn the call over to Robert to say a few words. Robert?

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Robert A. Baffi, BioMarin Pharmaceutical Inc. - President of Global Manufacturing & Technical Operations [4]

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Thank you, J.J. Innovation has always been at the core of BioMarin's success. During my 20 years, as the Head of Technical Operations, and has instill in the company a few guiding principles to foster a culture of innovation: first, let science inform and lead decision-making; second, let compliance focus our efforts on patient safety and clinical outcomes; and third, let ingenuity create adaptivity and resiliency in our approach to drug development. These 3 tenants, infused with the talents of the most dedicated people I've ever worked with, have consistently enabled us to take research ideas rapidly through development, navigating the complexity of the regulatory approval process in a highly effective and differentiated manner to meet the needs of patients.

BioMarin's leadership team has shared and supported the vision for creating a fully integrated company with technical operations, powers clinical studies and commercial demand, and is an integral component of strategic technology development paradigms for assuring the timely delivery of an uninterrupted supply of product.

Furthermore, innovative and appropriately implemented CMC strategies linked to a faster clinical design allows for rapid development and high success rates that benefit both patients and shareholders alike. As a company, we are going through multiple transitions simultaneously, challenging in some ways, invigorating in others. Our transition to profitability this year provides the resources to develop more innovative therapies. Our transition to gene therapy product leverages our clinical manufacturing and commercial capabilities and place us squarely at the forefront of the emerging technological advancement in precision medicine. Our transition and technical operation leadership provides the opportunity to build an innovative approach to drug development to fuel our growth.

When I first saw that to Dr. Greg Guyer's CV, I could not help but be impressed with the scope of his responsibility and the experiential variety and diversity of his career. In many ways, while a different journey, it shared a lot of commonality with my own and that is not to let us vote to BioMarin. I am confident and committed that the transition in technical operations at BioMarin from me to Greg will build on the legacy of science, compliance and ingenuity, for our patients that will benefit from the products that will emerge from our efforts, for our employees and their careers, and for our shareholders as we become profitable.

In terms of licensure of our gene therapy manufacturing facility in support of BMN 270 approval, I'm pleased to share that the Health Products Regulatory Authority of Ireland conducted, on behalf of the European Medicines Agency, a pre-approval inspection in Q1. This inspection involves a detailed review of the facility, equipment, process, and analytical studies and relevant documentations generated in support of validation, production and testing. Following this inspection, a CGMP certification was granted, allowing for commercial production and distribution of BMN 270 in the EU when the product is approved.

At present, the inspection of the facility by FDA is expected to be completed during Q2, allowing full licensure in the U.S. of the facility consistent with the August 21 PDUFA action date. We have more than 400 doses of commercial BMN 270 ready for potential launch later this year, and remain very enthusiastic about the prospects for introducing the first gene therapy product for a bleeding disorder to the hemophilia community as soon as possible.

I thank you for your support throughout my time at BioMarin. And now I'd like to turn the call over to Jeff to discuss the commercial business update. Jeff?

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Jeffrey Robert Ajer, BioMarin Pharmaceutical Inc. - Executive VP & Chief Commercial Officer [5]

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Thank you, Robert. As we begin 2020, I'm very pleased with the team's performance across all brands and all regions during the quarter. As J.J. mentioned, we achieved our highest quarterly revenue on record, with total revenues of $502 million in the first quarter with net product revenues marketed by BioMarin, up 24% to $433 million. This achievement reflects the fundamental strength and growth of our business despite near-term challenges related to COVID-19, which I will address in a moment.

On to results in the quarter and starting with Palynziq. In the U.S., the trend of increasing revenue based on a steadily growing base of patients on commercial therapy, including progression from induction and titration to daily maintenance dosing, continued in Q1. In the early part of the quarter, we did experience a seasonal slowing of new patient enrollments and patient starts, somewhat mirroring our historical experience with Kuvan in the United States. We are reporting $35 million in Palynziq revenue for the first quarter, with the majority of that revenue coming from the U.S.

In Europe, in the first quarter, multiple clinics across Germany continued to actively treat patients with Palynziq and early uptake signals are encouraging. During the quarter, we made significant progress in Germany, adding clinics that now have some experience prescribing Palynziq and managing patients through the induction and titration phase to daily maintenance dosing. As the number of commercial patients in Germany steadily grows, we anticipate meaningful revenue contribution from the EU starting this year. We anticipate finalizing price and reimbursement negotiations in Germany by mid this year, an important step toward getting price and reimbursement approvals in other high priority European markets.

Kuvan contributed $122 million in revenues in the quarter or 14% growth year-over-year, with most of that growth coming from the United States. Vimizim revenues grew 9% year-over-year, contributing $137 million in the first quarter, driven by an 11% increase in patients year-over-year. This is reflective of the continued anticipated growth potential we expect for Vimizim.

Turning to Naglazyme. Revenues totaled $114 million, a 32% year-over-year growth for the well-established brand. As with Vimizim, the impact from uneven large order patterns makes the quarterly comparison difficult. The number of commercial patients on Naglazyme grew by 6% in the past year, and is indicative of the ongoing growth potential for this brand, nearly 15 years since being approved.

And finally, Brineura contributed $24 million in net product revenues, which represented 97% year-over-year growth. These revenues were essentially flat over Q4, and that was driven by a modest year-end inventory build in the EMEA region in Q4. Importantly, the growth in Brineura revenues compared to prior year reflects an underlying growth of 86% in commercial patients. We are seeing a net increase in patients benefiting from Brineura treatment due to the success of our disease awareness and patient identification programs.

Taken together, we are pleased with first quarter results and demand for our products. And while we experienced minimal financial impact in the first quarter due to COVID-19, we anticipate the potential for a higher degree of impact during the remainder of 2020 as disruptions of day-to-day operations of clinics and hospitals flow through our business.

Our global commercial teams will continue to adjust to implement innovative approaches to engage with clinics and patients to ensure continuity of access to our medicines. Where possible, we are supporting home infusion efforts to help mitigate impact. However, some COVID-19 disruption, the new patient starts as well as to ongoing infusion center visits from existing patients are expected to continue. As a result, we are reducing total revenue guidance by 5% at the midpoint to between $1,850 million to $1,950 million for the full year 2020. The vast majority of today's updated total revenue guidance reflects adjustments to in line brands, including Vimizim, Naglazyme and Palynziq. And assumes our business will return to normalized demand patterns in the second half of 2020. Although we did not give BMN 270 2020 revenue guidance in February, our 2020 total revenue guidance did assume some contribution from BMN 270 in Europe.

And now I'd like to end my remarks with an update on our hemophilia gene therapy program and introduce you to the intended brand name, ROCTAVIAN. In previous calls, you've heard references to valrox, which was an abbreviated form of our INN or international nonproprietary name, valoctocogene roxaparvovec or alternatively, our program identifier, BMN 270. Both the FDA and the EMA have accepted ROCTAVIAN as our brand name, and we look forward to adopting ROCTAVIAN as we get closer to launch. In the meantime, we will cease to use valrox, so as to not confuse it with our intended brand name.

Other key launch readiness activities have continued to progress. We have essentially built out the commercial team in the United States, and have added key individuals to support priority in markets in the EU. The majority of these new employees have substantial and diverse experience in hemophilia. Our brand campaigns also continue to develop as anticipated and teams have pivoted to virtual and digital platforms, allowing for ongoing engagement with the marketplace in lieu of face-to-face interactions. Obviously, certain activities are more amenable to virtual engagements than others, and where COVID-19 is challenging us most in the short-term is with gene therapy educational programs and site readiness.

Fortunately, with the team already on board, we anticipate being well prepared to launch if we receive regulatory approvals. In the meantime, we have recently finished some very positive pricing research, which validated payer willingness to embrace ROCTAVIAN with the current data set, and we'll look forward to providing you with updates on our pricing at launch.

Thank you for your attention, and I will now turn the call over to Hank to provide an R&D update. Hank?

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Henry J. Fuchs, BioMarin Pharmaceutical Inc. - President of Worldwide Research & Development [6]

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Thanks, Jeff. I'd also like to echo J.J.'s expression of deep and heartfelt appreciation for the time Robert has spent with us. And also to welcome Greg Guyer, to the organization where the secret sauce is bottled.

The R&D organization is delighted that our next significant product opportunities continue to progress, particularly under the circumstances brought about by COVID-19. The ability to come to work mostly virtually and focus on the advancement of our innovative products to date, ROCTAVIAN and vosoritide has been especially gratifying and a welcome distraction from the ongoing pandemic. I want to acknowledge and thank our teams for their commitment and contributions during these challenging times. I have been impressed by your flexibility and your ability to keep the story going while we are dealing with the pandemic.

Starting with ROCTAVIAN, and with a strong and memorable brand name, congratulations, Jeff, the FDA is committed to meet the August 21 PDUFA action date. In Europe, our marketing authorization application filing remains on accelerated assessment at this time. However, the review procedure is to be extended by at least 3 months due to COVID-19 delays. Further, as is the case with most filings that initially receive accelerated assessment, we believe there is a high possibility that our M&A will revert to a standard review procedure from accelerated assessment. Based on these assumptions, we expect the CHMP opinion by late '20 or early '21.

We continue to plan to share our 3 -- our 4-year update of the 16, 13 vector genome per kilo dose as well as the 3-year update on the 40, 13 vector genome per kilo dose in the middle of the year, but the form is as yet to be defined given the changing environment for medical meetings. We have a data analysis plan in place. We'll move forward with business as usual, but the venue and method for providing the update is still fluid at this point. So thanks for bearing with us.

Importantly, we do not expect COVID-19 to impact the time lines for completion of the ROCTAVIAN Phase III trial. Enrollment was completed in November of last year, and one of the benefits of being a onetime treatment is that patients do not need to receive therapy on a chronic basis. We're also confident that the integrity of the ongoing data collection for the study -- for this pivotal study is being sufficiently maintained as home health care solutions align nicely with the collection of the primary endpoint annualized bleed rate data.

Turning now to vosoritide for the treatment of achondroplasia. As J.J. mentioned, we plan to submit a global marketing applications in the third quarter of this year. Our multipronged development program, including a long-term Phase II clinical results in 5 to 18-year-old children, comprehensive natural history data, the ongoing study of newborns through 5 years and the highly statistically significant placebo-controlled Phase III trial makes for a very comprehensive data package spanning more than 5 years of treatment with children with achondroplasia. Again, we're the beneficiary of fortunate timing and that our pivotal submission data read out prior to the pandemic, and now much of the work can be concluded remotely. If approved, vosoritide would be the first and only medicine designated for the treatment of achondroplasia in the U.S. and in European Union.

We continue to look forward to publishing the full data from the Phase III study later this year, and we're pleased to let you know that our late-breaker has been accepted in an upcoming medical Congress. The presentation will include 1 year growth velocity, height Z scores, body proportionality, safety and subgroup analyses. So stay tuned for more specifics as to when and where those data will appear.

