Archive for the ‘Gene Therapy Research’ Category

A new type of cancer cell therapy could avoid some of the serious side effects commonly associated with CAR-T treatments, and possibly offer an easier path to developing "off-the-shelf" treatments, suggest findings from a small study led by researchers at the MD Anderson Cancer Center in Houston, Texas.

The results, which were published Wednesday in the New England Journal of Medicine, are from just 11 patients. Other factors, such as the use of postremission therapy, limit what conclusions can be drawn about the researchers' approach, which relies on "natural killer" cells rather than the T cells used in cellular drugs like Novartis' Kymriah.

Still, the data offer a glimpse into why Japanese drugmaker Takedaagreed last November to license the CAR NK cell therapy from MD Anderson, part of the company's broader push into cell and gene treatments. Some of the data published Wednesday was previously disclosed by the pharma.

The success of cancer immunotherapy, of which CAR-T treatments are a major part, has put T cells at the center of a now decade-long research revival in oncology.

But T cells are only one component of the body's immune system, and scientists in academia and in biotech are exploring whether other cellular defenders could be similarly recruited.

Researchers at MD Anderson have turned to natural killer cells, which by design recognize and attack cancers or other invaders. Such cells have been tested as an anti-cancer treatment before,but using genetic engineering to improve their tumor-killing properties, which the MD Anderson team has done, is a newer innovation.

"To my knowledge, this is the largest body of evidence on the use of CAR NK cells in patients with cancer," said Katayoun Rezvani, the study's corresponding author and a professor of stem cell transplantation and cellular therapy at MD Anderson, in an interview.

Using NK cells derived from cord blood, Rezvani and her colleagues engineered the cells to express a receptor for a protein called CD19, commonly found on the surface of B-cell malignancies like leukemia and lymphoma. They also added a gene for interleukin-15 to boost the expansion and persistence of the infused NK cells, which without engineering would typically disappear after about two weeks.

While the CAR-T treatments Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) also target CD19, they are made from a patient's own T cells, which are extracted and then engineered outside the body. The personalized process is time-consuming and laborious, hampering the commercial uptake of both Kymriahand Yescarta.

By using cord blood, Rezvani and her team are pursuing an allogeneic, or "off-the-shelf," approach to cell treatment something many consider to be the next step for the field.

Initial data look promising. Seven of the 11 treated patients, who had either chronic lymphocytic leukemia or non-Hodgkin lymphoma, responded to treatment, with the cancers of three going into remission.Most notably, none experienced cytokine release syndrome or neurotoxicity, two severe side effects that commonly occur in patients treated with CAR-T therapy.

"The lack of toxicity is very exciting here," wrote Stephan Grupp, an oncologist at Children's Hospital of Philadelphia and a leader in the CAR-T field, in comments emailed to BioPharma Dive. He was not involved with the MD Anderson study.

"We really think that this is something inherent to the biology of the natural killer cells, which means their profile of toxicity is different than that of T cells,"Rezvanisaid.

Study participants did have blood toxicities that researchers associated with the chemotherapy given prior to infusion of the CAR NK cells.

While positive, the results are limited by several factors which make drawing broader conclusions about the ultimate potential of the treatment difficult.

Five of the seven responding patients received postremission treatment, including stem cell transplants, Rituxan (rituximab) and Revlimid (lenalidomide), so researchers did not assess the duration of response to CAR NK therapy.

Additionally, a fresh CAR NK cell product was manufactured for each patient in this study, rather than using the cord blood to produce multiple therapies as would be envisioned for a true off-the-shelf product.

"I think the potential for this approach to be 'off-the-shelf' is also a little speculative at this time," wrote Grupp.

"We would need to see multiple patients treated from the same expanded product with no HLAmatching to know if 'off-the-shelf' is going to be part of the story here," he added, referring to the process by which patients are matched to donor cells.

If cord blood-derived CAR NK cells were able to be given without matching to a patient's HLA genotype, any resulting treatment could be used more widely. Nine patients were partially matched in the MD Anderson study, while the last two were treated without consideration of HLA type.

The MD Anderson researchers plan to continue enrolling patients in the study and are working with Takeda to design a larger, multi-center trial.

The drugmaker is planning to advance the treatment, which it licensed and now calls TAK-007, into pivotal studies in two types of lymphoma and CLL by 2021, with a potential filing for approval in 2023.

"Targeting CD19 was a proof of concept and now that we've demonstrated that this CAR NK approach can work and is safe we want to use this platform to target other types of cancers," said Rezvani, indicating interest in multiple myeloma and acute myeloid leukemia.

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In small study, hints of promise for 'natural killer' cell therapy - BioPharma Dive

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With advances in modern medicine accelerating forward, demand for lab space and health-care facilities has also seen a rapid growth. In Cushman & Wakefields latest report on the life sciences industry, the sector has demonstrated steady and strong market fundamentals.

With an aging and growing population, the life sciences sector is continuously working on new technologies to improve quality of life. But to keep advancing, companies are seeking additional lab space and more employees. Combined with a steady flow of research capital, the life sciences sectors market fundamentals have made it more resilient to the cyclical nature of the real estate market.

READ ALSO: The Magnetism of Medical Office Buildings

Currently, there are 52.4 million people aged 65 and older in the U.S and by the next decade, that number is expected to exceed 73 million. Similarly, the report noted that the median age in the U.S. increased to 38.2 in 2018 and is expected to reach 40 by 2030. As the population grows, so does demand for new treatments, drugs and equipment.

With such inevitable demand, technological advances in life science categories like genomics, gene editing and cell and gene therapy have taken off. Specifically, the cell and gene therapy sector commanded a $1.1 billion market cap in 2018, but is expected to grow by 36.5 percent in the next five years and hit an $11 billion market cap. Similarly, both the genomics and gene editing sectors will also grow by more than 10 percent each in the next five years.

As these sectors grow, so does demand for employees. Starting from the end of 2013, the amount of jobs in the biotech research and development industry has increased by 70,000 each year, or roughly 7.5 percent. In the life sciences sector overall, there were 204,800 jobs in the industry in 2019, with 67 percent of them concentrated in 11 metropolitan life sciences hubs.

Considering the growing need for space and employees, rents have increased, while vacancy rates have declined throughout major markets. Throughout the U.S., vacancy rates dropped to 7.1 percent for lab space versus the 12.4 percent rate for office space. With lower vacancy rates, rent for lab space in the 12 most popular life sciences markets has increased to $43.10 per square foot, compared to office spaces average rent of $40.30 per square foot.

Certain markets have also developed into strong life sciences hubs, with areas like Boston/Cambridge having close to no vacancy. The markets of Boston/Cambridge and the San Francisco Peninsula offer the largest markets, with 21.8 million of life sciences space, but others like New Jersey, have also risen to the top due to its history of being home to major pharmaceutical manufacturers. Markets like Boston/Cambridge, the San Francisco Peninsula and Oakland/East Bay all experienced an explosive growth in their talent pools in the last decade.

Earlier this year, Boston Properties partnered with Alexandria Real Estate Equities to combine their nearby properties and develop their first life sciences real estate development in a San Francisco biotech campus.

Despite the last two economic downturns, the life sciences sectors employment has increased 87.9 percent since 2000. While the industry is still subject to the ups and downs of the market, the report noted that the life sciences market is more likely to weather the storm than other sectors.

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Behind the Projected Growth of Life Sciences in 2020 - Multi-Housing News

Facts and Factors Market Researchhas published a new report titled Gene Therapy Market By Type (Germ Line Gene Therapy and Somatic Gene Therapy), By Vector Type (Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome), and By Therapy Area (Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 2018 2027.

According to the report, the globalgene therapy market was valued at approximately USD 919 million in 2018 and is expected to reach a value of around USD 6,892 million by 2027, at a CAGR of around 25.1% between 2019 and 2027.

Gene therapy is the kind of experimental method that makes use of genes for treating or preventing disease by inserting foreign genetic material like DNA or RNA into the persons cells. Scientists are studying gene therapy for treating various kinds of immuno-deficiencies, Parkinsons disease, HIV, and cancer by using myriad approaches. Today, many of the approaches to gene therapy are undergoing most intensive & rigorously testing. This includes replacing the mutated gene causing disease with the healthy gene copy. Another approach includes knocking out or inactivating a mutated gene operating improperly. Yet another approach includes a new gene into the body to combat the disease.

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New product approval & commercialization to drive the market trends

Between the periods from 2012 to 2018, nearly five single-use gene treatments received approval from the U.S. FDA for treating a rare form of genetic disorders. Moreover, gene treatments that have received approval are being tested by pharmaceutical firms in the market. Apart from this, current approvals of gene therapy products across the U.S., as well as European countries for treating a plethora of life-threatening diseases, are anticipated to steer the growth of gene therapy industry over the forecast timeline. Moreover, gene therapy can also be used for treating neurodegenerative disorders like Alzheimer, amyotrophic lateral sclerosis, and spinal muscular atrophy.

Furthermore, many of the reputed pharma firms like Bristol-Myers Squibb, BioMarin, and Pfizer are investing massively into the research activities pertaining to gene therapy. Apart from this, a rise in the occurrence of cancer is prompting the demand to treat the disease. Gene therapy is one of the key treatment kinds that will propel the market growth over the forecast period. However, inadequate reimbursement policies pertaining to the one-time gene treatments will downgrade market expansion.

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In addition to this, conducting of randomized controlled trials can pose a threat to the expansion of the gene therapy industry as a result of the gene therapy features & projected patient population. Nevertheless, the ability of the gene therapy to eliminate the number of ailments with faulty or missing genes like hemophilia A will promote the market growth over the forecast period and thereby nullify the negative impact of hindrances on the business growth.

Somatic gene therapy to dominate the type segment

The growth of the segment over the forecast timeline is credited to the ability to treat the targeted cells in the patient population. The treatment is not passed to future generations and is restricted to only the patient who receives the somatic gene therapy. Moreover, it is used for treating a huge number of disorders like cystic fibrosis, cancer, and muscular dystrophy.

Cancer to lead the therapy area segment over the forecast period

The segmental expansion is attributed to a large number of pipeline drugs registered over the past few years along with increasing occurrence of cancer as a result of genetic changes.

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North America to dominate the overall regional market share during the forecast timespan

North American market, which accrued revenue of USD 380 million in 2018, is set to contribute majorly towards the overall market revenue by 2027. The regional market surge is credited to robust healthcare amenities, high per capita healthcare spending, and improvement in the reimbursement policies.

The key players included in this market are Advanced Cell & Gene Therapy, Audentes Therapeutics, Benitec Biopharma, Biogen, Blubird Bio, Inc., Bristol-Myers Squibb Company, CHIESI Farmaceutici SPA, Eurofins Scientific, Geneta Science, Genzyme Corporation, Gilead, GlaxoSmithKline PLC, Human Stem Cells institute, Novartis AG, Orchard Therapeutics, Pfizer Inc., Sangamo therapeutics, Spark therapeutics, and Voyager Therapeutics.

Browse the fullGene Therapy Market By Type (Germ Line Gene Therapy and Somatic Gene Therapy), By Vector Type (Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome), and By Therapy Area (Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 2018 2027Report athttps://www.fnfresearch.com/gene-therapy-market-by-type-germ-line-gene

This report segments the gene therapy market as follows:

Global Gene Therapy Market: By Type Segment Analysis

Global Gene Therapy Market: By Vector Type Segment Analysis

Global Gene Therapy Market: By Therapy Area Segment Analysis

Global Gene Therapy Market: Regional Segment Analysis

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Global Gene Therapy Market Set to Grow USD 6892 Million By 2027 - TheInfobiz

In its recently added report by UpMarketResearch.com has provided unique insights about Gene Therapy Industry Market for the given period. One of the main objectives of this report is to categorize the various dynamics of the market and to offer latest updates such as mergers and acquisitions, various technological developments, new entrants in the market, which make an impact on different segments.

This Gene Therapy Industry Market report is based on synthesis, analysis, and interpretation of information gathered regarding the target market from various sources. Our analysts have analyzed the information and data and gained insights using a mix of primary and secondary research efforts with the primary objective to provide a holistic view of the market. In addition, an in-house study has been made of the global economic conditions and other economic indicators and factors to assess their respective impact on the market historically, as well as the current impact in order to make informed forecasts about the scenarios in future.

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The Gene Therapy Industry Market report is a trove of information pertaining to the various aspects of this industry space. Encompassing the ongoing as well as forecast trends likely to fuel the business graph of the Gene Therapy Industry Market across various geographies, the report also provides details about the driving factors that would help propel this industry to new heights during the projected period. Alongside a collection of the driving parameters, the Gene Therapy Industry Market reports also include a spate of other dynamics pertaining to the industry, such as the nominal risks prevailing in this marketplace as well as the growth prospects that this business sphere has in the future.

Some of key competitors or manufacturers included in this report are:company 1company 2company 3company 4company 5company 6company 7company 8company 9

Gene Therapy Industry Market Drivers & Challenges:The report covers the major driving factors influencing the revenue scale of the market and details about the surging demand for the product from the key geological regions.The latest trends and challenges that prominent industry contenders could face are highlighted in the report.

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The significant applications and potential business areas are also added to this report.The technological advancements, value and volume governing factors are explained in detail. The pricing structures, raw material analysis, market concentration scenario are analysed. In-depth information on upstream raw materials sourcing, downstream buyers, raw materials cost, labour cost and industry chain view is presented.The report uses tools such as comparison tables, graphs, pie charts, progress charts, etc. to give a clear picture of the market growth. Additionally, an overview of each market segments such as product type, application, end users, and region are offered in the report.

Market Segmentation By Type: Type 1Type 2Type 3

Market Segmentation By Applications: Application 1Application 2Application 3

The Regions covered are:Asia-PacificNorth AmericaEuropeSouth AmericaMiddle East & Africa

To provide the clarified representation of the current and upcoming growth trends of the market, the report provides the execution and attributes of the Gene Therapy Industry Market that are analyzed on the basis of the qualitative and quantitative process. Through the report, one can be able to take quick and precise business decisions by getting familiar with every aspect of the market. The Gene Therapy Industry Market report represents the analyzed data through graphs, charts, and figures for less complexity and better understandability about the Gene Therapy Industry Market.

To conclude, the Gene Therapy Industry Market report will provide the clients with a high-yielding market analysis assisting them to understand the market status and come up with new market avenues to capture hold of the market share.

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Table Of Contents:Chapter 1 Market OverviewChapter 2 Industry ChainChapter 3 Environmental AnalysisChapter 4 Market Segmentation by TypeChapter 5 Market Segmentation by ApplicationChapter 6 Market Segmentation by RegionChapter 7 Market CompetitiveChapter 8 Major VendorsChapter 9 Conclusion

To purchase this report, Visit: https://www.upmarketresearch.com/buy/gene-therapy-industry-market-research-report-2019

About UpMarketResearch:Up Market Research (https://www.upmarketresearch.com) is a leading distributor of market research report with more than 800+ global clients. As a market research company, we take pride in equipping our clients with insights and data that holds the power to truly make a difference to their business. Our mission is singular and well-defined we want to help our clients envisage their business environment so that they are able to make informed, strategic and therefore successful decisions for themselves.

Contact Info UpMarketResearchName Alex MathewsEmail Alex@UpMarketResearch.comOrganization UpMarketResearchAddress 500 East E Street, Ontario, CA 91764, United States.

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Global Gene Therapy Industry Market 2019 Trends, Market Share, Industry Size, Opportunities, Analysis and Forecast To 2025 - Chronicle 99

Facts and Factors Market Research has published a new report titled Plasmid Market By General Type (Conjugative and Non-Conjugative), by Specific Plasmid Types (F-Plasmids, Col Plasmids, Resistance Plasmids, Cryptic Plasmids, Degradative Plasmids, and Virulence Plasmids), and by Application (Transfection, Recombinant DNA Technology, Gene Therapy, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 2019 2027.