The Phase II study of vosoritide in 0 to 5 year olds, referred to as study 206, is proceeding well, and we are very pleased that safety data from children ages 6 months to 5 years participating in that study will be available as part of our registration package. We're grateful that the timing of key studies has aligned well with our pre-COVID-19 plans.

Moving to BMN 307, our investigational gene therapy for phenylketonuria. We're continuing to prepare new sites to open in order to enroll patients when it is safely do so given the COVID-19 circumstances. We're excited about the prospect of BMN 307 as it represents a third treatment for phenylketonuria in our PKU franchise and a second gene therapy development program, leveraging our learnings and capabilities from ROCTAVIAN. Currently, we expect the study to start later -- we expect to start the study later in 2020.

The R&D organization is energized by the opportunities before us in 2020, with both ROCTAVIAN for severe hemophilia A and vosoritide for children with achondroplasia advancing towards potential approvals. We are hopeful that these innovative treatments will be available in the very near future. We look forward to updating you on our progress over the coming quarters, and thank you for your continued support.

And I'll now turn the call over to Brian to review the financials.

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Brian R. Mueller, BioMarin Pharmaceutical Inc. - Senior VP of Finance, Acting CFO & CAO [7]

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Thank you, Hank. Please refer to today's press release summarizing our financial results for full details on the first quarter of 2020, and as usual, our comprehensive report on the quarter will be available in our upcoming Form 10-Q, which we are on track to file over the next couple of days.

As Jeff mentioned, we are experiencing some modest impacts from the COVID-19 pandemic and as a result, we have updated full year total revenue guidance to between $1.85 billion to $1.95 billion. As Jeff noted,our updated revenue guidance is based on the assumption that our business will return to normalized demand patterns in the second half of the year.

Importantly, while we lowered our revenue guidance due to the impact of COVID-19 on our commercial business, we were able to analyze our 2020 spending projections and make adjustment that allowed us to maintain our prior GAAP and non-GAAP income guidance despite the lower revenue.

Moving to operating expenses. R&D expense for the first quarter of 2020 was $142 million and lower compared to R&D expense for the first quarter of 2019 of $184 million, mostly due to less R&D activity for ROCTAVIAN, given its late stage of development as well as Palynziq following its approvals in the U.S. and Europe.

SG&A expense for the first quarter of '20 was $187 million, which was higher than SG&A expense for the first quarter of 2019 of $162 million. The year-over-year increase was expected with the single largest driver, being the commercial preparation for the launch of ROCTAVIAN and the continued global launch of Palynziq. We also incurred some unpredicted foreign currency exchange losses during the month of March as the COVID pandemic negatively affected some of our assets denominated and some of the more volatile global currencies.

During the bottom line results, we reported GAAP net income of $81 million in the first quarter of 2020, compared to a GAAP net loss of $56.5 million in the first quarter of 2019. The improvement in GAAP income was primarily due to higher revenue, lower R&D expenses and the gain on the sale of the deferred tax assets. With higher revenues and lower R&D expenses, non-GAAP income of $117 million in the first quarter of 2020 grew substantially as compared to Q1 2019 non-GAAP income of $25 million. Both of these first quarter 2020 bottom line results gives us a great start towards achieving our 2020 goals of GAAP net income on an annual basis for the first time in the company's history, a considerable growth in non-GAAP income.

I'd also like to touch on the potential tax benefit that we mentioned last quarter that may be recognized in the second half of this year. Our current 2020 GAAP net income guidance of between $20 million to $80 million, excludes the potential impact of intra-entity intangible asset transfers between BioMarin entities. If these intangible asset transfers occur, we estimate that the tax effect could result in a onetime noncash income tax benefit of greater than $500 million. As I mentioned previously, you may have seen similar transactions completed by some of our larger peers in recent quarters.

Speaking to total cash and investments, we ended the first quarter of 2020 with $1.15 billion compared to $1.17 billion at the end of December 2019. The modest decrease in total cash and investments during Q1 2020 was largely due to some timing of operating cash flows. However, the significant improvement over the first quarter of 2019 where total cash and investments decreased by $105 million. This valid cash position, coupled with vibrant business fundamentals, put us in good standing to manage through the continued uncertainty related to COVID-19.

In closing, the stronger performance of the business during the first quarter of 2020, plus our positive financial outlook for the rest of the year, indicate that 2020 should be a transformational year for the company. And the prospects of value to come from ROCTAVIAN and vosoritide, if they are approved commercially, give us an enthusiasm about our future.

Thank you for your support, and we will now open the call to your questions. Operator?

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question is from the line of Robyn Karnauskas from Suntrust.

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Robyn Kay Shelton Karnauskas, SunTrust Robinson Humphrey, Inc., Research Division - Research Analyst [2]

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And just first off, congratulations to Greg. But to Rob, it was lovely working with you. I think you brought a breath of fresh air to working with management teams in all of my buyers (inaudible). So thank you so much. I learned a lot. I guess I'm going to start-up with some questions on ROCTAVIAN, and I hope I get that correctly. So first of all, what gives you confidence that in the United States, that there won't be any more delays? People ask me this nonstop. And then when you talk about assuming normal operations go -- resume in the second half, is that in the beginning of the second half? Do you have a time line for that? If it goes into fourth quarter, could we see further delays?

And the third question is, what are you hearing as far as like people willing to have gene therapy procedures done in the COVID environment as early as fourth quarter? Are people open to it? Is it separate from the hospitals? Or what are you hearing from the ground?

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Jean-Jacques Bienaim, BioMarin Pharmaceutical Inc. - Chairman & CEO [3]

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Okay. Hi. (inaudible) do you want to answer the question on the no delays with the FDA? Hank?

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More:
Edited Transcript of BMRN earnings conference call or presentation 29-Apr-20 8:15pm GMT - Yahoo Finance

Gene Therapy Market report covers the present and past market scenarios, market development patterns, and is likely to proceed with a continuing development over the forecast period. The research report on Market provides comprehensive analysis on market status and development trend, including types, applications, rising technology and region. A number of analysis tools such as SWOT analysis and Porters five forces analysis have been employed to provide an accurate understanding of this market.

Some of the key players of Market:Adaptimmune Therapeutics Plc., Anchiano Therapeutics Ltd., Achieve Life Sciences, Inc., Adverum Biotechnologies, Inc., Abeona Therapeutics Inc., Applied Genetic Technologies Corporation, Arbutus Biopharma Corporation,, Audentes Therapeutics, Inc., AveXis, Inc., Bluebird Bio, Inc., Celgene Corporation and more

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Segmentation by product type:Antigen, Cytokine, Tumor Suppressor, Suicide, Deficiency, Growth Factors, Receptors, and Others

Segmentation by application:Oncological Disorders, Rare Diseases, Cardiovascular Diseases, Neurological Disorders, Infectious Disease, and Other Diseases

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The report provides a detailed overview of the industry including both qualitative and quantitative information. It provides overview and forecast of the global Gene Therapy market based on product and application. It also provides market size and forecast till 2023 for overall Gene Therapy market with respect to five major regions, namely; North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South America (SAM), which is later sub-segmented by respective countries and segments.

Table of content

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Gene Therapy Market: how to drive decisions and growth in a business by top key players like Audentes Therapeutics, Inc., AveXis, Inc., Bluebird...

Scientists from the University of Sheffield have produced the first high-resolution images of the structure of the cell wall of bacteria, in a study that could further understanding of antimicrobial resistance.The research, published in Nature, revealed a new and unexpected structure of the outer bacterial layers of the bacterium Staphylococcus aureus.

The findings set a new framework for understanding how bacteria grow and how antibiotics work, overturning previous theories about the structure of the outer bacterial layers.

The images give unprecedented insight into the composition of the bacterial cell wall and will inform new approaches to developing antibiotics in order to combat antibiotic resistance. There are no other examples of studies of the cell wall in any organism at comparable resolution, down to the molecular scale.

Laia Pasquina Lemonche, a PhD Researcher from the University of Sheffields Department of Physics and Astronomy, said: Many antibiotics work by inhibiting the bacterias production of a cell wall, a strong but permeable skin around the bacteria which is critical for its survival.

We still dont understand how antibiotics like penicillin kill bacteria, but this isnt surprising because until now we had remarkably little information about the actual organisation of the bacterial cell wall. This study provides that essential stepping stone which we hope will lead to both a better understanding of how antibiotics work and to the future development of new approaches to combat antimicrobial resistance.

The team used an advanced microscopy technique called Atomic Force Microscopy (AFM), which works by using a sharp needle to feel the shape of a surface and build an image similar to a contour map, but at the scale of individual molecules.

Professor Jamie Hobbs, Professor of Physics at the University of Sheffield, said: It is by physicists and biologists working together that we've been able to make these breakthroughs in our understanding of the bacterial cell wall.

The researchers are now using the same techniques to understand how antibiotics change the architecture of the cell wall and also how changes in the cell wall are important in antimicrobial resistance.ReferencePasquina-Lemonche et al. (2020). The architecture of the Gram-positive bacterial cell wall. Nature. DOI: https://doi.org/10.1038/s41586-020-2236-6

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

Original post:
First High-resolution Images of Bacterial Cell Wall - Technology Networks

Complete study of the global Gene Therapies for Cancer Treatment market is carried out by the analysts in this report, taking into consideration key factors like drivers, challenges, recent trends, opportunities, advancements, and competitive landscape. This report offers a clear understanding of the present as well as future scenario of the global Gene Therapies for Cancer Treatment industry. Research techniques like PESTLE and Porters Five Forces analysis have been deployed by the researchers. They have also provided accurate data on Gene Therapies for Cancer Treatment production, capacity, price, cost, margin, and revenue to help the players gain a clear understanding into the overall existing and future market situation.

Key companies operating in the global Gene Therapies for Cancer Treatment market include , Takara Bio, Tocagen, VBL Therapeutics, Cold Genesys, Genprex, Momotaro-Gene, MultiVir, SynerGene Therapeutics, Ziopharm Oncology, Anchiano Therapeutics, Celgene, Celsion, Bluebird Bio Gene Therapies for Cancer Treatment

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Segmental Analysis

The report has classified the global Gene Therapies for Cancer Treatment industry into segments including product type and application. Every segment is evaluated based on growth rate and share. Besides, the analysts have studied the potential regions that may prove rewarding for the Gene Therapies for Cancer Treatment manufcaturers in the coming years. The regional analysis includes reliable predictions on value and volume, thereby helping market players to gain deep insights into the overall Gene Therapies for Cancer Treatment industry.

Global Gene Therapies for Cancer Treatment Market Segment By Type:

, Somatic Cell Gene Therapy (SCGT), Germline Gene Therapy (GGT) Gene Therapies for Cancer Treatment

Global Gene Therapies for Cancer Treatment Market Segment By Application:

, Cancer Research Centers, Diagnostic Laboratories, Cancer Hospitals, Others

Competitive Landscape

It is important for every market participant to be familiar with the competitive scenario in the global Gene Therapies for Cancer Treatment industry. In order to fulfil the requirements, the industry analysts have evaluated the strategic activities of the competitors to help the key players strengthen their foothold in the market and increase their competitiveness.

Key companies operating in the global Gene Therapies for Cancer Treatment market include , Takara Bio, Tocagen, VBL Therapeutics, Cold Genesys, Genprex, Momotaro-Gene, MultiVir, SynerGene Therapeutics, Ziopharm Oncology, Anchiano Therapeutics, Celgene, Celsion, Bluebird Bio Gene Therapies for Cancer Treatment

Key questions answered in the report:

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TOC

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered: Ranking by Gene Therapies for Cancer Treatment Revenue1.4 Market Analysis by Type1.4.1 Global Gene Therapies for Cancer Treatment Market Size Growth Rate by Type: 2020 VS 20261.4.2 Somatic Cell Gene Therapy (SCGT)1.4.3 Germline Gene Therapy (GGT)1.5 Market by Application1.5.1 Global Gene Therapies for Cancer Treatment Market Share by Application: 2020 VS 20261.5.2 Cancer Research Centers1.5.3 Diagnostic Laboratories1.5.4 Cancer Hospitals1.5.5 Others1.6 Coronavirus Disease 2019 (Covid-19): Gene Therapies for Cancer Treatment Industry Impact1.6.1 How the Covid-19 is Affecting the Gene Therapies for Cancer Treatment Industry1.6.1.1 Gene Therapies for Cancer Treatment Business Impact Assessment Covid-191.6.1.2 Supply Chain Challenges1.6.1.3 COVID-19s Impact On Crude Oil and Refined Products1.6.2 Market Trends and Gene Therapies for Cancer Treatment Potential Opportunities in the COVID-19 Landscape1.6.3 Measures / Proposal against Covid-191.6.3.1 Government Measures to Combat Covid-19 Impact1.6.3.2 Proposal for Gene Therapies for Cancer Treatment Players to Combat Covid-19 Impact1.7 Study Objectives1.8 Years Considered2 Global Growth Trends by Regions2.1 Gene Therapies for Cancer Treatment Market Perspective (2015-2026)2.2 Gene Therapies for Cancer Treatment Growth Trends by Regions2.2.1 Gene Therapies for Cancer Treatment Market Size by Regions: 2015 VS 2020 VS 20262.2.2 Gene Therapies for Cancer Treatment Historic Market Share by Regions (2015-2020)2.2.3 Gene Therapies for Cancer Treatment Forecasted Market Size by Regions (2021-2026)2.3 Industry Trends and Growth Strategy2.3.1 Market Top Trends2.3.2 Market Drivers2.3.3 Market Challenges2.3.4 Porters Five Forces Analysis2.3.5 Gene Therapies for Cancer Treatment Market Growth Strategy2.3.6 Primary Interviews with Key Gene Therapies for Cancer Treatment Players (Opinion Leaders)3 Competition Landscape by Key Players3.1 Global Top Gene Therapies for Cancer Treatment Players by Market Size3.1.1 Global Top Gene Therapies for Cancer Treatment Players by Revenue (2015-2020)3.1.2 Global Gene Therapies for Cancer Treatment Revenue Market Share by Players (2015-2020)3.1.3 Global Gene Therapies for Cancer Treatment Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.2 Global Gene Therapies for Cancer Treatment Market Concentration Ratio3.2.1 Global Gene Therapies for Cancer Treatment Market Concentration Ratio (CR5 and HHI)3.2.2 Global Top 10 and Top 5 Companies by Gene Therapies for Cancer Treatment Revenue in 20193.3 Gene Therapies for Cancer Treatment Key Players Head office and Area Served3.4 Key Players Gene Therapies for Cancer Treatment Product Solution and Service3.5 Date of Enter into Gene Therapies for Cancer Treatment Market3.6 Mergers & Acquisitions, Expansion Plans4 Breakdown Data by Type (2015-2026)4.1 Global Gene Therapies for Cancer Treatment Historic Market Size by Type (2015-2020)4.2 Global Gene Therapies for Cancer Treatment Forecasted Market Size by Type (2021-2026)5 Gene Therapies for Cancer Treatment Breakdown Data by Application (2015-2026)5.1 Global Gene Therapies for Cancer Treatment Market Size by Application (2015-2020)5.2 Global Gene Therapies for Cancer Treatment Forecasted Market Size by Application (2021-2026)6 North America6.1 North America Gene Therapies for Cancer Treatment Market Size (2015-2020)6.2 Gene Therapies for Cancer Treatment Key Players in North America (2019-2020)6.3 North America Gene Therapies for Cancer Treatment Market Size by Type (2015-2020)6.4 North America Gene Therapies for Cancer Treatment Market Size by Application (2015-2020)7 Europe7.1 Europe Gene Therapies for Cancer Treatment Market Size (2015-2020)7.2 Gene Therapies for Cancer Treatment Key Players in Europe (2019-2020)7.3 Europe Gene Therapies for Cancer Treatment Market Size by Type (2015-2020)7.4 Europe Gene Therapies for Cancer Treatment Market Size by Application (2015-2020)8 China8.1 China Gene Therapies for Cancer Treatment Market Size (2015-2020)8.2 Gene Therapies for Cancer Treatment Key Players in China (2019-2020)8.3 China Gene Therapies for Cancer Treatment Market Size by Type (2015-2020)8.4 China Gene Therapies for Cancer Treatment Market Size by Application (2015-2020)9 Japan9.1 Japan Gene Therapies for Cancer Treatment Market Size (2015-2020)9.2 Gene Therapies for Cancer Treatment Key Players in Japan (2019-2020)9.3 Japan Gene Therapies for Cancer Treatment Market Size by Type (2015-2020)9.4 Japan Gene Therapies for Cancer Treatment Market Size by Application (2015-2020)10 Southeast Asia10.1 Southeast Asia Gene Therapies for Cancer Treatment Market Size (2015-2020)10.2 Gene Therapies for Cancer Treatment Key Players in Southeast Asia (2019-2020)10.3 Southeast Asia Gene Therapies for Cancer Treatment Market Size by Type (2015-2020)10.4 Southeast Asia Gene Therapies for Cancer Treatment Market Size by Application (2015-2020)11 India11.1 India Gene Therapies for Cancer Treatment Market Size (2015-2020)11.2 Gene Therapies for Cancer Treatment Key Players in India (2019-2020)11.3 India Gene Therapies for Cancer Treatment Market Size by Type (2015-2020)11.4 India Gene Therapies for Cancer Treatment Market Size by Application (2015-2020)12 Central & South America12.1 Central & South America Gene Therapies for Cancer Treatment Market Size (2015-2020)12.2 Gene Therapies for Cancer Treatment Key Players in Central & South America (2019-2020)12.3 Central & South America Gene Therapies for Cancer Treatment Market Size by Type (2015-2020)12.4 Central & South America Gene Therapies for Cancer Treatment Market Size by Application (2015-2020)13 Key Players Profiles13.1 Takara Bio13.1.1 Takara Bio Company Details13.1.2 Takara Bio Business Overview and Its Total Revenue13.1.3 Takara Bio Gene Therapies for Cancer Treatment Introduction13.1.4 Takara Bio Revenue in Gene Therapies for Cancer Treatment Business (2015-2020))13.1.5 Takara Bio Recent Development13.2 Tocagen13.2.1 Tocagen Company Details13.2.2 Tocagen Business Overview and Its Total Revenue13.2.3 Tocagen Gene Therapies for Cancer Treatment Introduction13.2.4 Tocagen Revenue in Gene Therapies for Cancer Treatment Business (2015-2020)13.2.5 Tocagen Recent Development13.3 VBL Therapeutics13.3.1 VBL Therapeutics Company Details13.3.2 VBL Therapeutics Business Overview and Its Total Revenue13.3.3 VBL Therapeutics Gene Therapies for Cancer Treatment Introduction13.3.4 VBL Therapeutics Revenue in Gene Therapies for Cancer Treatment Business (2015-2020)13.3.5 VBL Therapeutics Recent Development13.4 Cold Genesys13.4.1 Cold Genesys Company Details13.4.2 Cold Genesys Business Overview and Its Total Revenue13.4.3 Cold Genesys Gene Therapies for Cancer Treatment Introduction13.4.4 Cold Genesys Revenue in Gene Therapies for Cancer Treatment Business (2015-2020)13.4.5 Cold Genesys Recent Development13.5 Genprex13.5.1 Genprex Company Details13.5.2 Genprex Business Overview and Its Total Revenue13.5.3 Genprex Gene Therapies for Cancer Treatment Introduction13.5.4 Genprex Revenue in Gene Therapies for Cancer Treatment Business (2015-2020)13.5.5 Genprex Recent Development13.6 Momotaro-Gene13.6.1 Momotaro-Gene Company Details13.6.2 Momotaro-Gene Business Overview and Its Total Revenue13.6.3 Momotaro-Gene Gene Therapies for Cancer Treatment Introduction13.6.4 Momotaro-Gene Revenue in Gene Therapies for Cancer Treatment Business (2015-2020)13.6.5 Momotaro-Gene Recent Development13.7 MultiVir13.7.1 MultiVir Company Details13.7.2 MultiVir Business Overview and Its Total Revenue13.7.3 MultiVir Gene Therapies for Cancer Treatment Introduction13.7.4 MultiVir Revenue in Gene Therapies for Cancer Treatment Business (2015-2020)13.7.5 MultiVir Recent Development13.8 SynerGene Therapeutics13.8.1 SynerGene Therapeutics Company Details13.8.2 SynerGene Therapeutics Business Overview and Its Total Revenue13.8.3 SynerGene Therapeutics Gene Therapies for Cancer Treatment Introduction13.8.4 SynerGene Therapeutics Revenue in Gene Therapies for Cancer Treatment Business (2015-2020)13.8.5 SynerGene Therapeutics Recent Development13.9 Ziopharm Oncology13.9.1 Ziopharm Oncology Company Details13.9.2 Ziopharm Oncology Business Overview and Its Total Revenue13.9.3 Ziopharm Oncology Gene Therapies for Cancer Treatment Introduction13.9.4 Ziopharm Oncology Revenue in Gene Therapies for Cancer Treatment Business (2015-2020)13.9.5 Ziopharm Oncology Recent Development13.10 Anchiano Therapeutics13.10.1 Anchiano Therapeutics Company Details13.10.2 Anchiano Therapeutics Business Overview and Its Total Revenue13.10.3 Anchiano Therapeutics Gene Therapies for Cancer Treatment Introduction13.10.4 Anchiano Therapeutics Revenue in Gene Therapies for Cancer Treatment Business (2015-2020)13.10.5 Anchiano Therapeutics Recent Development13.11 Celgene10.11.1 Celgene Company Details10.11.2 Celgene Business Overview and Its Total Revenue10.11.3 Celgene Gene Therapies for Cancer Treatment Introduction10.11.4 Celgene Revenue in Gene Therapies for Cancer Treatment Business (2015-2020)10.11.5 Celgene Recent Development13.12 Celsion10.12.1 Celsion Company Details10.12.2 Celsion Business Overview and Its Total Revenue10.12.3 Celsion Gene Therapies for Cancer Treatment Introduction10.12.4 Celsion Revenue in Gene Therapies for Cancer Treatment Business (2015-2020)10.12.5 Celsion Recent Development13.13 Bluebird Bio10.13.1 Bluebird Bio Company Details10.13.2 Bluebird Bio Business Overview and Its Total Revenue10.13.3 Bluebird Bio Gene Therapies for Cancer Treatment Introduction10.13.4 Bluebird Bio Revenue in Gene Therapies for Cancer Treatment Business (2015-2020)10.13.5 Bluebird Bio Recent Development14 Analysts Viewpoints/Conclusions15 Appendix15.1 Research Methodology15.1.1 Methodology/Research Approach15.1.2 Data Source15.2 Disclaimer15.3 Author Details

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Gene Therapies for Cancer Treatment Market Growth by Top Companies, Trends by Types and Application, Forecast to 2026|Takara Bio, Tocagen, VBL...