According to the report, the global plasmid market is anticipated to be valued approximately USD 89 million in 2018 and is projected to hit the revenue of around USD 447 million by 2027, at a CAGR of around 19.5% between 2019 and 2027.

Plasmids are small circular double-stranded DNA molecules having the ability to duplicate independently. Moreover, the plasmid is different from chromosomal DNA and do not depend on chromosomal DNA for duplication. Plasmids are also referred to as replicons and they are the units of DNA that are capable of replicating independently within a suitable host.

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Plasmids are primarily found in bacteria. However, they can also be detected in archaea and multicellular organisms. Plasmids generally carry minimum one gene. Most of the genes that plasmids carry benefit to host organisms.

Growing occurrence of cancer, genetic disorders, & contagious ailments to steer the market growth

Vectors are utilized in developing cancer treatments and this is likely to steer the growth of the plasmid market over the forecast timeline. In addition to this, continuous ongoing research targeting cancer and approvals of many of the biopharmaceutical medicines for treating cancer will impel the growth of the plasmid market over the forecast timeframe. Apart from this, the growing occurrence of chronic as well as contagious diseases producing escalating demand for improved therapies will proliferate the market scope over the forecast timespan.

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Moreover, escalating awareness about the gene treatment along with the acceptance of gene therapy for treating ailments like Alzheimer and attention deficit hyperactivity will amplify the market growth over the forecast timeline. Apparently, gene therapy has a high potential for treating genetic disorders, as well as chronic ailments like cancer, will further enlarge the business growth over the forecast timespan. Nevertheless, mutagenesis coupled with huge costs related to gene therapy can act as a restraining factor for the plasmid industry over the forecast timeframe.

F-plasmids to dominate the specific plasmid types segment

F-plasmids segment is predicted to accrue a revenue of nearly USD 124 million by 2027. Some of the plasmids like F-plasmids possess the genes referred to as transfer genes enabling conjugation and this is likely to drive the expansion of the segment over the forecast timeline.

Gene therapy segment to contribute majorly towards the application landscape

The growth of the gene therapy segment is credited to a rise in the investment in research activities pertaining to gene treatment. Apart from this, increase in the awareness about the gene therapy, government support, and adoption of gene therapy for treating cancer will amplify the expansion of the gene therapy segment over the period from 2019 to 2027.

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North America to dominate the overall regional market revenue share

North American market is likely to accrue a revenue of approximately USD 160 million by 2027. The growth of the market in the region is attributed to rising in the healthcare spending, approval of gene treatments for various chronic ailments, and rise in the investments in research activities pertaining to gene therapy applications in various domains.

Key players involved in the plasmid industry include Aldevron, GenScript, PlasmidFactory GmbH & Co. KG, VGXI, Inc., Oxford Genetics Ltd., Applied StemCell, Altogen Biosystems, Cobra Biologics, Copernicus Therapeutics, Inc., Takara Bio Inc., InvivoGen, Miltenyi Biotec, Medigene Sdn Bhd, MaxCyte, Inc., Mirus Bio LLC, MolMed S.p.A., GenePharma, and Polyplus Transfection.

Browse the fullPlasmid Market By General Type (Conjugative and Non-Conjugative), by Specific Plasmid Types (F-Plasmids, Col Plasmids, Resistance Plasmids, Cryptic Plasmids, Degradative Plasmids, and Virulence Plasmids), and by Application (Transfection, Recombinant DNA Technology, Gene Therapy, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 2019 2027Report athttps://www.fnfresearch.com/plasmid-market-by-general-type-conjugative-and-non

This report segments the plasmid market as follows:

Global Plasmid Market: By General Type Segment Analysis

Global Plasmid Market: By Specific Plasmid Types Segment Analysis

Global Plasmid Market: By Application Segment Analysis

Global Plasmid Market: Regional Segment Analysis

About Us:

Facts & Factors is a leading market research organization offering industry expertise and scrupulous consulting services to clients for their business development. The reports and services offered by Facts and Factors are used by prestigious academic institutions, start-ups, and companies globally to measure and understand the changing international and regional business backgrounds. Our clients/customers conviction on our solutions and services has pushed us in delivering always the best. Our advanced research solutions have helped them in appropriate decision-making and guidance for strategies to expand their business.

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Global Headquarters

Level 8, International Finance Center, Tower 2,8 Century Avenue, Shanghai,Postal 200120, ChinaTel: +86 21 80360450

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Global Plasmid Market Will Reach USD 447 Million By 2027 - TheInfobiz

The Global Gene Therapy Market Is Expected To Reach Around USD 2,269 Million By 2024 Research Report provides the newest industry data and industry future trends, allowing you to identify the products and end users driving Revenue growth and profitability. A leading market research firm,Zion Market Researchadded industry report onGene Therapy Marketconsisting of 110+ pages with TOC (Table of Contents) including a list of tables & figuresduring the forecast period and Gene Therapy Market report offers a comprehensive research updates and information related to market growth, demand, opportunities in the global Gene Therapy Market.

FREE | Request Sample Report of Gene Therapy Market Report @https://www.zionmarketresearch.com/sample/gene-therapy-market

Our Free Complimentary Sample Report Accommodate a Brief Introduction of the research report, TOC, List of Tables and Figures, Competitive Landscape and Geographic Segmentation, Innovation and Future Developments Based on Research Methodology

The global Gene Therapy Market report offers an extensive analysis of the realistic data collected from the global Gene Therapy Market. It demonstrates major drifts and key drivers playing an important role in the growth of the global Gene Therapy Market during the foretold time. The report focuses on the analysis of the key features such as drivers, new development opportunities, and restraints influencing the expansion of the Gene Therapy Market for the forecasted period.

The report covers a detailed analysis of the development of the Gene Therapy Market for the upcoming time. The global Gene Therapy Market is segmented based on the various product categories, delivery channels, and applications.

Major Market Players Included in This Report:

UniQure N.V, Spark Therapeutics LLC, Bluebird Bio, Juno Therapeutics, GlaxoSmithKline, Celgene Corporation, Shire Plc, Sangamo Biosciences, Dimension Therapeutics

A complete value chain of the global Gene Therapy Market is emphasized in the global Gene Therapy Market report along with the review of the downstream and upstream components influencing the global Gene Therapy Market. It analyzes the expansion of every segment of the Gene Therapy Market. The data presented in the research report is collected from various industry organizations to estimate the development of each segment of the global Gene Therapy Market in the coming period.

The global Gene Therapy Market research report presents market dynamics and inclinations influencing the growth of the global Gene Therapy Market. It uses SWOT analysis to review the competitive players of the Gene Therapy Market. Furthermore, the report also includes a synopsis of the various business strategies of the key players of the Gene Therapy Market.

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Promising Regions & Countries Mentioned In The Gene Therapy Market Report:

The report focuses on the latest market trends and major growth opportunities assisting in the expansion of the global Gene Therapy Market. On the basis of topography, the global Gene Therapy Market is classified into Europe, North America, Latin America, Middle East & Africa, and the Asia Pacific.

The Gene Therapy Market report provides company market size, share analysis in order to give a broader overview of the key players in the market. Additionally, the report also includes key strategic developments of the market including acquisitions & mergers, new product launch, agreements, partnerships, collaborations & joint ventures, research & development, product and regional expansion of major participants involved in the market on the global and regional basis.

Browse Press Release:https://www.zionmarketresearch.com/news/gene-therapy-market

Following 15 Chapters represents the Gene Therapy Market globally:

Chapter 1,enlist the goal of global Gene Therapy Market covering the market introduction, product image, market summary, development scope, Gene Therapy Market presence;

Chapter 2,studies the key global Gene Therapy Market competitors, their sales volume, market profits and price of Gene Therapy Market in 2018 and 2026;

Chapter 3,shows the competitive landscape view of global Gene Therapy Market on the basis of dominant market players and their share in the market growth in 2018 and 2026;

Chapter 4,conducts the region-wise study of the global Gene Therapy Market based on the sales ratio in each region, and market share from 2018 to 2026;

Chapter 5,6,7,8 and 9demonstrates the key countries present in these regions which have revenue share in Gene Therapy Market;

Chapter 10 and 11describes the market based on Gene Therapy Market product category, a wide range of applications, growth based on market trend, type and application from 2018 to 2026;

Chapter 12shows the global Gene Therapy Market plans during the forecast period from 2018 to 2026 separated by regions, type, and product application.

Chapter 13, 14, 15mentions the global Gene Therapy Market sales channels, market vendors, dealers, market information and study conclusions, appendix and data sources.

Inquire more about this report @https://www.zionmarketresearch.com/inquiry/gene-therapy-market

Available Array of Customizations:

Country-level bifurcation of data in terms of Product Type (Concentration, Temperature, Combustion, Conductivity, and Others) and Application (Petrochemical, Metallurgy, Electricity, and Others) for any specific country/countries.

Expansion of scope and data forecasts until 2030

Company Market Share for specific country/countries and regions

Customized Report Framework for Go-To Market Strategy

Customized Report Framework for Merger & Acquisitions and Partnerships/JVs Feasibility

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Global Gene Therapy Market Hits Record Value of USD 2269 Million By 2024 - Global Newspaper 24

Thought leaders in cancer cell and gene therapy research will share the latest advances, address the greatest challenges and showcase the most innovative programs in progress today.

Alliance for Cancer Gene Therapy (ACGT) will host its inaugural ACGT 2020 Cancer Summit on April 16, 2020 at the Alexandria Center for Life Science in New York City. The ACGT 2020 Cancer Summit which launches ACGTs 20th anniversary will bring together researchers, companies, investors and advocates in cancer cell and gene therapy to discuss the latest advances, with a focus on combating solid tumors.

A partial list of ACGT 2020 Cancer Summit speakers includes:

Presenting sponsors include Alexandria Real Estate Equities, Inc./Alexandria Venture Investment. Additionally, STAT will be a media partner for the ACGT 2020 Cancer Summit. For more sponsorship information and early bird registration, please visit https://summit2020.acgtfoundation.org

Media Registration

Media registration is free of charge for all valid press card holders or via provision of formal journalist credentials. Register early by contacting ACGT.pr@HDMZ.com to begin receiving advance meeting materials, media alerts, and access to meeting presenters.

About Alliance for Cancer Gene Therapy

For nearly 20 years, Alliance for Cancer Gene Therapy (ACGT) has funded research that is bringing innovative treatment options to people living with deadly cancers treatments that save lives and offer new hope to all cancer patients. Founded by Barbara and Edward Netter after their daughter-in-laws death from breast cancer, ACGT funds researchers who are pioneering the potential of cancer cell and gene therapy talented visionaries whose scientific advancements are driving the development of groundbreaking treatments for ovarian, prostate, sarcoma, glioblastoma, melanoma and pancreatic cancers. One hundred percent of all public funds raised by ACGT directly support program and research, thanks to separate funding to support administrative expenses.

For more information, visit acgtfoundation.org, call 203-358-5055, or join the ACGT community on Facebook, Twitter, Instagram and YouTube.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200203005084/en/

Contacts

Media Inquiries: Emily Maxwell 312-506-5220emily.maxwell@hdmz.com

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Save the Date: Alliance for Cancer Gene Therapy 2020 Cancer Summit to be Held in New York City - Yahoo Finance

The cell and gene therapy sector is booming. At the end of 2019, there were 1,069 registered clinical trials in the field. By 2024, the cell and gene therapy market is estimated to reach revenues of $6.6B (5.9B). But there is a problem. As a result of the rapid growth of gene and cell therapies, the demand for plasmids is skyrocketing. And the industry is struggling to meet it.

Plasmids are the key building blocks needed to manufacture viral vectors, which are the most common strategy to develop gene therapies, explains Stefano Baila, Director of Operations and Business Development at Anemocyte. Plasmids are not only crucial for the development of gene therapies, but also for therapies that involve the genetic modification of cells, such as CAR-T. This has greatly increased the demand for plasmids and I would say that the industry was probably not ready to address this demand.

While the industry is struggling with the lack of plasmids on the market, another challenge is quality. Those providers that can meet the high-quality requirements for plasmids have long waiting lists of up to 12 months, says Baila. For companies moving into phase 3 or commercial production, it becomes crucial that the good manufacturing practice (GMP) requirements are met.

But another result of the rapidly evolving gene and cell therapy space is the fact that the regulatory requirements are not quite up to scratch. The regulatory framework around plasmid production is very confusing for the industry at the moment, says Baila. The main guidelines refer to the quality of the product, but the level of quality remains open to interpretation. More clarity would definitely help once and for all to define the exact quality levels required at different stages of drug development.

Consequently, plasmid providers have to be able to address all quality levels required at each stage of drug development. As the first biotech manufacturing organization (BMO) worldwide, Italian company Anemocyte has met this challenge by focusing greatly on the industrys needs. Their keyword is flexibility.

Before starting their work on plasmid manufacturing, the team spent several months interviewing companies about their difficulties and needs regarding the bottleneck in plasmid production and regulatory issues.

For us, it was key to understand the needs of the industry and find a possible solution, Baila explains. Our research resulted in a brand new facility, which is designed with adaptable manufacturing spaces that enable flexible time management. This ensures that the manufacturing process continues to roll without creating a bottleneck.

As a next step, the Anemocyte team had to decide whether to use a classified cleanroom or just a regular lab for the manufacturing process. We decided to keep the bar pretty high, so we are working with a cleanroom facility where we apply the GMP standard, says Baila. We maintain a high quality and also address the time issues that all companies seem to share as their main challenge in cell and gene therapy development.

The flexibility of its manufacturing facility allows the Anemocyte team to easily adapt to its customers needs. Our customers have control over what we do, explains Marco Ferrari, CEO of Anemocyte. They have the opportunity to be involved in the process, and decisions and actions are discussed and shared with them to ensure their product is produced at the high standard they expect.

Moreover, Anemocyte pays attention to new technologies and innovations. The fast evolution of the cell and gene therapy industry greatly increases the demand for new solutions, Ferrari explains. Staying on top of innovation is therefore mandatory today. Our approach is to stay ahead of the trends and be capable of deploying useful solutions for our customers.

Anemocytes manufacturing facility is built in such a modular way that it can be replicated and adapted to meet the rising demand for plasmids. This, as Ferrari puts it, ensures that the Anemocyte team will not be caught off guard when more companies come knocking at our door. This is an advantage for long-term customers because the manufacturing facility can be duplicated and built according to the customers needs.

As the worlds first BMO, Anemocyte pays specific attention to innovation and the ongoing trends in the industry. One of the emerging trends is the use of nonviral vectors for the development of gene therapies.

Even in the nonviral approach, plasmids play a key role, explains Baila. A part of the nonviral strategy is the transfer of plasmids into cells via mechanical or chemical methods. So, in one way or another, plasmids will always be needed.

Our investment in the nonviral gene modification space is an example of how we are tackling potential future trends that are still under the radar or explored at an academic level, adds Ferrari.

Dont sit on a waiting list to get your plasmids produced! Get in touch with the team at Anemocyte or learn more about the company and the development of plasmids for cell and gene therapies here!

Images via Shutterstock.com

Author: Larissa Warneck, Science Journalist at Labiotech.eu

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How Can We Tackle the Bottleneck in Plasmid Production for Gene... - Labiotech.eu

$250,000 in Vilcek Foundation Prizes awarded to Xiaowei Zhuang, Kivan Birsoy, Viviana Gradinaru, and Martin Jonikas.

NEW YORK, Feb. 3, 2020 /PRNewswire/ -- The Vilcek Foundation is pleased to announce the winners of the 2020 Vilcek Prizes in Biomedical Science. Awarded annually, the Vilcek Prizes in Biomedical Science honor the contributions of immigrants to scientific research, discovery, and innovation in the United States. The Vilcek Prizes in Biomedical Science are a part of the Vilcek Foundation's prizes program, which honors the contributions of foreign-born persons in the arts, sciences, and humanities in the United States.

The Vilcek Prize in Biomedical Science recognizes an immigrant scientist for outstanding career contributions to biomedical science and global scientific research. The prize comprises an unrestricted cash prize of $100,000 and a commemorative award. The winner of the 2020 Vilcek Prize in Biomedical Science is Xiaowei Zhuang, the David B. Arnold Jr. Professor of Science at Harvard University and a Howard Hughes Medical Institute Investigator.

"Honoring the achievements of immigrant scientists and to articulate their enormous role in upholding the United States' world leadership in science is especially important at this juncture in view of the current administration's efforts to curtail immigration to this country," said Jan Vilcek, cofounder and chairman of the Vilcek Foundation. "Xiaowei Zhuang exemplifies a foreign-born scientist whose work has demonstrably shaped the further advancement of scientific technologies and discovery in the United States. The imaging tools she has developed have changed how we see and understand the structures and basic functions of living organisms at a molecular level, fundamentally expanding research horizons and possibilities, and cementing the United States as a global leader in science."

Xiaowei Zhuang developed methods to directly observe individual molecules in cells at high resolution and at genomic scale. She invented STORM, a super-resolution imaging method that surpasses the diffraction limit of conventional light microscopy and enables light microscopy at molecular-scale resolution. Using STORM, Zhuang has unveiled novel structures in cells, such as a periodic membrane skeleton in neurons, which provides a platform for signal transduction and lends mechanical stability. Zhuang invented a single-cell transcriptome and genome imaging method, MERFISH, which allows thousands of genes to be simultaneously imaged and quantified in individual cells. MERFISH is a key tool in the Human Cell Atlas projectan international effort aimed at cataloguing cell types that constitute humans. Zhuang has used this tool to identify novel types of neurons in the mouse brain and uncovered insights into the neuronal control of behavior. Zhuang was born in Rugao, China.

The Vilcek Prizes for Creative Promise in Biomedical Science are awarded to young immigrant scientists whose early career work represents a profound advance in their respective fields. Each prizewinner receives a $50,000 cash prize and a commemorative award. Kivan Birsoy, Viviana Gradinaru, and Martin Jonikas are the winners of the 2020 Vilcek Prizes for Creative Promise in Biomedical Science.

Kivan Birsoy has fashioned tools to probe the complex metabolism of cancer cells. Using CRISPR, Birsoy demonstrated how mitochondriathe powerhouses of animal cellssupport tumor growth. He found that the synthesis of the amino acid aspartate rather than energy generation is key to mitochondria's ability to fuel tumors. He custom-designed instruments called Nutrostats for the long-term laboratory culture of cancer cells under nutrient levels closely mimicking natural conditions. Combining the Nutrostats with DNA barcoding and CRISPR-based genetic screens, Birsoy showed that a rare but deadly type of lymphoma requires LDL-cholesterol for survival. His work has identified altered tumor metabolism as a potential therapeutic target in cancer. Birsoy was born in Izmir, Turkey.

Viviana Gradinaru has developed advanced tools in optogeneticsan approach that uses light to control brain cellsto unearth deep insights into the brain. Her work has uncovered circuits that may underpin sleep disturbances in neuropsychiatric disorders. She has also engineered vehicles for gene therapy that, when injected into the bloodstream of rodents, cross the blood-brain barrier and deliver target genes to the brain. Gradinaru also developed a tissue-clearing technique called PARS-CLARITY to render the entire body of a rodent transparent for fine-scale visualization and mapping of circuits as well as gene expression analysis. Her work could boost the development of gene therapies for an array of human disorders. Gradinaru was born in Vaslui, Romania.

Martin Jonikas has used molecular biology to advance the single-celled green alga Chlamydomonas as a widely used model for photosynthesisa biochemical process at the heart of global food production. Jonikas and colleagues have unraveled the molecular makeup, structure, and assembly of pyrenoids, which are cellular compartments found in photosynthetic organisms that can absorb planet-warming carbon dioxide. This work could aid efforts to engineer pyrenoids into species that lack them, paving the way toward environment-friendly food production and helping to combat climate change. Jonikas was born in Paris, France.

The foundation has also announced the recipients of the 2020 Vilcek Foundation Prizes in Literature, and the recipient of the 2020 Vilcek Prize for Excellence in the Administration of Justice. All of the prizewinners will be celebrated at the Vilcek Foundation Prizes Gala in New York in April 2020.

The Vilcek Foundation

The Vilcek Foundation was established in 2000 by Jan and Marica Vilcek, immigrants from the former Czechoslovakia. The mission of the foundation, to honor immigrant contributions to the United States, and more broadly, to foster appreciation of the arts and sciences, was inspired by the couple's respective careers in biomedical science and art history. The foundation awards annual prizes to immigrant biomedical scientists and artists, sponsors cultural programs, and manages the Vilcek Foundation Art Collections. To learn more, please visit vilcek.org.

Contact:Elizabeth BoylanThe Vilcek Foundation212-472-2500 or elizabeth.boylan@vilcek.org

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Vilcek Prizes Recognize Immigrant Scientists for the Development of Tools and Technologies Advancing Scientific Discovery - BioSpace

Somerset Feb 3, 2020 (Thomson StreetEvents) -- Edited Transcript of Catalent Inc earnings conference call or presentation Monday, February 3, 2020 at 1:15:00pm GMT

* John R. Chiminski

Catalent, Inc. - Chairman & CEO

* Wetteny N. Joseph

Catalent, Inc. - Senior VP & CFO

UBS Investment Bank, Research Division - Senior Equity Research Analyst of Healthcare Life Sciences

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

* Jacob K. Johnson

William Blair & Company L.L.C., Research Division - Partner & Healthcare Services Analyst

* Tycho W. Peterson

Ladies and gentlemen, thank you for standing by, and welcome to the Catalent Second Quarter Fiscal Year 2020 Earnings Conference Call. (Operator Instructions) Please be advised that today's conference is being recorded. (Operator Instructions)

I would now like to hand the conference over to your speaker today, Paul Surdez, Vice President, Investor Relations. Thank you. Please go ahead, sir.

Good morning, everyone, and thank you for joining us today to review Catalent's Second Quarter Fiscal Year 2020 Financial Results. With me today are John Chiminski, Chair and Chief Executive Officer; and Wetteny Joseph, Senior Vice President and Chief Financial Officer.

In addition to reviewing our second quarter earnings release issued earlier this morning, we will also refer you to our other press release issued today regarding our agreement to acquire cell therapy leader, MaSTherCell. Please see our agenda for this call on Slide 2 of our supplemental presentation, which is available on our Investor Relations website at http://www.catalent.com.

During our call today, management will make forward-looking statements and refer to non-GAAP financial measures. It is possible that actual results could differ from management's expectations. We refer you to Slide 3 for more detail. Slides 3, 4 and 5 discuss the non-GAAP measures, and our just-issued earnings release provides reconciliations to the nearest GAAP measures. Catalent's Form 10-Q, to be filed with the SEC later today, has additional information on the risks and uncertainties that may bear on our operating results, performance and financial condition.

Now I would like to turn the call over to John Chiminski.

John R. Chiminski, Catalent, Inc. - Chairman & CEO [3]

Thanks, Paul, and welcome, everyone, to the call. In addition to reporting strong Q2 results, we're excited to announce this morning our plan to further expand our biologics footprint by acquiring MaSTherCell, a leader in cell therapy development and manufacturing.

Before reviewing the strategy behind adding MaSTherCell to the Catalent family, let me summarize our financial highlights from the second quarter. As you can see on Slide 6, our revenue for the second quarter increased 16% as reported or 17% in constant currency to $721 million, with 7% of the constant currency growth being organic, which is above our expectations for the long-term organic growth of our base business.

Our adjusted EBITDA of $171 million for the quarter was above the second quarter of fiscal year 2019 on a constant currency basis by 16%, with 5% being organic. Our adjusted net income for the second quarter was $72 million or $0.45 per diluted share, unchanged from the per share adjusted net income in the prior fiscal year.

Three of our 4 reporting segments had strong performances as Biologics, Softgel and Oral Technologies and Clinical Supply Services each contributed to the organic revenue and adjusted EBITDA growth, partly offset by headwinds in our Oral and Specialty Delivery segment. Wetteny will detail these results later in the call.

Now moving on to the operational update. First, we announced 2 important executive appointments in January that provide additional depth and breadth to our leadership team. We recruited Karen Flynn to return to Catalent after 10 years of leading operations and commercial activity for a well-respected biopharma services company to be President of our Biologics segment and our Chief Commercial Officer. Karen, who is replacing the retiring Barry Littlejohns, will execute our biologics strategy and further expand our Biologics drug substance, drug product and gene therapy businesses. We also recruited another former Catalent executive with decades of experience in the biopharmaceutical industry, Ricci Whitlow, as our President of Clinical Supply Services in place of the retiring Paul Hegwood. In addition to growing our CSS business with our traditional customer base, she will be focused on growing its footprint through cross-selling opportunities with our Biologics and other long-cycle businesses. Karen and Ricci, like Barry and Paul before them, report to our COO, Alessandro Maselli. They replace distinguished leaders who are celebrated here at Catalent for growing their businesses and for their tireless efforts to help establish our patient-first culture.

Next, last week, the Catalent Board of Directors approved the deployment of additional capital for further expansion of our gene therapy commercial facilities at BWI, which expansion will support operations on the BWI campus as well as our other gene therapy facilities in BioPark, Rockville and Gaithersburg. This investment is above and beyond the CapEx previously approved for the build-out of the 10 suites in our BWI facility, all of which are on track to be operational at the end of this calendar year. The additional CapEx approved last week will allow us to achieve higher revenue potential from the Paragon acquisition than anticipated at the time of the original acquisition last May once all the projects are completed.

Additionally, early last month, we took ownership of Bristol-Myers Squibb's oral solid biologics and sterile product manufacturing and packaging facility in Anagni, Italy, which we had agreed to acquire in June. This multipurpose site enhances our global network and provides us drug product sterile fill/finish capacity and oral solid-dose manufacturing in Europe and comes with an agreement to continue to manufacture BMS's current product portfolio at the site. The Anagni facility expands our European capabilities in biologics drug product, solid oral dose manufacturing and packaging to accelerate development programs and provides greater commercial supply capacity.

The acquisition of Anagni is another example of our progress in realizing our global biologics strategy, which continues to develop and strengthen across our network. As an additional example, I'm pleased to announce that the Bloomington site received yet another commercial product approval in January, bringing its total to 22 versus the 12 it was producing at the time of the acquisition, with several additional launches on the horizon. The previously announced $200 million investment in Bloomington and Madison are progressing according to plan and will help us serve the existing pipeline of late-stage clinical work and other opportunities for these high-margin sites.

Another important element of our biologics strategy is our entrance into the gene therapy space last year. The acquisitions of Paragon Bioservices and related gene therapy assets provided Catalent with new expertise and capabilities in one of the fastest-growing techniques for therapeutic intervention today and position us for accelerated long-term growth. The integration of these gene therapy assets into the Catalent portfolio is progressing ahead of our expectation and has been a key contributor to our strong year-to-date financial results. The CapEx approval I previously highlighted was supported by this early outperformance as well as by research remission from an independent third-party consultant, which indicates the gene therapy pipeline will continue to increase much more rapidly than the manufacturing assets needed to service the demand.

Paragon provided us with a platform for development of an expanded offering in biologics, enabling entry into technology categories adjacent to the development and production of viral vectors for gene therapies. The success we've experienced thus far with Paragon provides us with the confidence and blueprint to further expand our biologics offering into cell therapy, which we are announcing this morning.

Please turn to Slide 7 for an overview of our agreement to acquire MaSTherCell, a technology-focused cell therapy development and manufacturing partner to cell therapy innovators. MaSTherCell's service offerings include the development and manufacture of both autologous and allogeneic cell therapies as well as a variety of related analytical services. It has worked with a range of therapies, including those based on the so-called CAR-T cells, tumor-infiltrating lymphocytes as well as T cell receptors and other cell types.

MaSTherCell, which was founded in 2011, has sites in Belgium and Texas. Its current operating facility near Brussels provides preclinical and clinical stage services, and MaSTherCell is in the process of building a commercial-scale production and fill/finish facility nearby, which is expected to open in late 2021. MaSTherCell is also in the final stages of completing the build-out of a preclinical and clinical stage facility near Houston, Texas and has future plans to expand into commercial there as well.

Cell therapy, like gene therapy, is attracting enormous funding as both the number of active programs and the level of funding have rapidly expanded over the last 5 years. There are now more than 500 public and private companies with cell therapy programs and hundreds of active cell therapy-based investigational new drug applications. Much of the focus today is in oncology, but we're seeing applications expand in other therapeutic areas, such as autoimmune diseases, cardiology and neurology. Our research indicates that the cell therapy pipeline is growing in the mid-teens range with over 800 cell therapy assets in the pipeline today and also estimate cell therapy manufacturing to be approximately 65% outsourced, which is comparable to viral vectors.

Also, similar to viral vector manufacturing, cell therapy capacity is scarce, and the trend of demand outstripping supply is projected to become more acute despite investments in additional capacity being made across the industry. We see MaSTherCell as a complementary addition to our gene therapy capabilities and the rest of our Biologics portfolio.

We also believe that MaSTherCell will be a strong, strategic fit for Catalent as we're well positioned to combine MaSTherCell's team of experts and differentiated capabilities with our extensive resources and our significant experience in scaling new platforms to help MaSTherCell build out its development and commercial manufacturing capabilities. Furthermore, we believe MaSTherCell rounds out our program to be the leader in gene and cell therapy, creating deeper and broader relationships with customers, and like we've seen with Paragon, open up cross-selling opportunities across Catalent's other technology platforms.

From a structural perspective, this is an all-cash transaction with a total purchasing price of $315 million subject to customary purchase adjustments. Catalent expects to finance this transaction with either a partial drawdown of its revolving credit facility with proceeds from a possible future incremental capital raise. Any such raise may also include funds for capital expenditures in support of our gene therapy programs and other strategic initiatives.

Slide 8 illustrates how our actions continue to fundamentally transform our business and increase our share of the R&D pipeline by significantly increasing our exposure to the faster-growing area of the industry that is biologics. We've done this through significant organic and inorganic investments, putting to work nearly $3 billion over the last 5 years.

In the 12-month period ended December 31, our Biologics segment represented 27% of our portfolio. In the quarter we're reporting today, it's now just over 30%. And when factoring in our long-term organic revenue growth guidance of 6% to 8%, combined with strategic acquisitions like MaSTherCell and Anagni, we believe we're on pace for 50% of our revenues to be driven from Biologics segment by the end of fiscal 2024 with total company revenues projected to be approximately $4.5 billion. Given the greater margin contributions from our Biologics segment, we believe adjusted EBITDA margins in 2024 will expand to at least 28%, up approximately 300 basis points from our expected levels in 2020.

We're proud that the combination of organic and inorganic investments we're making in biologics is already delivering substantial benefits to patients. We believe our strategy that drove us to uniquely combine capabilities to support the fastest-growing areas of drug development with Catalent's historical leadership and deep expertise in global contract drug manufacturing will continue to create significant value for our company, our customers and our shareholders.

Now I'll turn over the call over to Wetteny, who will take you through our second quarter financial results and the details related to our updated financial guidance.

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Wetteny N. Joseph, Catalent, Inc. - Senior VP & CFO [4]

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Thanks, John. I will begin this morning with a discussion on segment performance, where both the fiscal 2019 and fiscal 2020 second quarter results are presented on the basis of the revised reporting segments we introduced last quarter. Please turn to Slide 9, which presents our Softgel and Oral Technologies business. As in past earnings calls, my commentary around segment growth will be in constant currency.

Softgel and Oral Technologies revenue of $267.9 million increased 3% during the quarter, with segment EBITDA increasing 19%. After excluding the impact of the October 2019 divestiture of the segment's manufacturing site in Braeside, Australia, segment revenue and EBITDA grew 9% and 24%, respectively. The growth primarily relates to volume increases across the consumer health portfolio within Europe as well as increased demand in the prescription product business in North America, which is partially attributable to recently launched products.