Minneapolis May 1, 2020 (Thomson StreetEvents) -- Edited Transcript of Bio-Techne Corp earnings conference call or presentation Thursday, April 30, 2020 at 1:00:00pm GMT

* Charles R. Kummeth

* James T. Hippel

Robert W. Baird & Co. Incorporated, Research Division - Senior Research Analyst

Stifel, Nicolaus & Company, Incorporated, Research Division - MD & Senior Analyst

* Jacob K. Johnson

Janney Montgomery Scott LLC, Research Division - MD, Head of Healthcare Research & Senior Equity Research Analyst

SVB Leerink LLC, Research Division - MD of Life Science Tools & Diagnostics and Senior Research Analyst

Good morning, and welcome to the Bio-Techne earnings conference call for the third quarter of fiscal year 2020. (Operator Instructions) I would now like to turn the call over to David Clair, Bio-Techne's Senior Director, Investor Relations and Corporate Development.

David Clair, Bio-Techne Corporation - Senior Director of Corporate Development [2]

Good morning, and thank you for joining us. On the call with me this morning are Chuck Kummeth, Chief Executive Officer; and Jim Hippel, Chief Financial Officer of Bio-Techne. Before we begin, let me briefly cover our safe harbor statement. Some of the comments made during this conference call may be considered forward-looking statements, including beliefs and expectations about the company's future results as well as the potential impact of the COVID-19 pandemic on our operations and financial results. The company's 10-K for fiscal year 2019 identifies certain factors that could cause the company's actual results to differ materially from those projected in the forward-looking statements made during this call. The company does not undertake to update any forward-looking statements as a result of any new information or future events or developments. The 10-K as well as the company's other SEC filings are available on the company's website within its Investor Relations section. During the call, non-GAAP financial measures may be used to provide information pertinent to ongoing business performance. Tables reconciling these measures to most comparable GAAP measures are available in the company's press release issued earlier this morning on the Bio-Techne Corporation website at http://www.bio-techne.com. I'll now turn the call over to Chuck.

Charles R. Kummeth, Bio-Techne Corporation - CEO, President & Director [3]

Thanks, Dave, and good morning, everyone. Thank you for joining us for our third quarter conference call. This has been an extraordinary quarter. First, I want to thank all the employees, suppliers and partners of Bio-Techne for their exemplary service as we have worked through the COVID-19 pandemic. We are happy to report that we have no coronavirus cases in any of our global sites and implemented proactive safety measures very early in the crisis in February to protect our employees. Our balance sheet is sound with a very strong cash position and low leverage. We have not conducted any restructuring or furloughs to date, easing the minds of our employees and enabling them to focus on serving our life science research customers and clinical diagnostics partners.

While COVID-19 negatively impacted our business this quarter, primarily due to the temporary shutdown of academic labs, we are playing a critical role in the global fight to develop tests and cures for the virus. Overall, we were having a very strong Q3 until the virus pandemic had a pronounced impact on our business in mid-March. But even so, we ended the quarter with 6% organic growth. Adjusting for the impact of coronavirus in the quarter, we estimate our organic growth would have been closer to 9%.

Our team also delivered incredibly strong operational results in Q3, with adjusted operating margins expanding year-over-year by 130 basis points to 36.5%, ahead of schedule and with strong cash flow.

Now I'll get into some details of the quarter. Starting with our performance by geography, the North American market was very strong with low double-digit growth organically in the quarter. Biopharma growth in the region was over 20% while academia increased mid-single digit. In general, both end markets were impacted by COVID-19-related shutdowns starting in mid-March, although our academic market experienced a disproportionate negative impact from the virus. The good news is that these are academic labs, not large industrial manufacturing lines. As researchers return to the lab and get back to work, we anticipate utilization of our consumables will snap back very quickly.

Overall, biopharma research activity has been less impacted by the virus, with many labs continuing to operate at varying capacities. I don't believe we are alone in our belief that once this pandemic is under control, life sciences research will likely increase significantly. It appears everyone in society from governments through the private sector now knows what an antibody is, this can only be good for our industry. With current stay-at-home policies, many of our customers are spending much more time -- of their time online. Therefore, we continue to focus on our digital marketing efforts that have driven significant increases in website traffic across our brands over the past couple of years. Search engine optimization, continual refinement of our website and digital advertising will remain a growth lever for the company going forward and will be even more important as a differentiator in the current pandemic environment. We have never done more webinars and digital projects to reach out to our customers. For example, we recently held an Exosome Diagnostics webinar for urologists, many of whom are working from home now and had over 100 participants.

Moving on to Europe, which was positioned for a sequential recovery until the virus hit, finished the quarter with revenue down from last year by 7%. Here, we had a very strong quarter in Biologics as well as with our Simple Plex platform, which is seeing strong growth in COVID-19-related patient care decisions by monitoring cytokine storm onset and progression. However, this strength was not enough to offset the March shutdowns in academia, which especially impacted our Simple Western, Genomics, assay and reagent businesses in the quarter. It is still uncertain when European labs will begin to open back up, but we're expecting this to be country-by-country process.

So far, in April, our German business is showing signs of an initial recovery, with Italy potentially not far behind. Our largest business is in the U.K., which is likely to be a month or so behind Germany in the recovery process.

Finally, we had a surprisingly strong Q3 in Asia. Given the complete shutdown in China in late February, we were prepared for a quarter of negative organic growth in this country, but finished with positive organic growth in the mid-single digits. This fantastic finish reflects the outstanding work done there by the local team. Our employees in China are back to work serving -- servicing a research market that has largely turned back on and expecting to revert to their long-term trend of double-digit growth in Q4. The rest of Asia also performed extraordinary well in Q3 with growth in the mid-teens. However, many of these countries, including Japan, Korea and India are currently in varying states of lockdown and expected to remain so for most of Q4. Accordingly, we are expecting double-digit negative growth outside of China in Q4.

Before I turn the call over to Jim for the financial review, I would like to provide some more detail on the initiatives that our company is working on to help our customers develop tests and find remedies for COVID-19. I will also provide an update on some of the strategic growth initiatives that will have the largest impact to our company for years to come.

Starting with our COVID-19 initiatives, we are experiencing significant demand in ramping production of our proteins and antibodies used in coronavirus research, including reagents that are components in serological assays. Also, we are working with several health care providers in perfecting antibody assays using the traditional ELISA platform that can detect an individual exposure profile to COVID-19, something that many health experts believe will require mass testing before world economies can resume any sort of pre-COVID-19 normalcy. Also, as I mentioned in my commentary regarding Europe, we've experienced significant global interest in demand to leverage Ella as an automation tool for the detection and monitoring of COVID-19 patients that are potentially experiencing cytokine release syndrome. This dangerous condition represents a critical and potentially fatal point in disease management.

In our Genomics division, we are supplying COVID-19 RNAscope Pro for virus detection in tissue, allowing researchers to confirm the organs that are impacted by this virus. And in Exosome Diagnostics, we have validated and preparing to perform COVID-19 real-time qPCR testing at our labs in both Munich, Germany and Waltham, Massachusetts. Following the implementation of processes and instruments to automate the test, we'll be capable of processing over 200 samples a day, and we can ramp further after that. This lab-developed test will provide rapid and reliable detection of patients with active COVID-19 infections.

These are just a few examples of how practically every division of our company has pivoted resources to find innovative solutions for COVID-19 and participate on a global level to help eradicate this horrible virus. We are uniquely positioned to provide reagents and assays for all aspects of COVID-19 research and diagnostics, and our mission thrives in helping solve these types of problems.

Next, I will provide an update on 2 of our important strategic growth platforms, cell and gene therapy as well as Exosome Diagnostics. Starting with cell and gene therapy, we continue to make progress on the construction of our new dedicated GMP protein factory. Construction of the facility remains on track to provide GMP proteins in large-scale to our cell and gene therapy customers by the second half of fiscal 2021. We are very encouraged by initial interest in demand from biopharma customers as we actively market our coming GMP protein production capabilities and capacities. In fact, during the quarter, we executed our first large-scale GMP protein supply agreement. This customer is still making its way through the regulatory approval process for its therapy, making the GMP protein with a revenue likely in fiscal '21.

Many other customers have been engaging us with interest in long-term supply agreements to ensure continuity of supply for their present clinical studies and to meet their future commercial needs. In the meantime, our GMP portfolio has been growing at a rapid pace with a number of immune cytokines being used to grow cells in clinical trials.

Also within our cell and gene therapy business, we have begun operationalizing the joint venture with Wilson Wolf and Fresenius Kabi that we announced last quarter. As a reminder, this commercial consortium offers a complete and simplified cell and gene therapy workflow solution using products from all 3 parties. During Q3, we focused on establishing a unified sales structure, a customer-facing website and point-of-sale and creating impactful marketing collateral, featuring all 3 parents offerings. In addition, the emerging interest in natural killer cell therapies for oncology has been demonstrating some great clinical potential. And the JV is well positioned for the renewed natural killer interest, leveraging our GMP proteins, cloud, media and PC buster platforms for cell genetic engineering of difficult to grow natural killer cells.

Now an update on Exosome Diagnostics and the ExoDx prostate test. There were several positive developments in Q3, and there is still much to do to make sure this noninvasive prostate cancer test becomes available to all patients with heightened PSA levels who are contemplating a more expensive, more risky and painful tissue biopsy. Following the final local coverage decision, or LCD, from our medical -- Medicare Administrative Contractor or MAC, National Government Services, NGS, we have been billing Medicare for applicable patient tests and are receiving steady reimbursement for tests submitted to this important payer. Recall that our -- we are currently preparing for the reconsideration process with our MAC, NGS to get the LCD more accurately mirroring the more inclusive NCCN Guidelines, which will expand the potential market size of the test. Based on our prior experience, we anticipate a public comment period to take place in the summer, followed by a final LCD in the fall.