Revenue in the consumer health business also increased in North America and Latin America due to the prior year shortage in ibuprofen API supply. Additionally, the strong segment EBITDA performance was driven by improved capacity utilization and favorable product mix across the network.

Slide 10 shows that our Biologics segment recorded revenue of $225.2 million in the quarter, which is up 66% versus the comparable prior year period, with segment EBITDA growing 61% quarter-over-quarter. Note that a large portion of both the revenue and the segment EBITDA growth was inorganic and driven by the gene therapy acquisitions, which contributed 56 percentage points to revenue and 49 percentage points to EBITDA growth. Excluding acquisitions, the segment recorded organic revenue growth of 10% in the second quarter and segment EBITDA growth of 12%.

Recent investments in our Biologics business continued to translate into growth during the second quarter as we recorded strong growth in drug product volumes in the U.S. As a reminder, drug substance revenue continues to be impacted by the completion of a limited-duration customer contract, which had a particularly high drop-through of EBITDA, following the completion of the client build-out of its own capacity. The customer's strategy to move its production in-house was fully contemplated when we entered into the contract, and the precise timing was less defined given typical production complexities. We continue to expect this to be a comparison headwind for our drug substance business for another quarter as we work to onboard new customers to increase our utilization levels. Drug acceptance, after excluding the completion of this noncell line clinical manufacturing contract, also grew year-on-year.

As I mentioned, we just closed on the Anagni acquisition on the 1st of January. As we did not know the timing of the close when we gave initial guidance in August, the site was not included in our original estimate, but is now reflected in our current guidance updated today. As the site is multipurpose, its future financial reporting is likely to be split between Biologics and OSD segments, and we will provide you more details when we report our third quarter.

To close out the commentary on Biologics, I'd like to echo John's excitement about bringing MaSTherCell's cell therapy expertise to Catalent, which enables us to establish a position in this exciting new therapeutic platform and stay at the forefront of bringing new, advanced therapies to scale. Catalent provides MaSTherCell access to growth capital, leverages its functional and system expertise and provides access to additional customers. However, given the company's early stage, MaSTherCell is not expected to provide meaningful EBITDA in the next 2 years as profit generated in its current clinical services will be consumed by its commercial build-out. We expect to provide additional color next quarter following the expected closing of the transaction.

Slide 11 shows that our Oral and Specialty Delivery segment recorded revenue of $143.2 million in the quarter, which is down 7% versus the comparable prior year period, with segment EBITDA declining 28% quarter-over-quarter. While we experienced growth in our orally delivered commercial products, this was more than offset by decreased volumes in the segment's respiratory and ophthalmic specialty delivery platform. This business experienced very strong demand a year ago as it generated revenues in anticipation of new product introductions. However, these NPIs have not yet materialized, creating a headwind for the segment this quarter, which despite expected sequential improvement will result in a year-on-year headwind for the remainder of the year and is factored in our new guidance. Despite the softness we are experiencing this quarter, we believe the OSD segment continues to have a very strong development pipeline, including several late-stage quasi-development programs that will drive future long-term growth.

In order to provide additional insight into our long-cycle businesses, which include Softgel and Oral Technologies, Biologics and Oral and Specialty Delivery, we are disclosing our long-cycle development revenue and the number of new product introductions as well as revenue from these NPIs. As a reminder, these metrics are only directional indicators of our business since we do not control the sales or marketing of these products nor can we predict the ultimate commercial success of them.

For the first 6 months of fiscal year 2020, we recorded development revenue across both small and large molecule of $422.5 million, which is more than 36% above the development revenue recorded in the first half of the prior fiscal year. Additional disclosure on our development revenue is included on our Form 10-Q to be filed today with the SEC. In addition, we introduced 87 new products in the first 6 months of fiscal year 2020, which are expected to contribute approximately $27 million revenue in the fiscal year.

Now as shown on Slide 12, our Clinical Supply Services segment posted revenue of $87.9 million or 9% growth over the second quarter of the prior year, segment EBITDA of $24 million or 15% growth. The strong growth in both revenue and segment EBITDA was driven by strong demand in the segment's storage and distribution and manufacturing and packaging businesses. All the segment revenue and EBITDA growth recorded within CSS was organic.

As of December 31, 2019, our backlog for the CSS segment was $390 million, a 4.5% sequential increase. The segment recorded net new business wins of $104 million during the second quarter, which is a decrease of 2.3% compared to the very high level of net new business wins recorded in the second quarter of the prior year. The segment's trailing 12-month book-to-bill ratio remains at 1.2x.

Slide 13 and 14 contain reference information for our second quarter and year-to-date segment results, both as reported and in constant currency.

Slide 15 provides a reconciliation of EBITDA from operations from the most approximate GAAP measure, which is net earnings. This bridge would assist in tying out our reported figures to our computation of adjusted EBITDA, which is detailed on the next slide.

Moving to adjusted EBITDA on Slide 16. Second quarter adjusted EBITDA increased 17% to $171 million or 23.7% of revenue compared to 23.4% of revenue reported in the second quarter of prior year. On a constant currency basis, our second quarter adjusted EBITDA increased 18%, including 5% organic growth.

On Slide 17, you can see that second quarter adjusted net income was $72 million or $0.45 per diluted share compared to adjusted net income of $65.4 million, also representing $0.45 per diluted share in the second quarter a year ago.

Slide 18 shows our debt-related ratios and our capital allocation priorities. Our total net leverage ratio as of December 31 was 4.2x, which has modestly reduced from the ratio as of the end of the prior quarter. Pro forma for completed acquisitions, our total net leverage ratio was 4.0x, which is an improvement of approximately 1/2 of a turn compared to the ratio at the time we announced the Paragon transaction. Given the free cash flow generation of the company and its growing adjusted EBITDA, the company naturally delevers between 0.5 and 0.75 of a turn per year. Additionally, continued investments in Biologics, including the new CapEx approved by our Board last week for our gene therapy business, led us to increase our fiscal year 2020 projections for CapEx spending. Taking into account customer funding, capital expenditures are now expected to be approximately 13% to 14% of net revenue compared to our initial assumption of 11% to 12% of net revenue. Our capital allocation priorities remain unchanged and focus first and foremost on organic growth followed by strategic M&A.

Now we'll turn to our financial outlook for fiscal year 2020 on Slide '19. As John reviewed in his opening comments, we are raising our financial guidance to reflect the acquisition of Anagni and for the continued growth of the gene therapy business and are also slightly tightening these ranges to reflect the passage of time. No contribution from MaSTherCell is assumed in this revised guidance, which regardless of when it closes will be immaterial to our full year 2020 results.

We now expect full year revenue in the range of $2.87 billion to $2.95 billion compared to our previous guidance of $2.78 billion to $2.88 billion. Note, this new guidance continues to assume organic revenue growth of 4% to 7%. Full year adjusted EBITDA -- for full year adjusted EBITDA, we now expect a range of $711 million to $735 million compared to our previous expectation of $700 million to $730 million. This new range continues to assume our original organic adjusted EBITDA growth assumption of 9% to 12%. Note the greater increase in our revenue guidance relative to our adjusted EBITDA guidance will result in a somewhat lower adjusted EBITDA margin level for 2020 than our original guidance. We now expect adjusted EBITDA margin to increase over fiscal year 2019 results of 23.8% by approximately 100 basis points at the midpoint of the new range versus the previous expectation of an approximate 150 basis point increase. This is largely due to the addition of Anagni, which, as expected, currently has lower utilization levels until it adds more customers.

We're also updating our full year adjusted income guidance to a range of $307 million to $331 million compared to the previous guidance of $300 million to $330 million. We now expect that our fully diluted share count on a weighted average basis for the fiscal year ending June 30 will be in the range of 160 million to 161 million shares, which continues to account the preferred shares we issued in May to fund part of the Paragon acquisition as if they all were converted to common shares in accordance with their terms. We continue to expect our consolidated effective tax rate to be between 24% and 26% for the fiscal year.

Finally, Tom Castellano is also in the room with us today, and I'd like to personally thank him for the outstanding job he has done leading the Investor Relations function for Catalent since our IPO. Tom will continue to add great value to the company in his new leadership role as Global Vice President of Operational Finance and as the finance leader for our Biologics segment. Tom has transitioned his IR responsibilities to Paul Surdez, who joined us last month and many of you know from his time leading Investor Relations at other public health care companies.

Operator, we'd now like to open the call 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 Tycho Peterson with JPMorgan.

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Tycho W. Peterson, JP Morgan Chase & Co, Research Division - Senior Analyst [2]

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I'll start with MaSTherCell. I know it's a smaller deal than Paragon, but I'm just wondering if you could compare and contrast the 2. How you think about kind of the cellular market versus the gene therapy market? How should we think about CapEx needs? Any customer concentration risks? And then as we think about kind of the longer-term guidance of 10% to 15% for Biologics, what do you think the cellular therapy market opportunity could do to that growth rate?

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John R. Chiminski, Catalent, Inc. - Chairman & CEO [3]

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Sure. A lot there, Tycho. So let me just step back and look at the big picture here. First of all, I think on our acquisition of Paragon in the gene therapy space really gave us the confidence to enter into another very fast-expanding space in cell therapy. When we take a look at the number of cell therapy trials that are ongoing, it actually significantly exceeds those in the gene therapy area, and it's growing kind of in the mid-teens growth rate.

I would say that from an acquisition standpoint, I would say that we have acquired MaSTherCell a little bit earlier in the cycle than we have from a Paragon standpoint. So obviously, a smaller acquisition compared to Paragon, but I would say we're probably catching it 2 to 3 years earlier in the cycle. So they're still early on. They've got a very strong position. I would say they're really the leading stand-alone cell therapy CDMO player, and they've got some tremendous capability. I would say from a customer standpoint, I think it's very similar to our acquisition of Paragon where you've got a couple of marquee base customers there, but then have behind that, a broad slate of overall customers, both in the autologous as well as the allogeneic area.

From a CapEx standpoint, I would say that on a comparative basis to Paragon, they're smaller numbers based upon the overall technology, but it is going to require some additional CapEx for us to build out the commercial facilities that they already have started in the Belgian area as well as the preclinical and clinical facility they have in Houston and an anticipated additional commercial facility there. So we've anticipated that in terms of looking at our CapEx going forward, which Wetteny can further detail out.

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Wetteny N. Joseph, Catalent, Inc. - Senior VP & CFO [4]

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Yes. Tycho, the one thing I would add is, as John mentioned in his prepared commentary, MaSTherCell is in the midst of expanding clinical operations with a new facility in the U.S. in addition to a commercial facility that they're in the middle of in Europe. So as those come on and ramp up, we would expect to attract even more customers into the business as we continue to scale it from a customer standpoint.

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Tycho W. Peterson, JP Morgan Chase & Co, Research Division - Senior Analyst [5]

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Okay. And then just one follow-up on oral and specialty. You talked about the delays in product approvals and maybe some pressure there for the next couple of quarters. I guess should we be modeling that business down then the next couple of quarters? And is -- when does Zydis Ultra start to kind of contribute as well? Is that going to be beneficial at all?

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Wetteny N. Joseph, Catalent, Inc. - Senior VP & CFO [6]

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Yes. So in terms of the remainder of the year, I would say, given my prepared commentary here, I would expect some continued headwind for the OSD segment for the balance of the fiscal year. That's all factored into the guidance that we just gave as well for the year, just giving you some additional color there. Although I would expect the business to show sequential improvement quarter-on-quarter. From a growth rate standpoint, it would still be a headwind for the balance of the year.

In terms of Zydis Ultra , as we've talked about, this is an exciting area for us to expand the base of our Zydis offering to be able to bring on molecules with bigger drug loading than we did before. We have gone through pilot stages, proving that the technology can work. We are in the midst of a capital expansion to scale that to commercial levels and have already signed a number of programs with customers to leverage that technology. But this is factored into our long-term confidence in this business segment as well in terms of its ability to grow at the 5% to 7% in the long term. But those are -- in terms of Zydis Ultra, we're talking further out before we'd start to see meaningful revenue from Zydis Ultra.

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Operator [7]

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Our next question comes from Dan Brennan with UBS.

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Daniel Gregory Brennan, UBS Investment Bank, Research Division - Senior Equity Research Analyst of Healthcare Life Sciences [8]

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Congrats on the quarter and the deal. First, just on Paragon, just -- can you give us a little flavor? It came in other than we expected this quarter, I guess, not surprising given the commentary intra-quarter and the overall market. But can you give us a little flavor for kind of what you're seeing there? And then secondarily, can you kind of clarify a little bit on the increased CapEx plans, kind of any clarification on kind of what the future revenue contribution as you build-out the capacity in Paragon? Because I know, John, you've alluded to that in your prepared remarks.

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Edited Transcript of CTLT earnings conference call or presentation 3-Feb-20 1:15pm GMT - Yahoo Finance

The complete range of information related to the Gene Therapies Market is obtained through various sources and this obtained the bulk of the information is arranged, processed, and represented by a group of specialists through the application of different methodological techniques and analytical tools such as SWOT analysis to generate a whole set of trade based study regarding the Gene Therapies Market.

ReportsnReports added a new report on The Gene Therapies Market report delivers the clean elaborated structure of the Report comprising each and every business related information of the market at a global level.

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Gene Therapies: A Growing Pipeline and Approval of CAR-T Cell Therapies Reflect Increasing Commercial Appeal, Despite the Difficulties Faced by products such as Glybera and Strimvelis discusses all gene therapies, including not only those that involve genetic modification, but also RNAi, CAR-T cell and aptamer-based therapies.

In 2017 alone, the marketing approvals of Kymriah, Yescarta and Luxturna added to this growing class of products. However, due to various challenges, progress in developing this technology and achieving commercial uptake over the past few decades has been slow.

In the short term, there is a shortfall in global gene and cell therapy manufacturing capacity, and in the long-term the personalized nature of many gene and cell therapies represents a further challenge that will not be met by the currently used, centralized biopharmaceutical manufacturing model.

Additionally, the very small number of patients in orphan genetic diseases that these therapies are especially well suited for has caused commercial difficulties for drugs such as Glybera and Strimvelis, and the high pricing of these therapies in response to the low patient number presented strong reimbursement difficulties.

However, the pipeline for gene therapies is robust; there are 985 in vivo gene therapies, and a further 354 CAR-T cell therapies currently in pipeline development.

Most are at an early stage of development, with 76% at the Discovery or Preclinical stage.

There are also currently 23 gene therapy programs in Phase III development. This report provides a comprehensive view of the clinical, R&D, commercial and competitive landscape of Gene Therapy, and assesses key developments in delivery vector technology, and challenges and advances associated with the production of such vectors.

Reasons to accessthis report:

Understand the current status of the field of therapeutic gene therapies, and the relative clinical and commercial success of currently marketed products,Assess the pipeline for gene therapies split by therapy area, vector type and intervention type, and stage of development. Additionally, a granular assessment of the pipeline is provided across the four major therapy areas for gene therapy: oncology, central nervous system disorders, ophthalmology, and genetic disorders, Gain a picture of the current competitive landscape, with a detailed breakdown of companies actively involved in the gene therapy pipeline, Understand the level of involvement in the landscape on the part of big pharma companies, and the extent to how gene therapies fit into the overall portfolios of companies in this field,Understand the strategic consolidations landscape in gene therapies across the past decade.

Access this Report @ http://www.reportsnreports.com/purchasme=1344314

Scope of this report:

Why do gene therapies still occupy only a minimal market share in their respective indications?,What can be learned from the gene therapies that have already reached the market, especially from products that have faced commercial difficulties, such as Glybera and Strimvelis?,What are the prospects of recent approvals such as Luxturna, Kymriah and Strimvelis?,What are the relative advantages and disadvantages of each vector type and which hold the most promise?,What proportion of the overall gene therapy R&D pipeline is occupied by each vector type?,How will the entry of gene therapies into the market change the global manufacturing landscape?