Continuing on the reimbursement front, we announced the receipt of an unlimited 10-year reimbursement contract with General Services Administration, or GSA, making the ExoDx prostate test available to more than 140 government entities, including the Veterans Administration health care system. Our conversations with private payer community continue to progress, but many payers want to see a clinical utility study published prior to issuing favorable coverage decisions. We expect such publication in a leading peer-reviewed urology journal within the next couple of months. The clinical utility study shows improved patient compliance to either defer prostate biopsy or proceed with biopsy compared to the control arm when implementing the ExoDx prostate test in clinical practice. Due to this increased compliance to proceed with biopsy, physicians detected 30% more cases of clinically significant or high-grade prostate cancer compared to the standard of care control alarm. Implementing EPI not only saves the health care system money by avoiding unnecessary biopsies but also increases compliance among patients that need biopsies, leading to higher cancer detection rates. We anticipate the clinical utility and economic value shown in this study will resonate with the private payer community, increasing the frequency of our conversations and strengthening the argument for favorable coverage decisions.

Currently, the coronavirus pandemic has had an impact on a number of individual-seeking wellness business which in turn has had an impact on the number of PSA tests and ultimately the number of ExoDx prostate tests conducted. We saw this play out during the month of March, and the decline in test has continued into April. We responded by recently launching an at home collection kit for our ExoDx prostate test, enabling men who are sheltering in place and thus unable to see a health care professional to know if a biopsy should be prioritized. The solution was launched with a patient-targeted marketing strategy, including Search Engine Optimization, a Facebook campaign and webinars to drive awareness that patients do not need to go into the urologist's office to have access to this valuable test. We believe the flexibility of providing a urine sample at the convenience of the patient will be yet another key differentiator of the ExoDx prostate test from the competition.

With a pipeline of additional tests, companion diagnostic applications and partnership opportunities, there are multiple avenues to create value with Exosome Diagnostics. We have a few partnerships in place and are in active discussions with several diagnostics and biopharmaceutical companies for potential applications of Exosome Diagnostics technology. In summary, I'm very proud of the way our team executed in our fiscal third quarter, especially considering the global challenges created by COVID-19. The pandemic creates some near-term challenges, but also some near-term opportunities as we deploy reagents to enable vaccine and therapeutic discoveries and diagnostic solutions. Stepping back from the current environment, our pillars for growth are fully in place. We remain in the very early innings of realizing a tremendous liquid biopsy and cell and gene therapy potential opportunities. And our proteomic and genomic and analytical tools remain extremely well positioned in very underpenetrated markets. I firmly believe that the environment for life science research and diagnostics will be even stronger as we emerge from this pandemic, and we have the financial strength and product portfolio to capitalize on these opportunities. With that, I'll turn the call over to Jim.

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James T. Hippel, Bio-Techne Corporation - Senior VP of Finance & CFO [4]

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Thanks, Chuck. I will provide an overview of our Q3 financial performance for the total company, and provide some additional color on the performance of each of our segments, and give some initial thoughts on potential scenarios for our Q4.

Starting with the overall third quarter financial performance. Adjusted EPS was $1.39 versus $1.21 one year ago, with foreign exchange positively impacting EPS by $0.07. Most of the foreign exchange impact was due to nonoperating foreign exchange related to our cash pooling arrangement. GAAP EPS for the quarter was $0.92 compared with $1.15 in the prior year. The biggest driver for the decrease in GAAP EPS was $400,000 in realized and unrealized gains on our investment in ChemoCentryx compared to the $12.3 million in unrealized gains in the prior year. Q3 revenue was $194.7 million, an increase of 5% year-over-year on a reported basis. Organically, revenue increased 6%, with foreign exchange translation having an unfavorable impact of 1% and less than 1% growth contribution from acquisitions.

By geography, the U.S. grew in the low double digits, while Europe declined approximately 7% and China grew mid-single digits. As for the rest of Asia, organic growth was in the mid-teens. By end market, which excludes Asia, our Diagnostics division and other OEM customers, biopharma growth was in the low teens, while academic sales decreased by the low single digits.

Moving on to the details of the P&L. Total company adjusted gross margin was 71.5% in the quarter compared to 71.3% in the prior year. The increase was due to volume leverage, partially offset by foreign currency headwinds and, to a lesser extent, the B-MoGen acquisition. Adjusted SG&A in Q3 was 26.8% of revenue, a 90 basis point improvement compared to the prior year, with volume leverage partially offset by investments in our core business to drive near and long-term growth. R&D expense in Q3 was 8.2% of revenue, 20 basis points lower than the prior year, primarily due to volume leverage. The resulting adjusted operating margin for Q3 was 36.5%, an increase of 130 basis points from the prior year period and 310 basis points higher than our second fiscal quarter result.

Looking at our numbers below operating income. Net interest expense in Q3 was $4.2 million, decreasing $0.7 million compared to the prior year period. The decrease was due to a substantial reduction of our bank debt during the first half of fiscal 2020. Our bank debt on the balance sheet as of the end of Q3 stood at $420 million. Other adjusted nonoperating income was $3 million for the quarter compared to $1.5 million of other expense from Q3 last year, primarily reflecting the foreign exchange impact related to our cash pooling arrangements. For GAAP reporting, other nonoperating income includes realized and unrealized gains from our investment in ChemoCentryx.

Moving further down the P&L, our adjusted effective tax rate in Q3 was 21.3%, and we are anticipating Q4's rate to be closer to our year-to-date adjusted tax rate of 21.6%.

Turning to cash flow and return of capital. $49.5 million of cash was generated from operations in the quarter, up 25% over Q3 of last year. We also added to our cash position by monetizing approximately $30 million of our ChemoCentryx investment during the quarter. In Q3, our net investment in capital expenditures was $9.3 million, mostly driven by construction of our new GMP protein factory, which remains on schedule for completion by the end of the calendar year. And during Q3, we returned capital to shareholders with $12.3 million of dividends and approximately $50 million of share buybacks, leaving 39.4 million average diluted shares outstanding at the end of the quarter.

Next, I'll discuss the performance of our reporting segments, starting with the Protein Sciences segment. Q3 reported sales were $145.5 million, with reported revenue increasing 6%. Organic growth was also 6%, with foreign exchange having an unfavorable impact of 1% on revenue growth and acquisitions contributing 1% to revenue growth. Growth in the segment was driven by strong demand for protein reagents and both our Biologics and Simple Plex instrument platforms, partially offset by the impacts of COVID-19 customer shutdowns. Operating margin for the Protein Sciences segment was 44.7%, a decrease of 40 basis points year-over-year due to unfavorable foreign exchange and the B-MoGen acquisition.

Turning to the Diagnostics and Genomics segment. Q3 reported sales were $49.4 million, an increase of 5% from the prior year. Organically, revenues also grew 5%, with foreign exchange translation having an immaterial impact on revenue. Our Genomic divisions RNAscope contributed solid double-digit growth in the Americas, but this growth was partially offset by COVID-19 customer shutdowns in China as well as Europe. Exosome Diagnostics contributed approximately 3% to the segment's growth in Q3, with Medicare collections over $1 million following the final lab coverage decision by NGS in December.

Moving on to operating margin for the Diagnostics and Genomics segment, at 14.3%, the segment's operating margin improved from 7.6% reported in the prior year. The increase reflects a near double in operating profit due to less dilution from Exosome Diagnostics, volume leverage from our Genomics division and productivity gains from our diagnostic tools division. As many of you know, it is not our policy to provide specific annual or quarterly guidance, but instead provide some high level color, and how we anticipate our business to perform relative to our longer-term strategic plan. Clearly, the evolving COVID-19 pandemic and related customer impact was not contemplated in our long-term strategic plan nor a shorter-term annual plan. Therefore, I thought it'd be helpful to provide some color on the impact we've seen from COVID-19 so far in April and possible scenarios of how it could play out for our Q4.

Let me begin with the headwinds we've seen so far in April. With most academic institutions shut down in the Americas and Europe and some nonessential research put on pause by biopharma, our run rate in reagent and assay businesses in these geographies has dropped off considerably from prior year levels in the range of minus 20% to minus 40%, depending on the product line and specific regions. Encouragingly, there have been recent talk in both Europe and the U.S. about slowly and systematically opening up universities for professors to conduct their research, even if uncertain about students returning in the fall. Obviously, when it is safe to do so and this talk becomes action, our run rates will gradually improve as researchers return to their labs.

Chuck has already mentioned the headwinds we face in Asia outside of China due to reoccurrences of the virus in places like Japan and Singapore as well as the full walk-down in India. However, China is looking like a nice tailwind for us in Q4 with growth rebounding well into the double digits. Other tailwinds for Q4 include our Biologics and Simple Plex instrument product lines. Just as they led our growth in Q3, these platforms have started out just as strong in April, and we expect a high demand for them to continue, especially the Simple Plex platform, which is specific to COVID-19 treatment-related needs.

And finally, we should experience some tailwinds from all the other COVID-19 initiatives that Chuck previously discussed. But as many of these products are still currently under development, knowing exactly how much they will contribute to Q4 revenue is difficult to project. All this being said, our best estimate of how these variables will play out, results in a Q4 organic growth being somewhere in the range between minus 10% and minus 20%. Because we do not anticipate that our higher-margin reagent business so -- because we do anticipate that our higher-margin reagent business will be more severely impacted by the shutdown. This will likely have a material negative impact on gross margins for the quarter. Our operating margin will likely be further pressured by our operating cost base being relatively flat to prior year on much lower revenues. Right now, we are assuming that we have seen the worst impact from COVID-19 in April, and that conditions will gradually improve from here. And when they do, we will emerge as an even stronger company as we continue down our path of executing on our strategic plans and key growth initiatives.

In summary, despite unprecedented conditions created by COVID-19, we delivered 6% organic growth in the third quarter. We are in a position of financial strength with cash and short-term investments in excess of $250 million and a leverage ratio of just 1.4x. One thing this global pandemic has clearly highlighted is the need for life sciences research, and we believe that global demand for our tools, which enable drug discovery and production, genomic and proteomic analysis and diagnostic solutions will emerge from this pandemic stronger than when it began. Our global teams and product portfolio are positioned to capture that stronger demand in addition to our original growth plans when the virus-related headwinds subside. With that, that concludes my prepared comments, and I will turn the call back over to the operator for the line for questions.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from the line of Puneet Souda with Leerink Partners.

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Puneet Souda, SVB Leerink LLC, Research Division - MD of Life Science Tools & Diagnostics and Senior Research Analyst [2]

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So Chuck, first of all, congrats on a solid quarter here despite the disruption. And it's good to see the resiliency in China and very tough compares that you had already from last year. So in April, if you could -- thanks for the comments, but I wanted to understand how is -- how do you view the recovery here for academic and biopharma segments, just sort of given the behavior academic has been impacted more heavily? Any sort of early signs in the latter part of April that you're seeing here that give you a better picture into May in either of the biopharma segments or the academic segments? Then I have a couple of questions.

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Charles R. Kummeth, Bio-Techne Corporation - CEO, President & Director [3]

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Sure. Well, biopharma is still pretty resilient. And I think as we talked about, I think we have some upside even there with a lot of instrumentation that is hotter than ever. Biologics is doing really well. We are starting to see that drift into cell and gene therapy qualification. So we expect that platform to continue to do well. Simple Western is a little bit under pressure because unlike biologics, it's kind of 50-50 with academia. So a little softer. It's anyone -- yes, when things start opening up. I mean, you're hearing it already state-by-state, including lawsuits. There's a lot of fatigue out there. People want to go back to work. And with that, labs will reopen. And I think people are also getting creative with what they can start doing. And then we are seeing more activity online and a lot of webinar activity. And so end of April, maybe not so much material. By May, we're hoping some. Certainly, the -- in Q1, we expect to see things starting to come back. So we're hoping this is more of a V versus a long U. And that's kind of where we're looking at it.

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James T. Hippel, Bio-Techne Corporation - Senior VP of Finance & CFO [4]

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Another way -- another way to think about it too is we're gradually opening up the economy to go back to work. A lot of it is going to require social distancing practices while at work. There's an argument that can be made that in labs, in general, it's potentially easier for those practices to be put in place, perhaps in other types of industries. So another reason why we think that once the economy does, it starts to gradually reopen up life science research will be one of the earlier phases of that.

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Charles R. Kummeth, Bio-Techne Corporation - CEO, President & Director [5]

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Well, I know in our own labs, which is remarkably a lot like every other lab, that we're pretty social distant anyway. People aren't just on top of one another in these labs. So I think there's a good -- it's just going to take time for people to acknowledge that and kind of get into it. But the world is going to be different. There will be masks that work for quite some time, I'm sure, and a lot of different things. But a lot of our people wore that stuff and gloves anyway. So I don't think there's been too much issue there. Pretty amazing to see China just kind of turned back on. If there any good data point that if you come back and follow good social distancing, everybody wears a mask, it looks like there's a good promise that we don't have a reescalation. So anyone guess over what happens with wave 1, 2, 3 over the next coming years. I'm sure there'll be some of that, too, but we're very, very optimistic that when this is all done, the paltry $40 billion NIH budget is going to go up dramatically. So it's a rounding year compared to what's been lost and what's been thrown around here for stimulus. So research is going to go up, and we're perfectly positioned. And by the way, we're going to be plugging some of this hole too with COVID-19 projects, which we alluded to. I wish we had more positive press release type news to give you, but we're working on many projects here than some that could really scale. But it's too early to tell you. If and when we come out with tests, serological or other, you'll know that it will have the R&D systems brand-new quality behind them. So it won't -- it will be something remarkable and with high sensitivity and specificity, and we're working on technology platforms around what we're good at, antibodies and proteins that can be differentiated from everybody out there and who is chasing the stuff. So stay tuned, more to come.

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Puneet Souda, SVB Leerink LLC, Research Division - MD of Life Science Tools & Diagnostics and Senior Research Analyst [6]

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Okay. That's helpful. So following on that, in COVID research, I know it's tough to quantify this, but do you expect to see any benefit from the $25 billion COVID testing and testing research allocation for the -- as part of the coronavirus relief fund? And what's your expectation here? Just in terms of the portfolio, the breadth of the portfolio that you have, from antibodies to cytokines. I mean, how should we think about what parts of the portfolio are likely to benefit when that NIH -- potential NIH increase that could happen in 2021? And so what parts of the portfolio should we expect to benefit from that because there is some expectation that could be directed more towards infectious diseases versus other traditional oncology research and other areas?

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Charles R. Kummeth, Bio-Techne Corporation - CEO, President & Director [7]

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Well, it's anyone's guess how much will go one way versus the other in infectious diseases, but in terms of our portfolio, all of it. I can't imagine that there won't be an amazing interest continuing in the proteins that were developed. We're selling a lot of proteins that things are going up a lot in a lot of categories that are used in this research, and also with antibodies. And poly through monos, it's been pretty exciting. We almost can't keep up in some areas. So we're working on scalable solutions that can resupport partners for things like serological. For -- there's been a lot of relaxation with the FDA to try and have something work out there, and there's been some negativity around that. But I can guarantee you that we're on top of this and involved in many fronts and our reagencies are going to do really well. The tools are going to do well. You got to measure this stuff. We are the worldwide leader in ELISA Kits. And ELISA Kit is the best way to do a serologic. It's the cheapest, it's fast. Everybody has automation. So we're a world leader in Luminex with many partners. Luminex is another way to attack a lot of stuff in there. Everyone's got their flavor of these different partnerships and deals. And it's just become so vast. And like we talked about here, when do you see a new $1 trillion plus market open up overnight? And it's -- there are a lot of opportunities here, and we get the best of both worlds to chase the new market, but also do something wonderful for society. We're going to help eradicate this virus. We're involved on more fronts than we can even tell you. In terms of resources pivoted, virtually over 200 employees have been refocused around these projects in the company. And they may not all work out. We may win in some, lose in others or be a fast follower in others, but a lot of it or more, been around a lot of innovation, a long time. And these things always lead the stuff. And the funding is going to trickle down to touch many parts of our business. There's no doubt in my mind. It's hard to quantify right now, but it's going to be -- you got to look beyond Q4 and even Q1. You got to look into next year and the year after, we're going to be stronger than ever with the base business, and we're going to have all this incremental new business on top of it. And then we still have our cell and gene therapy and Exosome Diagnostics platform is just starting to ramp. So for us, it's a better story than ever. As we start looking out 1, 2, 3 years, I mean, in our opinion, in Q3. Our Q3 results just kind of make that indicative. We had a pretty darn good quarter.

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Puneet Souda, SVB Leerink LLC, Research Division - MD of Life Science Tools & Diagnostics and Senior Research Analyst [8]

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Yes. Last one, if I could ask on China. That was a surprisingly good quarter given sort of compared to what we were heading into. The shape of resiliency that you're seeing there currently, what's your expectation? How long do you think -- I mean, sort of how long is that sustainable? There are some questions being raised on the recurrence of the virus. Obviously, China was ahead of the curve. So what are you hearing from your team on the ground there?

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Charles R. Kummeth, Bio-Techne Corporation - CEO, President & Director [9]

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Well, we have our whole team in, and we have our management in, too, including our leaders had to go in and go through quarantine to get back in the country. The country has got very, very strict guidelines to get in. So they're not letting anybody in without going through quarantine. So that's a big part of why they're staying safer probably. Everybody is wearing masks, according to our team. If you're -- China is one of those countries where there's a lot of lines in and so social distance up to this point was a very small distance. It's going to change and it is changing. And there's not been a material resurgence. So if there's no resurgence, we think it's, like we said, back to double-digit or higher already this quarter. So nothing but up. If there's a resurgence and there's new rules going in place, we'll have to adjust. But right now, there's nothing to worry about there. I'm frankly, much more concerned about what happens in India. And Japan and Korea are both a little behind the curve right now as well. So although we had a wonderful Q3, it's because really, they were just late to the game of the lockdowns. So as they work through their lockdowns this quarter, we'll have that hit, but hopefully, they'll have the same coming out that China did. But judging what we're seeing in Brazil right now, I don't like seeing that because I think India is really at risk. But -- and that's a big growth platform for not this year per se materially, but looking 5 years out, so it could put some risk into that. But I think Korea and Japan will be fine after a quarter here. And I think China is moving forward. We don't see a real hit. Europe is a slower recovery and it's country-by-country. We were having an outstanding quarter until this hit. And -- but I do think we are on track with a lot of fixing of Europe already, execution-wise, which is in place. And so as they go back to work and labs reopen, I think we'll be in a much better spot in Europe as well. U.S. has been phenomenal. So -- and it remains so. And this is where we're going to see a lot of our COVID-19 incremental growth this quarter and beyond. So hopefully, we'll have nothing to show but extra positive news there. So this is almost sadly the event we're looking for, for Ella. Ella is finally going to see its day. And we're up -- we expect 75% growth in Ella this quarter and probably a double for next year again. So it's going to become a material platform, finally, when this is all said and done. So -- and our ability to get it through all the FDA-related clearances has become easier, obviously. So...

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Operator [10]

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Our next question comes from the line of Catherine Schulte with Baird.

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Catherine Walden Ramsey Schulte, Robert W. Baird & Co. Incorporated, Research Division - Senior Research Analyst [11]

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Congrats on a nice quarter. I guess, first, just going back to China. We've heard from other companies that academic and government labs have lagged in terms of reopening. Sounds like you are not seeing that, but just curious to hear, what was the low watermark for activity in China? Was that at 1 point down 20% to 40% as well? And what did that path to recovery look like?

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Charles R. Kummeth, Bio-Techne Corporation - CEO, President & Director [12]

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It never went negative. And we ended mid-single digit. We were expecting -- we do it like everyone, we got a monthly flash. We had a monthly process, and we were expecting maybe minus 2, minus 3. That's the team was thinking, but it never got down there, and we ended up mid-single digit. I think you're seeing a difference us versus others. And other peers have a lot heavier instrumentation level in their portfolio than we do. We're also not that big, right? So on the reagents front and the things that can back up to speed quick in lab, that's kind of us. So it's a quicker recovery. If you're dealing in mass spec and HPLC and things like that, it's going to be slower. So I think you're seeing some of that. And -- but we were on a nice growth level anyway. So I'm not surprised to see this as long as labs are back at work. The kind of research they do with us with a lot of reagents level testing is something that will snap back quickly, which we also expect to see as well following in Europe and U.S. that's why we made the comments that we did about a fast recovery once labs reopen.

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James T. Hippel, Bio-Techne Corporation - Senior VP of Finance & CFO [13]

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Edited Transcript of TECH earnings conference call or presentation 30-Apr-20 1:00pm GMT - Yahoo Finance

Cryopreservation has become an indispensable step in the daily routine of scientific research as well as in a number of medical applications, ranging from assisted reproduction and transplantations to cell-based therapies and biomarker identification. It is hardly possible to picture todays scientific and medical advancements without this technique.The successful development and implementation of all the therapeutic and scientific discoveries involving cryopreservation relies on the correct and safe translation of the method from the laboratory to the clinical and manufacturing scale.

With the need to correctly use this technique, more research is focusing on optimizing cryopreservation methods and investigating what the long-term effects and consequences are on the physiology of the cryopreserved material.

An important part of cell therapy research is focused on adult stem cells (ASCs). ASCs can be derived from different sources such as peripheral blood, bone marrow or adipose tissue and display strong promises because of their capacity to differentiate into any cell type of the human body.In recent work3, the team of Michael Pepper at the Institute for Cellular and Molecular Medicine in Pretoria, South Africa, explored the effects of cryopreservation on the differentiation ability of adipose tissue-derived stem cells (ADSCs). After analyzing gene expression of key adipogenic genes and the degree of differentiating cells, characterized with high levels of CD36 and intracellular lipid droplets, the scientists reported that slow freeze cryopreservation of cells shortly after their isolation causes no alterations on their ability to differentiate. Pepper is convinced of the necessity to perform such analysis when cryopreserving important cell pools: It is critical to do a post-thaw analysis of cell function to determine how the cryopreservation may have affected the cells.His team is analyzing the effects of cryopreservation on other cell types largely used in cell-based therapies such as hematological stem cells and peripheral blood mononuclear cells (PBMCs). Although they didnt observe major alterations in terms of immunophenotyping or the post-thaw proliferation of the cells, Pepper expresses his concern that more subtle characteristics might be affected.