Table of Contents for Gene Therapies Market:

1 Table of Contents 2

1.1 List of Tables 3

1.2 List of Figures 3

2 Gene Therapy Overview 5

2.1 Types of Gene Therapy 7

2.1.1 Types of Intervention 8

2.1.2 Types of Vector 13

3 Currently Approved Gene Therapies 22

3.1 Glybera (alipogene tiparvovec) 22

3.2 Kynamro (mipomersen) 23

3.3 Macugen (pegaptanib) 24

3.4 Vitravene (fomivirsen) 25

3.5 Gendicine (rAd-p53) 26

3.6 Oncorine (rAd5-H101) 26

3.7 Neovasculgen (Pl-VEGF165) 27

3.8 Exondys 51 (eteplirsen) 28

3.9 Spinraza (nusinersen) 29

3.10 Strimvelis (GSK-2696273) 30

3.11 Kymriah (tisagenlecleucel) 32

3.12 Yescarta (axicabtagene ciloleucel) 33

3.13 Imlygic (talimogene laherparepvec) 34

3.14 Zalmoxis (Allogenic T cells encoding LNGFR and HSV-TK) 36

3.15 Luxturna (voretigene neparvovec) 37

4 Gene Therapy Production Strategies 38

4.1 Production of Viral Vectors 38

4.1.1 Case Study: Challenges in the Manufacture of AAV Vectors 40

4.2 Production of Cell-based Gene Therapies 42

5 Challenges to Gene Therapy Development 44

6 Gene Therapy Pipeline and Emerging Technologies 46

6.1 Gene Therapy Pipeline by Therapy Area and Stage of Development 46

6.2 Gene Therapy Pipeline by Intervention and Vector Type 48

6.3 Pipeline for CAR-T Cell Therapies 51

6.4 Company Positioning 52

6.4.1 Companies by Therapy Area 53

6.4.2 Companies by Stage of Development 54

6.4.3 Companies by Intervention Type 54

6.4.4 Companies by Vector Type 56

6.4.5 Companies Developing CAR-T Cell Therapies by Stage 57

6.5 Early Genome Editing Technologies 57

6.5.1 Zinc Finger Nucleases 58

6.5.2 TALEN 58

6.5.3 CRISPR-Cas9 59

7 Strategic Consolidations 62

7.1 Licensing Deals 62

7.1.1 Licensing Deals by Region Value and Year 62

7.1.2 Licensing Deals by Stage of Development and Value 64

7.1.3 Licensing Deals by Intervention and Vector Type 65

7.2 Co-development deals 66

7.2.1 Co-development Deals by Region, Year and Value 66

7.2.2 Co-development Deals by Stage of Development and Value 68

7.2.3 Co-development Deals by Intervention and Vector Type 69

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New release: 2020 Gene Therapies Market Report - WhaTech Technology and Markets News

Exosome Diagnostics Market Report analysis including industry Overview, Country Analysis, Key Trends, Key Retail Innovations, Competitive Landscape and Sector Analysis for upcoming years.

ReportsnReports added a new report on The Exosome Diagnostics Market Technologies report delivers the clean elaborated structure of the Report comprising each and every business related information of the market at a global level. The complete range of information related to the Exosome Diagnostics Market Technologies is obtained through various sources and this obtained the bulk of the information is arranged, processed, and represented by a group of specialists through the application of different methodological techniques and analytical tools such as SWOT analysis to generate a whole set of trade based study regarding the Exosome Diagnostics Market Technologies.

Download a Free PDF Sample of Exosome Diagnostics Market Technologies Research Report at:

http://www.reportsnreports.com/contactme=1781607

Top Companies mentioned in this report are Capricor Therapeutics Inc, Evox Therapeutics Ltd, ReNeuron Group Plc, Stem Cell Medicine Ltd, Tavec Inc, Codiak Biosciences Inc, Therapeutic Solutions International Inc, ArunA Biomedical Inc, Ciloa 85.

This latest report is on Exosome Diagnostics Market Technologies which explores the application of exosome technologies within the pharmaceutical and healthcare industries. Exosomes are small cell-derived vesicles that are abundant in bodily fluids, including blood, urine and cerebrospinal fluid as well as in in vitro cell culture.

These vesicles are being used in a variety of therapeutic applications, including as therapeutic biomarkers, drug delivery systems and therapies in their own right. Research within this area remains in the nascent stages, although a number of clinical trials have been registered within the field.

Exosomes have several diverse therapeutic applications, largely centering on stem cell and gene therapy.

Exosomes have been identified as endogenous carriers of RNA within the body, allowing for the intracellular transportation of genetic material to target cells.

As such, developers have worked to engineer exosomes for the delivery of therapeutic miRNA and siRNA-based gene therapies. As RNA is highly unstable within the body, a number of different biological vector systems have been developed to enhance their transport within the circulation, including viruses and liposomes.

Similarly, exosomes derived from stem cells have also been identified for their therapeutic applications, particularly in the treatment of cancer and cardiovascular disease. Exosome technologies offer several advantages over existing biologic-based drug delivery systems.

Reasons to buy this Report:

Develop a comprehensive understanding of exosome technologies and their potential for use within the healthcare sector, Analyze the pipeline landscape and gain insight into the key companies investing in exosomes technologies, Identify trends in interventional and observational clinical trials relevant to exosomes.

Get this Report @ http://www.reportsnreports.com/purchasme=1781607

Scope of this Report:

What are the features of the exosome lifecycle?,How are therapeutic exosomes prepared?,How do exosome therapies in development differ in terms of stage of development, molecule type and therapy area?,Which companies are investing in exosome technologies?,How many clinical trials investigate exosomes as biomarkers, therapeutics and vectors?

Table of contents for Exosome Diagnostics Market Technologies:

1 Table of Contents 4

1.1 List of Tables 6

1.2 List of Figures 7

2 Exosomes in Healthcare 8

2.1 Overview of Exosomes 8

2.2 Drug Delivery Systems 9

2.2.1 Modified Release Drug Delivery Systems 9

2.2.2 Targeted Drug Delivery Systems 10

2.2.3 Liposomes 12

2.2.4 Viruses 14

2.2.5 Exosomes 17

2.3 The Exosome Lifecycle 18

2.4 Exosomes in Biology 18

2.5 Exosomes in Medicine 19

2.5.1 Biomarkers 19

2.5.2 Vaccines 20

2.6 Exosomes as a Therapeutic Target 20

2.7 Exosomes as Drug Delivery Vehicles 21

2.8 Therapeutic Preparation of Exosomes 21

2.8.1 Isolation and Purification 22

2.8.2 Drug Loading 22

2.8.3 Characterization 23

2.8.4 Bioengineering 23

2.8.5 Biodistribution and In Vivo Studies 23

2.8.6 Advantages of Exosome Therapies 24

2.8.7 Disadvantages of Exosome Therapies 24

2.9 Exosomes in Therapeutic Research 25

2.9.1 Exosome Gene Therapies 25

2.9.2 Exosome in Stem Cell Therapy 26

2.10 Exosomes in Oncology 27

2.10.1 Immunotherapy 27

2.10.2 Gene Therapy 28

2.10.3 Drug Delivery 29

2.10.4 Biomarkers 30

2.11 Exosomes in CNS Disease 30

2.11.1 Tackling the Blood-Brain Barrier 30

2.11.2 Exosomes in CNS Drug Delivery 31

2.11.3 Gene Therapy 32

2.12 Exosomes in Other Diseases 33

2.12.1 Cardiovascular Disease 33

2.12.2 Metabolic Disease 33

3 Assessment of Pipeline Product Innovation 36

3.1 Overview 36

3.2 Exosome Pipeline by Stage of Development and Molecule Type 36

3.3 Pipeline by Molecular Target 37

3.4 Pipeline by Therapy Area and Indication 38

3.5 Pipeline Product Profiles 38

3.5.1 AB-126 - ArunA Biomedical Inc. 38

3.5.2 ALX-029 and ALX-102 - Alxerion Biotech 39

3.5.3 Biologics for Autism - Stem Cell Medicine Ltd 39

3.5.4 Biologic for Breast Cancer - Exovita Biosciences Inc. 39

3.5.5 Biologics for Idiopathic Pulmonary Fibrosis and Non-alcoholic Steatohepatitis - Regenasome Pty 39

3.5.6 Biologic for Lysosomal Storage Disorder - Exerkine 39

3.5.7 Biologics for Prostate Cancer - Cells for Cells 40

3.5.8 CAP-2003 - Capricor Therapeutics Inc. 40

3.5.9 CAP-1002 - Capricor Therapeutics Inc. 41

3.5.10 CIL-15001 and CIL-15002 - Ciloa 42

3.5.11 ExoPr0 - ReNeuron Group Plc 42

3.5.12 MVAX-001 - MolecuVax Inc. 43

3.5.13 Oligonucleotides to Activate miR124 for Acute Ischemic Stroke - Isfahan University of Medical Sciences 44

3.5.14 Oligonucleotides to Inhibit KRAS for Pancreatic Cancer - Codiak BioSciences Inc. 44

3.5.15 Proteins for Neurology and Proteins for CNS Disorders and Oligonucleotides for Neurology - Evox Therapeutics Ltd 44

3.5.16 TVC-201 and TVC-300 - Tavec Inc. 45

4 Assessment of Clinical Trial Landscape 48

4.1 Interventional Clinical Trials 48

4.1.1 Clinical Trials by Therapy Type 48

4.1.2 Clinical Trials by Therapy Area 49

4.1.3 Clinical Trials by Stage of Development 50

4.1.4 Clinical Trials by Start Date and Status 50

4.2 Observational Clinical Trials 51

4.2.1 Clinical Trials by Therapy Type 51

4.2.2 Clinical Trials by Therapy Area 51

4.2.3 Clinical Trials by Stage of Development 52

4.2.4 Clinical Trials by Start Date and Status 53

4.2.5 List of All Clinical Trials 54

5 Company Analysis and Positioning 67

5.1 Company Profiles 67

5.1.1 Capricor Therapeutics Inc. 67

5.1.2 Evox Therapeutics Ltd 72

5.1.3 ReNeuron Group Plc 73

5.1.4 Stem Cell Medicine Ltd 77

5.1.5 Tavec Inc. 78

5.1.6 Codiak Biosciences Inc. 80

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Current research: 2020 Latest Report on Exosome Diagnostics Market Report Technologies, Analyze the Pipeline Landscape and Key Companies - WhaTech...

BOSTON--(BUSINESS WIRE)--Decibel Therapeutics, a development-stage biotechnology company developing novel therapeutics for hearing loss and balance disorders, today announced a new strategic research focus on regenerative medicine approaches for the inner ear. The company is also announcing a collaboration and option agreement that gives Decibel exclusive access to novel compounds targeting proteins in a critical regenerative pathway.

Decibels research focus on regeneration will be powered by the companys research and translation platform. The company has built one of the most sophisticated single cell genomics and bioinformatics platforms in the industry to identify and validate targets. Decibel has also developed unique insights into regulatory pathways and inner ear delivery mechanisms that together enable precise control over gene expression in the inner ear and differentiate its AAV-based gene therapy programs.

Our deep understanding of the biology of the inner ear and our advanced technological capabilities come together to create a powerful platform for regenerative medicine therapies for hearing and balance disorders, said Laurence Reid, Ph.D., acting CEO of Decibel. We see an exciting opportunity to leverage this platform to address a broad range of hearing and balance disorders that severely compromise quality of life for hundreds of millions of people around the world.

The first program in Decibels regeneration portfolio aims to restore balance function using an AAV-based gene therapy (DB-201), which utilizes a cell-specific promoter to selectively deliver a regeneration-promoting gene to target cells. In collaboration with Regeneron Pharmaceuticals, Decibel will initially evaluate DB-201 as a treatment for bilateral vestibulopathy, a debilitating condition that significantly impairs balance, mobility, and stability of vision. Ultimately, this program may have applicability in a broad range of age-related balance disorders. There are currently no approved medicines to restore balance. Decibel expects to initiate IND-enabling experiments for this program in the first half of 2020.

Decibel is also pursuing novel targets for the regeneration of critical cells in both the vestibule and cochlea of the inner ear; these targets may be addressable by gene therapy or other therapeutic modalities. As a key component of that program, Decibel today announced an exclusive worldwide option agreement with The Rockefeller University, which has discovered a novel series of small-molecule LATS inhibitors. LATS kinases are a core component of the Hippo signaling pathway, which plays a key role in regulating both tissue regeneration and the proliferation of cells in the inner ear that are crucial to hearing and balance. The agreement gives Decibel an exclusive option to license this series of compounds across all therapeutic areas.

The agreement also establishes a research collaboration between Decibel and A. James Hudspeth, M.D., Ph.D., the F.M. Kirby Professor at The Rockefeller University and the director of the F.M. Kirby Center for Sensory Neuroscience. Dr. Hudspeth is a world-renowned neuroscientist, a member of the National Academy of Sciences and the American Academy of Arts and Sciences, and a Howard Hughes Medical Institute investigator. Dr. Hudspeth has been the recipient of numerous prestigious awards, including the 2018 Kavli Prize in Neuroscience.

Rockefeller scientists are at the leading edge of discovery, and we are excited to see the work of Dr. Hudspeth move forward in partnership with Decibel, said Jeanne Farrell, Ph.D., associate vice president for technology advancement at The Rockefeller University. The ambitious pursuit of harnessing the power of regenerative medicine to create a new option for patients with hearing loss could transform how we address this unmet medical need in the future.

In parallel with its new research focus on regenerative strategies, Decibel will continue to advance key priority preclinical and clinical programs. DB-020, the companys clinical-stage candidate designed to prevent hearing damage in people receiving cisplatin chemotherapy, is in an ongoing Phase 1b trial. Decibel will also continue to progress DB-OTO, a gene therapy for the treatment of genetic congenital deafness, which is being developed in partnership with Regeneron Pharmaceuticals. The DB-OTO program aims to restore hearing to people born with profound hearing loss due to a mutation in the otoferlin gene and is expected to progress to clinical trials in 2021.

To support the new research focus, Decibel is restructuring its employee base and discontinuing some early-stage discovery programs.

About Decibel Therapeutics, Inc.Decibel Therapeutics, a development-stage biotechnology company, has established the worlds first comprehensive drug discovery, development, and translational research platform for hearing loss and balance disorders. Decibel is advancing a portfolio of discovery-stage programs aimed at restoring hearing and balance function to further our vision of a world in which the benefits and joys of hearing are available to all. Decibels lead therapeutic candidate, DB-020, is being investigated for the prevention of ototoxicity associated with cisplatin chemotherapy. For more information about Decibel Therapeutics, please visit decibeltx.com or follow @DecibelTx.

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Decibel Therapeutics Announces Strategic Research Focus on Regenerative Medicine for the Inner Ear - Business Wire

The biotech Hotbed known as Pharm Country, which includes New Jersey, Connecticut, New York Pennsylvania and Rhode Island, is an area of rapid growth and change. As a result, its no wonder that some of the biggest companies in the world call this Hotbed home.

In BioSpaces recently published 2019 Life Sciences Ideal Employer Report, more than 2,700 life sciences professionals weighed in on their Ideal Employers. BioSpace broke down the data to gain an insight into each of the BioSpace Hotbed regions and who those survey takers thought should be considered Ideal Employers. Similar to the Biotech Bay list and the top 10 list for Genetown, BioSpace takes a look at the top 10 2019 Life Sciences Ideal Employer Reports for Pharm Country.

As BioSpace notes in its Pharm Country report, not all of the companies included in this region made the 2019 Ideal Employer list, such as Janssen, Novo Nordisk and Spark Therapeutics. Some companies that did rank on the Ideal Employer List, such as Regeneron, rank higher here, most likely due to the fact its headquarters is located in Tarrytown, New York.