Correct cryopreservation of cells intended for therapeutic use is crucial. This is very important particularly as cells may persist for a long time in the recipient. This area of cell therapy research definitely requires more attention, Pepper says. Moreover, his words reflect on the need to evaluate not only the direct post-thaw recovery, but to look deeper into the late-onset effects cryopreservation might have and ensure that transplanted cells have preserved their therapeutic properties.

In contrast to slow freezing, vitrification relies on the fast freezing of the material by putting it in high concentration of cryoprotectant and in contact with liquid nitrogen. This method allows the direct transition of water from liquid to solid state without crystal formation. The highly concentrated cryoprotectant prevents ice formation and therefore there is no need for slow cooling.

Although vitrification has a great potential, there are a couple of parameters that are a point of concern. The quick and drastic freeze is possible thanks to the high concentration of cryoprotectant, but the latter is also associated with higher toxicity. In some cases, an additional limitation is the direct contact of the sample with liquid nitrogen which is a predisposition for viral or bacterial contamination.The team of Christiani Amorim at the Institute for Experimental and Clinical Research in Louvain, Belgium, is approaching the challenges of vitrification in the context of ovarian auto-transplantation. Ovarian auto-transplantation consists of preserving a piece of ovarian tissue with active follicles from the pre-therapeutic ovary of a cancer patient, as chemotherapy often has damaging effects on the reproductive organs. This tissue sample will be conserved and auto-transplanted onto the patients ovary when she has recovered and wishes to become pregnant.In their recent research4, the authors used stepped vitrification, in which the concentration of the cryoprotectant is gradually increased while simultaneously temperature decreases. This avoids ice crystal formation and also prevents cryoprotectant toxicity.Although stepped vitrification has previously given good results in bovine ovarian tissue5, this was not the case for human ovarian tissue. The scientists didnt detect normal follicles following thawing and linked this to high cryoprotectant toxicity. Indeed, they observed all signs of dimethyl sulfoxide (DMSO)-related cell membrane damage: significant organelle damage, cell membrane disintegration and apoptosis. These observations imply on the variability of outcomes that the method could give when applied to the same type of tissue but from a different organism.Amorim is positive about the future of their method and recognizes the need for further research on the topic: I can see a great potential in the stepped vitrification approach, but I also believe that there is a lot we still need to learn before thinking about using it as method of choice for human ovarian tissue cryopreservation. The high cryoprotectant concentration that should be applied in this approach is my first concern. () Our study clearly showed that 50% DMSO is too high, so we need to try lower concentrations or combine it with other cryoprotectants.

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Freezing Life: The Current Trends in Cryopreservation - Technology Networks

COVIDS WAR ON WOMEN During this plague year, there is almost never good news, only degrees of bad news. Even so, the pandemic has been different (and worse) for girls and women.

Its true that more men are dying than women from Covid-19 around the world but thats not exactly cause for celebration.

Another ambivalent data point: More workplace risk is falling on women, who are more likely to be considered essential workers. The upside to that is still having a job, but at what price? Swedens Foreign Minister Ann Linde pointed out today in a POLITICO interview that 70 percent of those working in health care and elderly care are women.

More of the daily grind tends to fall, on average, on women: From the increased cleaning and chores that come with more time spent in the home, which falls disproportionately to so many female household members, to the extra education and childcare work created through closures of school and day care, where men have also been known, on average, to skimp.

The real-life examples are heartbreaking: Alice Jorge, a woman living with a disability in Belgium who needs support from her sister and a visiting nurse, was recently asked to choose between keeping her Covid-19 positive caregiver or going without professional care. Three women bound to suffer no matter what choices they took.

Domestic violence is up sharply: A new research report by a consortium that includes Johns Hopkins University confirms this: 31 million additional cases of gender-based violence can be expected globally if lockdowns last for an average of six months.

Travel to shelters may be restricted, and a simple phone call to a helpline can itself trigger new violence. Support services are overwhelmed with requests: from a 47 percent increase in calls to Spains national hotline to a 113 percent spike at U.N.-supported hotlines in Ukraine.

We can expect 7 million unplanned pregnancies in 144 low- and middle-income countries, thanks in part to restricted access to contraception, not to mention the 2 million female genital mutilations and countless child marriages projected to increase by the United Nations population agency. The pandemic is deepening inequality, UNFPA Executive Director Natalia Kanem said, slamming the Swiss cheese of a safety net she sees in most countries.

Kanem speaks of childbirth horror stories: pregnant women unable to access caesarian procedures (many of which are unplanned) or blood pressure medication because of redeployed health care resources, or the woman gets to the clinic (and) the midwife isnt there, because theyre also redeployed or sick. Up to two-thirds of maternal and neonatal deaths globally occur because of the absence of properly trained midwives in better times.

During World War II, women on the U.S. homefront think Rosie the Riveter entered the workforce out of a call to sacrifice for the common good. During this pandemic, women are being called back but this time to the frontlines.

Welcome to POLITICO Nightly: Coronavirus Special Edition. Of course Matthew McConaugheys mask says just keep livin. Reach out with tips: [emailprotected] or on Twitter at @renurayasam.

A message from PhRMA:

In these unprecedented times, Americas biopharmaceutical companies are coming together to achieve one shared goal: beating COVID-19. We are working with governments and insurers to ensure that when new treatments and vaccines are approved, they will be available and affordable for patients. Explore our efforts.

THE COVID DOCTRINE For much of the nations 100 days at war with the coronavirus, Donald Trump has been a commander in chief in search of an exit strategy, Adam Cancryn writes. The president has promised the virus will simply disappear, touted unproven treatments as miracle cures and fantasized about a near future of economic resurgence and rapid return to normalcy. Yet as the White House shifts its focus away from the public health response and toward rebuilding an economy ravaged by the pandemic, there remains little clear sense even within his own administration of how close the U.S. is to victory, and what winning the war even looks like.

PINS AND NEEDLES Our executive health editor Joanne Kenen emails: Theres some good news on the vaccine front including word that the country is getting a new vaccine leader. Peter Marks has emerged as the Trump administrations unofficial vaccine czar (minus the cross and pearls) at the FDA, filling in the gap created by the abrupt ouster of Rick Bright from the Biomedical Advanced Research and Development Authority. Marks will advise BARDA and other agencies on vaccine and gene therapy approval, health care reporter Sarah Owermohle reports.

News of Marks growing involvement comes amid a spate of heartening though still, we cant emphasize enough, very preliminary news about vaccine development, here and abroad. Oxford has a candidate vaccine thats safe in humans; its still testing whether it can create a strong enough immune response to combat the coronavirus. Three other companies have announced accelerations of clinical trials, though widespread availability in the best possible scenario is still months away.

Even if we get a vaccine, Joanne writes, big questions have to be answered.

Who gets it second? First responders will get it first. But how are we going to define a first responder? Anyone who works at a hospital? Only doctors and nurses? Doctors in the community? Police? Firefighters? The military?

But then who gets it second? The elderly and immune compromised because they are vulnerable? The young and healthy because they transmit it? Essential workers because theyre essential? The well-connected? And who decides?

Its possible several vaccines will come on the market at around the same time in different countries, so there could be multiple answers to this question. But this is going to be a huge bioethical knot, colliding with geopolitics. Theres no guarantee that a U.S. company will get to market first and if the World Health Organization has a role in vaccine allocation, we can anticipate some obvious conflicts.

How effective is it? If we get a good but not great vaccine like the seasonal flu shots it will still reduce transmission, but it wont wipe out the virus completely. Well still have to deal with Covid-19, though on a more manageable scale.

Who pays for it? Even if insurers, governments or, in countries other than our own, national health systems pay for immunization, the costs can be passed on indirectly through higher taxes or higher premiums.

How much does it cost? Some of the companies say they dont plan to make a profit, but vaccines are expensive. In the U.S., Trump has largely shunned national approaches, leaving states to fend for themselves as they try to acquire lab testing supplies, protective gear, ventilators and other essential pandemic-fighting goods. A similarly fragmented approach could make vaccine acquisition more expensive and complicated.

How do we make enough of it? One of BARDAs roles is to help ramp up production, and theyve started addressing this. But to make 7 billion vaccines, enough for everyone around the globe, will require commitment, creativity and cooperation that the world hasnt been very good at of late.

How fast will poor countries get access? Good question.

Will the anti-vaxxers take it? Well see. Best guess is that some will, and some wont, because not everybody who opposes vaccines does so for the same reasons or with the same intensity. Some people who dont want their child to get a measles shot may weigh the costs and the benefits differently for a coronavirus vaccine. Amid rising fears of bioterrorism after 9/11, a poll found deep but not overwhelming support for a smallpox vaccination campaign. But that was a hypothetical threat. This one is all too real.

A SMALL BREAKTHROUGH More than three decades ago, researchers made their first big breakthrough against HIV, when they showed that the drug AZT could slow the progression of the virus. Its a moment that Anthony Fauci compared to todays results about the drug remdesivir, which a clinical trial showed could help Covid patients recover more quickly.

Faucis reference to AZT was a bit like a secret code, Sarah Owermohle tells us. He was suggesting that the remdesivir results were a breakthrough, but a modest one. AZT is the shorthand for azidothymidine, a drug that won FDA approval in March 1987, when HIV patients were desperate for any treatment even one with rough side effects that was dogged by questions about whether it actually extended life. It took another decade before the development of drugs that turned HIV from a death sentence into a chronic condition.

HIV and Covid-19 are complex, but distinctly different, viruses, and drug development times are a lot faster now than they were in the 1980s and 90s. But Faucis implication was clear: Remdesivir could be a good first step in fighting Covid, but probably isnt a miracle drug.

A SICK ECONOMY The U.S. economy shrank at a 4.8 percent annual rate last quarter as the pandemic shut down much of the country. A huge percentage of the decline came from the health care industry, with a halt in elective procedures harming profits.

CLAIMS DENIED As businesses in Georgia, Texas and other states throw open their doors, many employees are scared that their employers arent taking proper health precautions. Yet if they refuse offers to return to their jobs theyll be ineligible for unemployment, reporter Megan Cassella tells us.

Trump has declared meatpacking plants essential businesses even as they spawn outbreaks across the country. Frontline health workers are having trouble getting masks, gloves, gowns and other protection equipment, so what hope do nail salons and restaurants have of getting the gear they need?

But for now, Covid fears arent a valid reason not to go back to work.

Some states are trying to take steps so that workers who feel unsafe arent forced to choose a paycheck over their health. Colorado and New York are looking at how to give workers more flexibility. In Georgia, the state labor agency is encouraging employers to negotiate back-to-work plans with employees so that if a business partially reopens, workers who feel unsafe can continue to collect unemployment. In Texas, advocates are asking the workforce commission to add voluntarily leaving work due to COVID-19 as a valid reason to claim assistance.

But other states, like South Carolina and Tennessee, are telling workers they will lose unemployment aid the same week they turn down an offer.

Even in boom times, states reject a high share of unemployment claims. Well probably learn Thursday that another 3.5 million people filed for unemployment assistance last week. Thats on top of the 26 million whove already lost their jobs in the past five weeks.