See more here:
Pharm Country Professionals Weigh In on Their Ideal Employers - BioSpace

Top research study on Global Personalized Gene Therapy Treatments For Cancer Market is an extensive compilation of innovative developments, growth opportunities and revenue analysis of top-tier Personalized Gene Therapy Treatments For Cancer Industry aspirants. The report states the growth trajectory of Global Personalized Gene Therapy Treatments For Cancer Market growth during 2020-2026. Key industry aspects like SWOT analysis, Porters five forces analysis, and market statistics are mentioned. Global Personalized Gene Therapy Treatments For Cancer Industry is expected to reach xx million USD in 2020 and will grow at a CAGR of xx% during 2020-2026.

Checkout TOC And Research Coverage With Important Details Here: https://reportscheck.biz/report/44134/global-personalized-gene-therapy-treatments-for-cancer-industry-market-research-report/#table-of-content

The prime manufacturers of Personalized Gene Therapy Treatments For Cancer Market is as follows:

GenprexGenesysCelsionAdaptimmuneAdvantageneAmgenMaxiVaxBioCancellCelgeneBlubird BIOOncoSecBellicum

The production, regional trade, investment opportunities, mergers & acquisitions and sales channels of Personalized Gene Therapy Treatments For Cancer Industry are stated. The top manufacturers, product types, applications, and market share is stated. The regional Personalized Gene Therapy Treatments For Cancer analysis covers North America, Europe, China, Japan, India, South America, Middle East, and Africa and the rest of the world.

The product types covered in the report are as follows:

Type 1Type 2Type 3Type 4Type 5

The top applications in Personalized Gene Therapy Treatments For Cancer Market are as follows:

Application 1Application 2Application 3Application 4Application 5

Global Personalized Gene Therapy Treatments For Cancer Research Report offers complete details about industry chain structure, raw materials, pricing analysis, company profiles, and product specifications. The sales analysis, value chain optimization, strategic insights on Personalized Gene Therapy Treatments For Cancer Industry, product launches and market risks are mentioned in this report. The country-level analysis of Personalized Gene Therapy Treatments For Cancer Report covers USA, Canada, Mexico, Germany, France, UK, Russia, Italy, China, Japan, India, Korea, Australia, Brazil, Argentina, Colombia, Saudi Arabia, UAE, Egypt, South Africa and rest of the world. Market scope, revenue, information on product services and gross margin status is covered in this report. The import-export scenario, demand-supply, consumer behavior, and complete details on distributors, suppliers, traders, and dealers in Personalized Gene Therapy Treatments For Cancer Market are stated.

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Key Extracts From Table Of Content:

Section 1: Market Introduction and Overview

Section 2: Product Overview, Classification, Scope

Section 3: Competitive Personalized Gene Therapy Treatments For Cancer Market scenario based on Top Manufacturers

Section 4: Historic Study of Personalized Gene Therapy Treatments For Cancer Market Based on Region, Type, Application

Section 5: Company Profiles of Key Personalized Gene Therapy Treatments For Cancer Players, Market Share, Product Portfolio and Regional Presence

Section 6: Manufacturing Cost Analysis, Key Business Figures, Gross Margin, SWOT Analysis

Section 7: Regional Analysis, Market Status and Prospect From 2015-2026

Section 8: Market Dynamics, Marketing and Sales Channels, Distributors and Customer Analysis

Section 9: Financial Highlights of Personalized Gene Therapy Treatments For Cancer Market Including Total Revenue, Products, Services, Opportunities, and Market Risk Analysis

Section 10: Global Personalized Gene Therapy Treatments For Cancer Market Forecast Study, Marketing Channels, Cost Structures, Distributors and Consumer Study

Section 11: Region-wise Forecast Analysis of Sales, Revenue, Growth Rate Till 2026

Section 12: Research Findings, Conclusion, Data Sources, Research Methodology, and Disclaimer

A complete qualitative and competitive assessment of Personalized Gene Therapy Treatments For Cancer Market is conducted to offer valuable insights. This will enable the market aspirants in shaping their business plans and planning growth strategies. Primary and secondary research techniques like interviews, trade journals, surveys, and reputable paid database sources. A complete historical analysis from 2014-2019 and forecast analysis 2020-2026 with base year as 2019. Our competitive business landscape will help you to gain upper hand in competition.

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ReportsCheck.biz is one stop platform which caters to all your market research needs as well as custom and consulting services. We have an extensive database of research reports to meet the global, regional and country-level research requirements of our clients. We gather complete information about all products with the help of research methodology and verified data sources. We have an expert team to understand and map client requirements to provide precise research analysis. Our research solutions will help readers in aligning their business and analyzing business priorities.

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Global Personalized Gene Therapy Treatments For Cancer Market 2020 Reflects Accelerated Growth Driven By Rapid Urbanization And Changing Consumer...

Fior Marketshas the latest research report titledGlobalGene TherapyMarket Growth 2019-2024which presents the clean elaborated structure of the report comprising an in-depth evaluation of this industry and a commendable brief of its segmentation. Our team of expert researchers has obtained the complete range of information related to the globalGene Therapymarket. The information gathered from different sources has been arranged, processed, and represented by a group of specialists through the application of different methodological techniques and analytical tools such as SWOT analysis.

DOWNLOAD FREE SAMPLE REPORT :https://www.fiormarkets.com/report-detail/370313/request-sample

Historic And Future Forecast Analysis of The Market:

The research study divides the globalGene Therapymarket by top players/brands, regions, types, and end-user. It comprehensively delivers the market portion, improvement, patterns, and expectations for the period 2019-2024. The market is observing the arrival of local vendors entering the market. Additionally, key countries expected to display significant growth prospects in the future are included in the report. Moreover, the report provides key market dynamics, the profile of key market players, and a comprehensive outline of the market environment in terms of sales and production for the forecast period 2019-2024.

Key manufacturersare included based on the company profile, sales data and product specifications, etc.:Bluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical Inc., Advantagene

The exploration report contains the board investigation of the geographical scene of the market, which is evidently arranged into the locales

A Brief Outline of The Major Takeaways of The Market Report Has Been Enlisted Below:

The report contains a thorough overview of the competitive backdrop of the globalGene Therapymarket that encompasses leading firms. A concise synopsis of all the manufacturers, a product developed, and product application scopes have been included. The study calculates the sales registered by the products as well as the revenues earned over the foreseeable duration. The revenues accumulated by these applications as well as the sales projections for the projected timeframe are also included in the report. Data with respect to the market dynamics such as the potential growth opportunities, challenges have been covered in this report.

BROWSE COMPLETE REPORT AND TABLE OF CONTENTS:https://www.fiormarkets.com/report/global-gene-therapy-market-growth-status-and-outlook-370313.html

The GlobalGene TherapyMarket Report Answers Important Questions Which Include:

Concluding part of the reportoffers various traders, contributors engaged in theGene Therapyindustry along with research discoveries, results, data source and postscript.

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Mark StoneSales ManagerPhone:+1-201-465-4211Email:[emailprotected]Web:www.fiormarkets.com

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Global Gene Therapy Market 2019 By Demand drivers Bluebird Bio, Sangamo, Spark Therapeutics - Jewish Life News

DUBLIN, Jan. 30, 2020 /PRNewswire/ -- The "Gene Therapy Market by Vectors [Non-viral (Oligonucleotides), Viral (Retroviral (Gammaretroviral, Lentiviral)), Adeno-associated], Indication (Cancer, Neurological Diseases), Delivery Method (In Vivo, Ex Vivo), Region - Global Forecast to 2024" report has been added to ResearchAndMarkets.com's offering.

The global gene therapy market is projected to reach USD 13,005.6 million by 2024 from an estimated USD 3,814.9 million in 2019, at a CAGR of 27.8% during the forecast period.

This report provides a detailed picture of the global gene therapy market. It aims at estimating the size and future growth potential of the market across different segments (by vector, indication, delivery method, and region). The report also includes an in-depth competitive analysis of the key market players, along with their company profiles, recent developments, and key market strategies.

High incidence of cancer & other target diseases is a major factor driving the growth of the gene therapy market

The high incidence of cancer and other target diseases, availability of reimbursement, and the launch of new products are the major factors driving the growth of this market. In addition, the strong product pipeline of market players is expected to offer significant growth opportunities in the coming years. However, the high cost of treatment is expected to hamper the market growth to a certain extent in the coming years.

Neurological diseases segment accounted for the largest share of the gene therapy market, by indication, in 2018

Based on indication, the market is segmented into neurological diseases, cancer, hepatological diseases, Duchenne muscular dystrophy, and other indications. The neurological diseases segment accounted for the largest share of the market in 2018. This can be attributed to the increasing number of gene therapy products being approved for the treatment of neurological diseases and the high market penetration of oligonucleotide-based gene therapies.

Viral vectors segment to register the highest growth in the gene therapy market during the forecast period

The gene therapy market, by vector, has been segmented into viral and non-viral vectors. In 2018, the non-viral vectors segment accounted for the largest share of this market. However, the viral vectors segment is estimated to grow at the highest CAGR during the forecast period, primarily due to the increasing demand for CAR T-based gene therapies and the rising incidence of cancer.

North America will continue to dominate the gene therapy market during the forecast period

Geographically, the market is segmented into North America, Europe, the Asia Pacific, and the Rest of the World. In 2018, North America accounted for the largest share of the gene therapy market, followed by Europe. Factors such as the rising prevalence of chronic diseases, high healthcare expenditure, presence of advanced healthcare infrastructure, favorable reimbursement scenario, and the presence of major market players in the region are driving market growth in North America.

Key Topics Covered

1 Introduction

2 Research Methodology 2.1 Research Data2.2 Secondary Data2.2.1 Secondary Source2.3 Primary Data2.4 Market Size Estimation2.4.1 Bottom-Up Approach2.4.2 Bottom-Up Approach for Non-Viral Vectors and Viral Vectors Market2.4.3 Growth Forecast2.5 Market Breakdown and Data Triangulation2.6 Assumptions for the Study

3 Executive Summary

4 Premium Insights 4.1 Gene Therapy Market Overview4.2 North America: Market, By Vector (2018)4.3 Geographical Snapshot of the Market

5 Market Overview 5.1 Introduction5.2 Market Dynamics5.2.1 Drivers5.2.1.1 High Incidence of Cancer and Other Target Diseases5.2.1.2 Product Approvals5.2.1.3 Funding for Gene Therapy Research5.2.2 Opportunities5.2.2.1 Strong Product Pipeline5.2.3 Challenges5.2.3.1 High Cost of Treatments

6 Gene Therapy Market, By Vector 6.1 Introduction6.2 Non-Viral Vectors6.2.1 Oligonucleotides6.2.1.1 North America Accounted for the Largest Share of the Oligonucleotides Segment6.2.2 Other Non-Viral Vectors6.3 Viral Vectors6.3.1 Retroviral Vectors6.3.1.1 Gamma-Retroviral Vectors6.3.1.1.1 The Availability of A Wide Range of Gamma-Retroviral Vectors Supports the Growth of This Market6.3.1.2 Lentiviral Vectors6.3.1.2.1 North America Accounted for the Largest Share of the Lentiviral Vectors Segment6.3.2 Adeno-Associated Virus Vectors6.3.2.1 Possible Applications in In Vivo Applications Have Driven Interest in Adeno-Associated Virus Vectors6.3.3 Other Viral Vectors

7 Gene Therapy Market, By Indication 7.1 Introduction7.2 Neurological Diseases7.2.1 Neurological Diseases Account for the Largest Share of the Market7.3 Cancer7.3.1 Cancer is Expected to Show the Highest Growth in This Market7.4 Hepatological Diseases7.4.1 Increasing Prevalence of Hepatitis B Infections Will Support Market Growth7.5 Duchenne Muscular Dystrophy7.5.1 North America Accounted for the Largest Share of the Dmd Gene Therapy Segment7.6 Other Indications

8 Gene Therapy Market, By Delivery Method 8.1 Introduction8.2 In Vivo Gene Therapy8.3 Ex Vivo Gene Therapy

9 Gene Therapy Market, By Region 9.1 Introduction9.2 North America9.2.1 US9.2.1.1 The US Dominates the Global Market9.2.2 Canada9.2.2.1 Growing Burden of Cancer Will Support Market Growth in Canada9.3 Europe9.3.1 Germany9.3.1.1 Germany Accounted for the Largest Share of the Market in Europe9.3.2 France9.3.2.1 Increasing Cancer Incidence Supports Market Growth9.3.3 UK9.3.3.1 Rising Incidence of Melanoma Will Drive Demand in the UK9.3.4 Italy9.3.4.1 High Incidence of Targeted Diseases and Increasing Per Capita Healthcare Spending Will Drive Market Growth in Italy9.3.5 Spain9.3.5.1 Non-Viral Vectors Dominate the Spanish Market, By Vector9.3.6 Rest of Europe9.4 Asia Pacific9.4.1 Japan9.4.1.1 Japan Dominates the APAC Market for Gene Therapy9.4.2 China9.4.2.1 Rising Prevalence of Cancer and Established Base for Gene Therapy are Supportive Factors in China9.4.3 Rest of APAC9.5 Rest of the World

10 Competitive Landscape 10.1 Overview10.2 Market Share Analysis, 201810.3 Key Strategies10.4 Competitive Leadership Mapping (2018)10.4.1 Visionary Leaders10.4.2 Innovators10.4.3 Dynamic Differentiators10.4.4 Emerging Companies

11 Company Profiles 11.1 Biogen11.2 Gilead Sciences, Inc.11.3 Amgen, Inc.11.4 Novartis AG11.5 Orchard Therapeutics PLC11.6 Spark Therapeutics, Inc. (A Part of Hoffmann-La Roche)11.7 Molmed S.p.A.11.8 Anges, Inc.11.9 Bluebird Bio, Inc.11.10 Human Stem Cells Institute (HSCI)11.11 SIBIONO Genetech Co. Ltd.11.12 Shanghai Sunway Biotech Co. Ltd.11.13 Uniqure N.V.11.14 Gensight Biologics S.A.11.15 Celgene Corporation (A Bristol-Myers Squibb Company)11.16 Cellectis11.17 Sangamo Therapeutics11.18 Mustang Bio11.19 AGTC (Applied Genetic Technologies Corporation)11.20 Poseida Therapeutics, Inc.

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Global Gene Therapy Industry Outlook 2020-2024 - Strong Product Pipeline Gives Rise to Lucrative Growth Opportunities - P&T Community

After decades of clinical research targeting development of more personalised medicines, the biotechnology sector is now starting to deliver with the first generation of promising new cell and gene therapies.

Whether the optimism about the impact of these therapies plays out as expected or not, advances in this area of research are also having a significant effect on the pace of deal-making in the life sciences sector.

As many investigational cell and gene therapies continue to advance and demonstrate their clinical and commercial potential, companies including smaller biotechnology firms and larger pharmaceutical companies are positioning themselves for opportunities to acquire assets or develop collaborative relationships to take advantage of these new technologies.

Given that development of cell and gene therapies is still in its nascent stages and uncertainties remain about their long-term potential, some industry insiders have questions about the risks involved and how financial deals related to these drugs should be structured.

To better understand deal-making trends related to cell and gene therapies, we conducted an analysis of recent activity in the sector.

We reviewed more than 30 mergers, acquisitions, and licensing agreements executed between 2016-2019 and compared them to deals that were executed for the previous generation of breakthrough therapies, including monoclonal antibodies (mAbs).

In this analysis, we considered a range of factors including the size of the companies involved, target indications, deal structures, terms such as upfront payments and royalties, and the number and clinical stage of assets included in each deal.

One key difference in cell and gene therapy deals is that companies prefer to target partnerships and licensing deals. This shift may be a reflection of the unique challenges in development of these drugs

The review shows that overall the pace of deal-making in cell and gene therapy is faster and occurring much earlier in the drug development process compared to deals seen in the past.

More deals and earlier deals

The pace of deals in the cell and gene therapy sector has been building in momentum since the first of these drugs received regulatory approval nearly a decade ago. From 2010-2016 alone, pharmaceutical companies executed more than 50 partnerships and investments to access cell and gene therapies.[1]

Larger companies have often been involved in these deals from the beginning. In 2010, Novartis established a partnership deal with GenVec worth potentially $213M (excluding royalties) for development of adenovirus-based gene therapies. That same year Novartis also formed a strategic alliance with GlaxoSmithKline and the Telethon Institute of Gene Therapy (TIGET) to pursue additional gene therapy research and development.

Unlike deals made decades ago related to mAbs, larger pharmaceutical companies are not waiting for the cell and gene therapy sector to become well established before pursuing partnerships or M&A deals.

With mAbs, financial deals were often characterised by the acquisitions of late-stage and marketed products, significantly reducing levels of risk. Conversely, acquisitions and other deals in the cell and gene therapy space often include entire product pipelines, platform technologies, and manufacturing capabilities with the potential to deliver significant clinical value and commercial potential.

Source: CRA analysis

With growing eagerness to buy into the cell and gene therapy market, the fact that companies are targeting these drugs earlier in the development cycle is also driving them to consider innovative deal structures to reduce risk.

One key difference with respect to cell and gene therapy deals is that most companies prefer to target partnerships and licensing deals rather than mergers or acquisitions, which were more commonly associated with mAbs. This shift may be based in part on the lack of long-term safety and efficacy data associated with many cell and gene therapies, but it also may be a reflection of the unique challenges in development of these drugs.

Despite the risks and unknowns, interest in the sector continues to grow dramatically. Pfizers first move into cell and gene therapy, for example, was through a collaboration with Spark Therapeutics regarding clinical development and potential commercialization of its Phase 1/2 gene therapy for treatment of hemophilia B.

Subsequently, Pfizer further expanded its cell and gene therapy portfolio by acquiring Bamboo Therapeutics in 2016, which provided a Phase 1 product and several preclinical stage assets, and by partnering with Vivet Therapeutics in early 2019 on development of a Phase 1/2 drug for treatment of a rare liver disease. The deal with Bamboo Therapeutics is a good example of a company structuring a deal to gain access to several different products or platforms rather than only one asset, which was often the case with mAbs.

Transforming the M&A and licensing landscape

While it took more than 20 years for collaborative activity around mAbs to generate significant interest from Big Pharma and lead to billion-dollar deals, the cell and gene therapy sector has reached this level of activity in less than 10 years.

This may be driven by the claims that these drugs represent innovative platforms and approaches for treatment of many conditions, including rare diseases. As research advances, industry partners recognise the potential for these drugs to represent major standard of care advances in areas where effective treatments are limited, sub-optimal, or non-existent.

As the discovery of promising new cell and gene therapies continues to expand, the emphasis on collaboration and innovative structuring in licensing, partnering, and M&A arrangements is expected to grow rapidly. It will be increasingly essential for participants in early-stage deals to have the expertise and insight necessary to conduct a thorough assessment of the risks and potential benefits of each option in structuring a deal. By working to understand and address any issues, they can better protect their interests over the long term. Companies that do not develop this capability to assess deals quickly and precisely risk being left behind.

About the authors

Lev Gerlovin is a vice president in the Life Sciences Practice at CRA. He has more than 12 years of experience in life sciences strategy consulting, focused on commercial and market access strategies.

Pascale Diesel previously served as a vice president in the Life Sciences Practice at CRA from July 2017 through December 2019. Dr Diesel has worked in global development, marketing, planning, and business development and has more than a decade of strategic consulting experience focusing on portfolio optimisation and valuation.

The views expressed herein are the authors and not those of Charles River Associates (CRA) or any of the organisations with which the authors are affiliated.

[1] Kong X., Wan J., Hu H., Su S., Hu, Y., Evolving patterns in a collaboration network of global R&D on monoclonal antibodies, mABs, 2017, 9,7, 1041-1051.

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How cell and gene therapies are transforming pharma deal-making - - pharmaphorum

It's easy to grasp how Vertex Pharmaceuticals (NASDAQ:VRTX) got to where it is today. A decade ago, the biotech's market cap hovered around $8 billion. Vertex's lead pipeline candidate was hepatitis C virus (HCV) drugtelaprevir, which went on to win FDA approval in 2011. The drug was marketed under the brand name Incivek -- but only briefly. Vertex quit selling the HCV drug in 2014 because Gilead Sciences'HCV franchise was dominating the market.

However, Vertex had another program in development targeting cystic fibrosis (CF). Its first CF drug, Kalydeco, won FDA approval in 2012. The rest is history. Vertex went on to gain FDA approvals for three other CF drugs. It's now highly profitable with annual revenue approaching $4 billion. And its stock has skyrocketed more than 500% over the last 10 years.

Trying to predict where Vertex will be 10 years from now isn't as easy. But there are some clues from the present that point to the prospects for another highly successful decade for the biotech.

Image source: Getty Images.

The safest prediction of all for Vertex is that it will remain a juggernaut in CF in 2030. Vertex won FDA approval for its most powerful CF drug yet -- Trikafta -- in October 2019. European approval for the drug is likely on the way this year.

Vertex expects that Trikafta will expand the addressable patient population for its CF therapies by more than 50%. The company also has three other CF drugs in its pipeline, including two programs that, like Trikafta, are triple-drug combos.

Currently, there are no other approved drugs that treat the underlying cause of CF. AbbVie is evaluating a triple-drug CF combo that it picked up from Galapagosin a phase 1 clinical study, but it's way behind Vertex. Even if AbbVie's drug proves to be successful, it would at best be several years before the drug could win approval. By that time, Vertex will already have further entrenched itself in the CF market.

Although the patents for Kalydeco, Orkambi, and Symdeko will expire near the end of the decade, Vertex's patents for Trikafta won't expire until 2037. The company could face generic rivals for its older CF drugs, but there's no reason to expect that Vertex's CF franchise won't still be racking up huge sales.

Vertex doesn't plan on being a one-indication company 10 years from now, though. The biotech has been busy expanding its pipeline and advancing the most promising programs.

The most likely addition to Vertex's lineup in 2030 will be a pain medication. Vertex has already completed phase 2 clinical studies for experimental pain drug VX-150. Chief Medical Officer and soon-to-be CEO ReshmaKewalramanisaid in Vertex's Q3 conference call in October that the company is "advancing multiple selective NaV1.8 inhibitors through late-stage research and early clinical development."

I think that Vertex and its partner CRISPR Therapeutics also have a good chance of launching a few years from now a gene-editing therapy that effectively cures rare blood disorders beta-thalassemia and sickle cell disease. The two companies are currently evaluating gene-editing therapy CTX001 in phase 1/2 studies targeting both indications and have reported encouraging preliminary results.

Another indication that could be a big winner for Vertex by the end of this decade is alpha-1 antitrypsin deficiency (AATD). Like CF, AATD is a rare genetic disease caused by misfolding proteins. Vertex has two experimental AATD drugs in early stage testing. My hunch is that the company's CF expertise combined with the similarity between AATD and CF could boost the odds of success for this program.

Vertex also has an early stage program targeting APOL-1 mediated kidney diseases. The biotech hopes to advance a drug to phase 2 testing this year. If all goes well, this could be yet another new arena for Vertex to dominate by the end of the decade.

Then there's the huge potential game-changer. Vertex acquired privately held Semma Therapeutics for $950 million last year. Semma is developing a drug that could cure type 1 diabetes.

Semma's approach is to turnpluripotent stem cells into islets that produce insulin in the needed amounts to keep blood sugar levels in check. This program is in its very early innings right now. So far, Semma has conducted promising lab tests but hasn't initiated any clinical studies in humans.

Curing type 1 diabetes presents an enormous opportunity for Vertex. Over 1.5 million people have type 1 diabetes in the U.S. alone. Vertex has had its eye on several companies in recent years that have made progress in addressing issues related to islet transplantation to treat type 1 diabetes. The big biotech thinks that Semma has a solution and is confident enough about its prospects to write a really big check to acquire the small drugmaker.

Will Vertex really have successful drugs on the market that target five or more rare genetic diseases in addition to more common indications like pain and type 1 diabetes 10 years from now? There's no way to know for sure. Many promising early stage programs fail along the way.

However, there are some things we can be certain about with Vertex. It claims a commanding lead in CF. It has the expertise needed to develop therapies targeting other rare genetic diseases. It has plenty of money to continue investing in research and development and acquisitions. And it has multiple shots on goal. Not all of them have to pan out for Vertex to win.

My view is that Vertex is the best biotech stock on the market right now. I think that it's future looks really bright.

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Where Will Vertex Pharmaceuticals Be in 10 Years? - Motley Fool

Washington: A recent study has linked a variety of blood cancers to a dangerous gene mutation that triggers a chain of biological events that hinders the pathway through which DNA sends instructions to the rest of a cell.

This phenomenon has been revealed through research carried out by biologists at Cold Spring Harbor Laboratory (CSHL) in collaboration with an oncologist at Memorial Sloan Kettering Cancer Center (MSKCC).

The deadly chain of events begins with RNA splicing, a process that converts messages from DNA into instructions for making proteins in a cell. Errors in RNA splicing can result in poorly formed proteins that are unable to do their job.

The CSHL-MSKCC team found that in blood cancers, a process associated with splicing called Nonsense-mediated mRNA decay (NMD) is excessively active. After splicing converts DNA messages, the NMD process normally serves as quality control, destroying messages that contain mistakes before a broken protein is made.

The Krainer lab at CSHL determined that when the gene called SRSF2 is mutated, NMD destroys many more messages, including some that have not been likely targets of NMD before. Messages that are important for healthy blood cell production are one of these new targets.

The result of excessively active NMD is less healthy blood cells and more sickly or immature cellsa hallmark of blood cancer.

RNA splicing factor mutations are seen in virtually all forms of leukaemia, both chronic as well as acute myeloid leukaemias and also even chronic lymphocytic leukaemia, said Omar Abdel-Wahab, M.D. at MSKCC. Im a hematological oncologistI think a lot about blood cancerso this caught my attention right away.

Scientists have seen other cancers manipulate NMD into protecting solid tumours. However, the CSHL-MSKCC discovery, published in Genes and Development, is the first evidence of NMD contributing to blood cancer conditions.

To stop the mutated SRSF2 gene from affecting NMD, the researchers experimented with a technique called antisense oligonucleotide (ASO) therapy. As shown by CSHL Professor Adrian Krainer in past works, ASO therapy has been effective in combating other diseases resulting from defective RNA splicing.

The next step will be to test many ASOs in animals, perfecting the teams approach until it is ready for the clinic.

Before this collaboration, I knew very little about splicing factors or this process of NMD. Conversely, Adrians lab didnt have much disease-specific experience with blood cancers, so its been a synergistic experience, Abdel-Wahab added. By combining forces, weve really been able to address what weve learned.

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Deadly gene mutation leading to blood cancer - The Siasat Daily

OmniSpirant Limited are a leading European biotech startup company with ambitions to change the paradigm of treatment for respiratory disease. Chronic Obstructive Pulmonary Disease (COPD) is an umbrella term used to describe progressive lung diseases including emphysema, chronic bronchitis, and refractory (non-reversible) asthma. This disease is characterised by increasing breathlessness, frequent chest infections and persistent wheezing. COPD currently cannot be cured or fully reversed.

This debilitating disease today has a solution, developed by OmniSpirant, as we explain below. Until now, the current COPD therapeutics market has lacked any effective disease modifying treatments and the clinical stage pipeline is weak, given the massive disease prevalence; COPD is arguably the disease with the most severely unmet medical and patient needs.

Smoking is indeed the primary cause of this devastating disease, but 15-20% of COPD cases are due to exposures to occupational dust, chemicals, vapours or other airborne pollutants in the workplace. Air pollution is also a likely and underappreciated driver of the growth of the disease and declining lung function in COPD is strongly associated with ageing.

COPD affects up to 500 million patients globally and is the worlds fourth leading cause of death. This dire situation is projected to worsen with COPD becoming the third leading cause of death globally in 2030 and the leading cause of hospitalisations in the industrialised world. COPD is classified as a priority disease by the EU and WHO as it is the only leading cause of death that is rising in prevalence globally. The burden of this chronic respiratory disease is growing rapidly, fuelled by an ageing demographic, persistent smoking habits, and air pollution.

A recent study has estimated that air pollution may be a factor in as many as 47,000 COPD deaths per annum across the 28 EU Member States. Epidemiologic studies have found a measured prevalence of COPD in Europe of between 4% and 10% of adults (European COPD Coalition). However, COPD is widely undiagnosed and untreated especially in its early stages, so the actual prevalence may be higher. New therapies that can slow disease progression desperately need to be developed.

The disease costs tens of billions of euros annually to healthcare payers in reimbursement for largely ineffective pharmacological and medical interventions. In the key United States market, COPD is responsible for USD $72bn (~65bn) per year in direct healthcare expenditures. In the EU, estimated spending on inpatient, outpatient and pharmaceuticals exceeds 10bn per year and productivity losses are estimated at 28.5bn year.

The disease also causes an estimated 300,000 premature deaths in the EU annually (European Respiratory Society). These startling figures are forecast to rise dramatically as the disease prevalence is set to rise sharply.

Current COPD treatments do not include an effective disease modifying therapy which can reduce the exacerbation of symptoms and/or slow down COPD from progressing and worsening. State of the art therapies for COPD consists of combinations of oral, injected or inhaled bronchodilators, anti-muscarinics, corticosteroids, anti-inflammatories, and antibiotics, all of which are used to treat symptoms and reduce exacerbations of COPD with only modest results.

Except for a small minority of Alpha one Antitrypsin (AAT) deficient COPD patients (five in 10,000 carry the mutation responsible for AAT deficiency on both chromosomes), there are no available therapies which modulate disease progression. AAT is a protein that protects the lungs from the destructive actions of common illnesses and exposures, particularly tobacco smoke.

Furthermore, the COPD pipeline is also devoid of disease modifying treatments. The COPD pipeline is full of incremental advances on existing mainstay therapies which merely treat disease symptoms and do not target the root causes of the disease. There are a few innovative therapies in development but a small molecule or biologic agent such as a monoclonal antibody (or even combinations of several of these agents) are highly unlikely to provide a curative or even therapeutically useful intervention in a complex disease like COPD.

OmniSpirant believe that the solution to the COPD epidemic can be found in the new era of advanced therapeutics by combining several technological advances in the fields of cell culturing, genetic engineering and their innovative exosome technology platform. OmniSpirant are developing inhaled bioengineered exosome therapeutics, delivered by a tailored aerosol delivery method based on vibrating mesh nebuliser technology.

In the first instance, the presenting problem is that patients have established lung damage and an increased risk of developing lung cancer (independent of smoking history). OmniSpirant believe that microRNA/mRNA engineered stem cell exosomes can provide powerful anti-inflammatory and regenerative effects and also reduce the risk of patients developing lung malignancies.

Exosomes are naturally produced by cells and recent research highlights the vast potential of stem cell exosomes as transformative regenerative medicines. Stem cell exosomes have shown great regenerative potential in animal models of COPD by stimulation of repair mechanisms and reversing damage to the lung. Stem cells have also shown some promising results in COPD clinical trials.

Donor (Allogeneic) MSCs delivered intravenously in repeat-dose clinical trials for COPD (Prochymal Osiris Therapeutics) were found to be safe and well tolerated and reduced systemic inflammation, but no significant improvements in lung function were observed. We believe that the use of exosomes, as the therapeutic essence of stem cells, delivered by the inhaled route of administration will be capable of far greater efficacy by delivering far higher doses of exosomes directly to the affected lung tissues than intravenous delivery while typically only requiring about 1% of the overall dose. Furthermore, our exosomes will have enhanced delivery (via proprietary surface engineering) and are also bioengineered to enhance efficacy.

OmniSpirants novel technology platform is capable of delivering high doses of these exosomes across the mucus barrier and through cell membranes to deliver the therapeutic payload directly into the diseased lung cells. Such delivery has proven problematic for competing gene transfer technologies because the mucus in the lungs is a barrier that traps the carriers used to deliver gene therapies such as nanoparticles and viral vectors. These trapped gene therapy carriers are mostly cleared from the mucus layer before they can penetrate into the underlying cells and introduce their genetic cargo.