Our question for readers this week: Seeing any interesting, fun or meaningful signs related to the coronavirus? Snap a photo sometime this week and send it to Renu at [emailprotected], and well share the best ones on Friday.

GRAND OLD PACHYDERM Matt Wuerker dives into an old question on partisan symbols in the latest edition of Punchlines: Why is the elephant the symbol of the Republican Party?

MASS HYSTERIA Italian politicians clash with the Catholic clergy at their own peril, and Prime Minister Giuseppe Conte has risked doing just that by keeping churches closed because of coronavirus. The prime minister's decision to extend the ban on all religious ceremonies until further notice, except for funerals, has infuriated religious officials. When the lockdown started, most priests quietly accepted the need to suspend services and found alternative ways to connect with their flocks, such as holding ceremonies by video or taking confession by the roadside. But now that other places are gradually reopening, the clergy don't see why they should be last on the list.

82,000

The number of job losses forecast in the bus industry, according to a report released last week by the American Bus Association. The industry could see losses of up to $14 billion. Many of the 3,000 private bus companies in the U.S. are small, serving a range of uses from taking kids to school, sporting events and field trips, ferrying seniors on weekend getaways and connecting small towns with major destinations. (h/t transportation reporter Tanya Snyder)

Portuguese army chief of staff Gen. Jos Nunes da Fonseca attends a briefing of school workers on disinfection procedures. | Armando Franca/AP Photo

DEEP FRIED STATE Belgium, the North Sea homeland of moules frites and mayonnaise, is the world's biggest exporter of frozen fries, but it has been hammered by the pandemics trade slowdown. The Belgian potato industry has warned that more than 750,000 tons of potatoes could be thrown away more than 40 percent of the harvest. And though Belgium's potato industry has urged patriots to take a high-calorie hit for the team by heading down to their local friteries twice a week to help reduce the spud surplus, it's increasingly clear that 11 million Belgians won't be able to handle the deep-fried mission alone. With restaurants and bars closed, and large summer events canceled, fries wont be as ubiquitous as they often are this summer. "Our entire sector is facing a big crisis. We don't just invite all Belgians to eat more fries, but the entire world," said Ward Claerbout from Agristo, a potato processing company in the west of Belgium.

Correction: Tuesdays edition of POLITICO Nightly incorrectly stated which tracks Iowa will open without spectators. The state will reopen certain race tracks without spectators but not horse and dog tracks. We regret the error.

A message from PhRMA:

In these unprecedented times, Americas biopharmaceutical companies are coming together to achieve one shared goal: beating COVID-19. The investments weve made have prepared us to act swiftly: Working with governments and insurers to ensure that when new treatments and vaccines are approved, they will be available and affordable for patients Coordinating with governments and diagnostic partners to increase COVID-19 testing capability and capacity Protecting the integrity of the pharmaceutical supply chain and keeping our plants open to maintain a steady supply of medicines for patientsExplore our efforts.

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Covid's war on women - Politico

Advanced Therapy Medicinal Products Market: Overview

Lately, there has been remarkable shift in the therapeutics method. People are inclining towards personalized medication rather than pharmaceutical treatment methods. This is result of advancements in biological therapies. As a result, advanced therapy medicinal products market (ATMP) is emerging. Such products provide solution for conditions with no therapeutic alternatives. This is a key factor driving growth of the advanced therapy medicinal products market across the globe.

Get Sample Copy of the Report @https://www.tmrresearch.com/sample/sample?flag=B&rep_id=6520

At present, regulations for ATMPs is at its budding stage. Extensive research activities has been going on, which is resulting in Investigational New Drug (IND) applications.

The upcoming advanced therapy medicinal products market analysis report provides insight about the upcoming trends and restraining factors likely to shape growth of the market during forecast period (2019-2029). The report also provides a comprehensive analysis of the key companies of the market and offers details about the capacities and competencies of these companies. The market report also focusses on the markets competitive landscape and provides detail of the product portfolio of various companies.

Advanced Therapy Medicinal Products Market: Competitive Analysis

At present, the advanced therapy medicinal products market is growing at a lucrative rate. This growth rate is attributed to recent approval of various advanced therapy medicinal products. Post success of approved products, stakeholders are investing at enormously in clinical trials of advanced therapy medicinal products.

On the other hand, companies operating in the market are adopting various strategies to accelerate the product manufacturing rate. While most of the companies are relying on in-house production of therapies, few players such as Contract Manufacturing Organizations (CMOs) are opting for third-party service providers.

Upsurge in demand for gene therapy has resulted in widening of drug development landscape and rise in the number of new entrants. However, there is lack of production capabilities.

Also, several companies are strengthening their foothold in the global market by strategic alliances and acquiring small CAR T-cell therapy developers.

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Some of the key players operating in the advanced therapy medicinal products market are-

Advanced Therapy Medicinal Products Market: Key Trends

Despite high cost of the products, the market is expanding at lucrative rate. The growth rate is attributed to various health benefits provided by new classes of therapies.

Registering clinical benefits and efficiency of the products, stakeholders in the market are developing new strategies to overcome the challenged and boost application of advanced therapy medicinal products

Advanced Therapy Medicinal Products Market: Regional Outlook

Availability of significant number of FDA approved advanced therapy medicinal products in the U.S. has accounted for the prominent share of North America region, in terms of revenue. Recently, approval of products such as Yescarta, Zolgensma, and Kymriah has led to prominent investment in the U.S. advanced therapy medicinal products market.

Europe hold second-largest pharmaceutical market space across the globe. In coming years, cell therapy developers are anticipated to hold prominent share in the Europes drug revenue. Moreover, several academic institutes in Europe are conducting extensive research in early-stage cell therapy. This factor is likely to fuel the regional revenue contribution.

Meanwhile, global manufacturing companies operating in the market are enhancing their reach across Europe. This, in turn, may drive growth in the regional market.

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About TMR Research:

TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

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TMR Research,3739 Balboa St # 1097,San Francisco, CA 94121United StatesTel: +1-415-520-1050

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Advanced Therapy Medicinal Products Market Statistics, Segment, Trends and Forecast 2029 - Cole of Duty

XconomyTexas

Some former executives and investors behind the second gene therapy to win FDA approval are teaming up again. Their new company, Taysha Gene Therapies, is launching with $30 million in the bank and a plan to bring its first central nervous system (CNS) disorder program into human testing later this year.

Taysha is focusing on monogenic CNS diseases, which are caused by a single mutation in a single gene, says president and CEO RA Session II. The Dallas-based startup is developing treatments for rare genetic diseases as well as conditions that affect larger segments of the population, such as epilepsy and neurodegenerative disorders.

As long as its a monogenic CNS disease, were going to go after it, Session says.

Taysha has a pipeline of 15 gene therapies, and an option to add four more. The experimental treatments come from the University of Texas Southwestern (UTSW) Medical Center, which has a gene therapy program led by Steven Gray and Berge Minassian. Session, a former pharmaceutical industry executive who is currently an entrepreneur in residence at UTSW, says university administrators asked him to come up with ideas for funding the gene therapy research. Some entities were interested in taking on individual assets, but Sessions concluded that the best way to fund the entire portfolio was to create one company.

Rare diseases represent an unmet medical need, but even when the biology is clear a therapys commercial prospects can be murky. Drugs for chronic conditions can take several years to reach peak sales, but that peak then continues for years as more patients use a product, Session says. In rare diseases, this time to peak is typically achieved in the first year to 18 months of sales. Afterward, revenue falls because a rare disease by definition does not affect a big pool of potential new patients. Session says that in order to be financially sustainable, a drug company needs to constantly launch new products, and the UTSW assets give Taysha the opportunity to do just that.

It helps that Taysha is not starting from scratch. Session is a former business development executive for AveXis, a gene therapy developer that got its start in Dallas. Novartis (NYSE: NVS) acquired AveXis in 2018, and made the company its gene therapy division. Last year, AveXiss Zolgensma won FDA approval for spinal muscular atrophythe second gene therapy to receive the regulators nod.

Gene therapy uses an engineered virus to deliver a functioning gene that replaces a defective one. Tayshas gene therapies will be delivered by adeno-associated virus 9 (AAV9), a virus that can cross the blood-brain barrier. Its the same virus used for Zolgensma and Session says AveXiss work shows that the virus can be given to patients safely and effectively. Also, experience with the viral technology gives the company a leg up on knowing how to manufacture any therapies it produces quickly and at scale, he adds.

The seed financing announced Wednesday will fund Tayshas early work. PBM Capital, the first investor in AveXis, and Nolan Capital, the investment fund of former AveXis CEO Sean Nolan, co-led the round.

Tayshas lead program is TGTX-101, an experimental gene replacement therapy for GM2-gangliosidosis, a rare, inherited enzyme disorder that leads to the destruction of neurons in the brain and spinal cord. Session says this program is expected to begin clinical testing later this year. The company aims to ask the FDA for permission to start clinical trials for three additional gene therapies by the end of next year. Those programs include the mitochondrial disorder SURF1 deficiency; a form of epilepsy caused by a mutation to the SLC6A1 gene; and Rett syndrome, a neurological disorder caused by a genetic mutation.

In addition to advancing new gene therapies, Taysha is also developing new gene therapy technologies. Session says some of the research focuses on new capsids, the protein shells that enclose a virus. These next-generation capsids could potentially get a gene therapy to different cells in the body more efficiently and effectively, which could lead to therapies that require a lower dose, ultimately improving safety, he says.

Taysha is also developing technology for redosing a gene therapy. While these therapies are meant to be long-lasting, their durability over the course of a patients lifetime is still unknown, Session says. Redosing with the same therapy isnt possible because patients develop antibodies to the virus. Taysha is developing a way to redose via a new route of administrationthe vagus nerve. Session declined to elaborate, saying only that its early but its interesting. Its an issue the sector has been grasping at and trying to solve.

AveXis is still developing new gene therapies. A company executive told Xconomy earlier this year that an application to start human tests of a Rett syndrome therapy is expected to be ready by mid-2020. A number of companies on are on AveXiss heels, some of them focused in particular on the central nervous system and the brain. The CNS gene therapies at Passage Bio (NASDAQ: PASG) are from the University of Pennsylvania. New York-based Neurogene is also focused on rare monogenic neurological diseases. Prevail Therapeutics of New York and Voyager Therapeutics (NASDAQ: VYGR) are developing gene therapies for Parkinsons disease.

Taysha will develop its programs in partnership with UTSW. The university is handling discovery and preclinical research, as well as the studies leading up to an application to start clinical trials. UTSW will also handle manufacturing for clinical trials. Taysha will take the lead on clinical testing and regulatory work. If any of the gene therapies win regulatory approval, the company will take over manufacturing and handle commercialization.

Photo by Flickr user Katie Haugland Bowen via a Creative Commons license

Frank Vinluan is an Xconomy editor based in Research Triangle Park. You can reach him at fvinluan@xconomy.com.

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Taysha Launches With $30M to Bring Gene Therapy to CNS Disorders - Xconomy