The use of exosomes overcomes other issues associated with viral and non-viral vectors which include the generation of therapy-inactivating host immune responses and poor ability to cross cell membranes. Furthermore, traditional gene transfer vectors may be immunogenic and elicit adverse inflammatory responses.

OmniSpirants solution is a proprietary method of surface engineering exosomes so they can efficiently penetrate the protective mucus barrier and enter into the underlying cells. These stem cell exosomes are therapeutic (regenerative, anti-inflammatory, antimicrobial and antifibrotic), non-immunogenic, and can be tailored via genetic modification of the parent stem cells to create ideal inhaled gene therapy vectors for any lung disease.

The surface engineered exosomes have demonstrated 100% mucus penetration and target cell uptake in the gold standard in vitro model (well-differentiated bronchial epithelial cells in air liquid interface culture), which is game changing compared with the state of the art viral vectors which can achieve only 30% of cells at best. We believe that the enhanced delivery of stem cell exosomes can translate the promising regenerative effects witnessed in various animal models of inflammatory lung diseases into the clinic.

To treat COPD, our approach is to genetically modify the stem cells so that they produce exosomes carrying carefully selected nucleic acids which are tailored for treating the underlying causes of COPD, which has been linked to gene expression and cellular senescence. The genetic element to COPD runs much deeper than just AAT deficiency.

Abnormalities in scores of genes have been clearly shown to increase or decrease the risk of developing COPD and perturbed gene expression is apparent in hundreds of disease associated genes. MicroRNAs (miRNAs) are a recently discovered class of non-coding RNAs that play key roles in the regulation of gene expression and more than 2,000 miRNAs have been identified in the human genome to date. The fact that each miRNA has the ability to target multiple genes within a pathway makes miRNAs one of the most abundant classes of regulatory genes in humans, regulating up to 30% of human protein coding genes.

MiRNAs have been widely shown to be dysregulated in the affected lung tissues of COPD patients which makes an inhaled gene therapy a highly promising approach for treating COPD. Such a gene therapy could effectively modulate the disease altered microRNAs (and their target genes) to halt or even reverse the disease. Recent advances in cell culturing techniques, isolation of exosomes and proprietary cell engineering technologies hold the promise to bring this therapy to the afflicted masses. The BOLD project estimates that there are currently 36 million patients in the EU and US alone with GOLD Stage 2 disease or higher; we need to act quickly as this figure is set to rise dramatically in the coming decade.

OmniSpirant are currently seeking investors or partners to fund the preclinical development of OS002 and anticipate that clinical studies can be initiated within approximately four years an impactful investing opportunity as the rising prevalence of COPD means that by 2030 there may be over 4.5 million deaths annually worldwide and COPD is predicted to be the leading cause of hospitalisation. Lets work together to change those grim statistics.

OmniSpirant and their consortium partners were awarded a 9.3m Irish government grant award (Disruptive Technologies Innovation Fund) in December 2019 to advance the development of their novel COPD gene therapy.*

OmniSpirant have received funding from Horizon 2020, ReSpire, Grant agreement ID: 855463 and have been accelerated by EIT Health.

Gerry McCauleyCEOOmniSpirant Ltd+353 876306538gmccauley@omnispirant.comwww.omnispirant.com

Please note, this article will appear in issue 12 ofHealth Europa Quarterly, which will be available to read in February 2020.

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Innovation in the treatment of COPD - Health Europa

Austin was three years old and Max was a newborn when their mother, Jenn McNary, learned they had a rare genetic condition called Duchenne muscular dystrophy. The doctor painted a grim picture: Her boys would stop walking by age 12 or 13 and, shortly thereafter, they would require nighttime ventilation. They would each need a tracheotomy, a feeding tube, or both by their late teens. Death would come a few years later.

It hasn't worked out that way, thanks to two new drugs that became available after the boys' 2002 diagnosis. Exondys 51, a medicine that targets their genetic mutation, slows the disease's progression, and Emflaza, a corticosteroid, mitigates some of its symptoms. Thanks to these treatments, Austin now attends college and interns at a biotech company. Max attends his local high school in Newton, Massachusetts. Both are able to get around in wheelchairs, and neither needs ventilation. McNary just rented an apartment for her boys because they can function on their own with the help of an aide.

By all accounts, the drugs have been transformative, McNary said. But, she added, her boys "aren't going to be cured," and extending and improving their life for an unknown period of time comes at a high price. Emflaza came onto the market in 2017 at an annual cost of $65,000. Exondys 51 appeared in 2016 at $748,500. Neither of the drugs will help the young men walk again and, in the eyes of some U.S. health economists, the drugs are not worth the price.

That's why McNary hates the quality-adjusted life year (QALY, pronounced "qua-lee"), an economic calculation that attempts to quantify the value of a medical intervention, based in part on the quality of life it bestows on recipients.

First developed by U.S. economists in the late 1960s and early 1970s, variations of the QALY have been used for years by governments around the world to help determine what treatments citizens can obtain under public health care. In America's free-market health care system, however, QALY calculations have largely been avoided. As McNary and others like her are finding out, that's starting to change.

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As policymakers and insurance companies scramble to get a handle on skyrocketing health care costs, they are promoting the idea of paying for value. In this view, drugs designated as higher-value should be prioritized over lower-value treatments. But this raises a thorny question: Who gets to define "value"? Health economists and insurance companies who seek to use limited health care dollars judiciously? Or patients, parents, and doctors who want to receive the best health care for their situation?

Because the quality-adjusted life year threatens her sons' ability to get the medicine they need, McNary is clear about her answer. "To me, the QALY is a measurement that says that keeping my sons alive by providing incremental benefit but not totally curing them is never going to be valuable," McNary said. "Just mull that around in your head if you are less than perfect, you are worth less money."

* * *

In QALY math, a year of perfect health is equal to 1; death equates to 0. The value of other health states is derived from surveys of patients, caregivers, or the general public. Paralysis might be valued at .35, for example, and mild Alzheimer's disease at .52, depending on the survey. Those numbers can then be plugged into a formula that allows the relative cost-effectiveness of treatments to be compared to identify the best buys.

Economists developed the QALY concept more than 40 years ago to address a fundamental question: "Where should we spend whose money to undertake what programs to save which lives with what probability?' Richard J. Zeckhauser and Donald Shepard asked in a 1976 article describing the basic QALY formula. The next year, as U.S. health care spending topped $120 billion, Harvard health policy professor Milton C. Weinstein and his colleague, cardiologist William B. Stason, sounded an alarm bell. "It is now almost universally believed that the resources available to meet the demands for health care are limited," they wrote in the New England Journal of Medicine. "We, as a nation, will have to think very carefully about how to allocate the resources we are willing to make available for health care."

Their article cited by other authors more than a thousand times in the past four decades pointed out that resources were already being allocated by millions of individual decisions: hospitals rationing beds where they didn't have room for all patients, for example, and insurers agreeing to pay for some tests and treatments but not for others. Such decisions, they argued, were often inconsistent with the "societal objective of deriving the maximum health benefits from the dollars spent," an objective that could be achieved by putting the QALY to work.

In the intervening decades, some countries the United Kingdom, the Netherlands, and Sweden, for example have embraced QALY-based evaluations. In the U.K., cost-effectiveness studies are used, in part, to determine which therapies the National Health Service will provide for residents. The publicly-funded health system does not cover Orkambi, the first cystic fibrosis treatment that targets the cause of the disease, for example, because its cost-per-QALY far exceeds the U.K. cost-effectiveness threshold.

In the United States, however, QALY-based assessments have not gained traction until recently. "Perhaps the general reason is that we as patients and our providers don't want to be limited in the treatment options available," said Louis P. Garrison Jr., an economist in the Pharmaceutical Outcomes Research and Policy Program at the University of Washington.

In fact, QALY-based cost-effectiveness reviews are so controversial that the federal government has repeatedly quashed their use. In 1992, the Department of Health and Human Services rejected Oregon's attempt to use QALY-based cost-effectiveness assessments to determine what services its Medicaid program would cover. In 2010, as part of the Patient Protection and Affordable Care Act, Congress prohibited the use of QALYs by the Medicare program. It also banned the federal Patient-Centered Outcomes Research Institute from using QALY thresholds in its assessments of comparative treatments.

* * *

A QALY Primer

A QALY reflects quality of life and length of life. A year in "perfect health" is worth 1 QALY, death is worth 0 QALYs, and other health states fall between 0 and 1. The amount that a drug lengthens or improves the quality of life is calculated as "QALYs gained." The cost of getting a certain level of health improvement is the "cost per QALY gained," shown here for several interventions targeting asthma.

But more than half of U.S. residents are covered by private insurance companies, which are not prohibited from using QALY-based assessments to decide which medicines they will cover for their members. Traditionally, however, private insurers have generally not used QALYs explicitly in their decisions about what tests and treatments they will pay for, according to a recent report by the National Council on Disability. Instead, when major U.S. insurers decide to limit access to a given medication, they usually cite insufficient data to justify its use in a given situation.

Indeed, until recently, U.S. insurers did not have a source for QALY-based cost-effectiveness reports. That began to change in 2014, when the Institute for Clinical and Economic Review, a nonprofit research organization based in Boston, turned its attention to high-cost drugs. Founded in 2006 as a research project based at Harvard Medical School, ICER initially issued reports on broad topics such as obesity management and palliative care. But when Sovaldi, a drug for deadly hepatitis C, came on the market at the then-shocking price of $84,000 for a 12-week course of treatment, ICER kicked into action. Despite the high price, its assessment found that Sovaldi is cost-effective for some patients. Insurers took notice.

Since then, the organization has been churning out several drug-assessment reports each year. Each report includes its opinion of how much the drug is worth; drugs priced higher than that are deemed not cost-effective. ICER has no authority over anyone, but its reports have become popular reading for U.S. insurers. "If there is a drug of note being approved by the FDA, there's also likely going to be an ICER assessment of that drug that can factor into their decision-making," said David Whitrap, the research organization's vice president of communications and outreach.

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U.S. health care spending has risen dramatically since Weinstein and Stason expressed concern in the mid-1970s. In 2016, the U.S. spent nearly 18 percent of its gross domestic product on health care, far outstripping the average of 11 percent for 10 other high-income nations. High prices for prescription drugs is one reason. "We're seeing price tags now of $1 million, $2 million," said Seema Verma, administrator for the federal Centers for Medicare and Medicaid Services, at a conference recently. "That's completely unsustainable for the system."

That's why Peter Neumann, director of the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center, said cost-effectiveness analyses are needed more than ever. But there are many reasons for the resistance, Neumann and his co-authors wrote in the Journal of the American Medical Association, including "an inclination on the part of many individuals in the United States to minimize the underlying problem of resource scarcity and the consequent need to explicitly ration care."

Further, Ari Ne'eman, a disability rights activist and consultant to Partnership to Improve Patient Care, a coalition of advocacy groups, said the idea that two health conditions can be numerically compared to one another is simply wrong. "Proponents of the QALY will say it is this mathematically perfect measure that gives us a superpower ability to compare depression drugs to cystic fibrosis drugs to cancer drugs even though all of those drugs do different things because it lets you translate them back to this common measure," he said. "Our concern is that when you engage in that process of translation, you lose some significant nuance in terms of the amount of benefit that's being delivered."

The Partnership argues the QALY calculation is flawed because it assumes quality of life can be captured by a certain number, despite the fact that different surveys arrive at different numbers. For example, a 2006 quality-of-life survey in the U.S. assigns blindness/low vision as .69 on the 0-to-1 scale, while a 2011 survey in the U.K. gives blindness/low vision a score of .78.

Beyond the methodological issues, Ne'eman said, "there are all kinds of ethical problems with it." People with disabilities and chronic medical conditions may value a treatment that offers an incremental improvement in the quality or length of their lives, even though the "QALYs gained" are less than those for a treatment that prevents the loss of perfect health.

Former U.S. Representative Tony Coelho, a Democrat from California and a primary author of the Americans with Disabilities Act, is the Partnership's chairman. "I worry that more focus is being given to what is most cost-effective for the 'average patient' than creating a system that works for each individual patient," he wrote in 2018. "The medication I take for epilepsy isn't 'high value' for every patient. But it's the only one that works for me."

That's why, Ne'eman said, cost-effectiveness analyses must consider the fact that not all patients respond the same way to a drug. Some patients need drugs that aren't deemed cost-effective for the general population. It's important to account for that, he said. "Otherwise we're giving insurers a tool to deny care to people who need it."

When an insurer decides to cover a specific drug, that decision affects everybody who pays into the insurance pool. Michael Sherman, chief medical officer for the insurer Harvard Pilgrim Health Care, uses the example of a gene therapy that costs $1 million to treat a child who will die without it. Under the ACA, families will hit their out-of-pocket maximum at about $16,000, and many health plans have out-of-pocket maximums far below that. "The rest of that million dollars is going to be paid by everyone else that's the way it works in insurance," he said. When insurers see that kind of unanticipated budget impact, they raise premiums or out-of-pocket cost-sharing for everyone.

Like other proponents of the QALY, Neumann sees it as an imperfect but useful tool. "Any single number is never going to capture everything," he said.

"The problem is, if you're not going to use QALYs, what are you going to use?"

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That's an urgent question, particularly now when there is a huge pipeline for rare-disease therapies, often called orphan drugs. By 2024, orphan drug sales are expected to reach $242 billion.

In the U.S., a rare disease is defined as one that affects fewer than 200,000 people. While these conditions are individually rare, in the aggregate, an estimated 25 to 30 million Americans that's about one in 10 live with a rare disease. Most rare diseases affect children, and many are fatal or disabling.

Historically, drugmakers spent little effort developing treatments for rare diseases, but that changed with the passage of the Orphan Drug Act of 1983, which provides tax credits and a seven-year marketing exclusivity to companies that develop rare-disease treatments. Hundreds of such treatments have won FDA approval in recent years, with more than 560 medicines in the works.

Those treatments are generally expensive. On average, the per-patient cost for orphan drugs in the U.S. is almost 4.5 times more than for non-orphan drugs.

In the two decades ending in 2017, the average annual cost for orphan drugs was $123,543, based on the price at the time the drug launched, compared to $4,961 for traditional drugs. For Duchenne alone, more than 30 orphan therapies are in development. None of them are going to cure patients, McNary said. But she hopes new treatments, generally used in combination, will help her sons live longer, healthier lives and completely change the disease trajectory for younger patients whose disease has not yet progressed as far.

The barrier she worries about is cost-effectiveness analysis. In August, the Institute for Clinical and Economic Review published its assessment of treatments for Duchenne, which affects about 400 to 600 boys born in the U.S. each year. Emflaza, the corticosteroid, appears to be as good as or better than prednisone, another corticosteroid approved to treat the disease, but it would need a price cut of at least 73 percent to be considered cost-effective.

Exondys 51 approved by the FDA for about 13 percent of the Duchenne population got a worse review. In the clinical trials used to seek FDA approval, no clinical benefit, including motor function improvement, was demonstrated. (The FDA approved the drug because some of the patients treated with Exondys 51 had a slight increase in dystrophin levels in skeletal muscle.) In light of that, Exondys 51 was not deemed cost-effective at any price.

But Jenn McNary said the drug works for her sons. Austin, who was not eligible for the Exondys 51 clinical trial, stopped walking at age 10. Max got in the trial and started taking the drug at age 9."They have the same mutation, they have been raised by the same mother, so one would expect they would progress similarly," she said. "But Max walked until he was 17."

Austin was already in a wheelchair when, at age 15, he started taking Exondys 51. He regained some upper-body strength that changed his life, according to his mother. "He's able to use a urinal on his own, which makes is possible for him to have a job and to go to college without an aide," she said.

The Medicaid program in Massachusetts, where the McNarys live, won't pay for Max's Duchenne therapies. For the time-being, the drugmakers are giving him the drugs free through a patient-assistance program. Austin, because he's enrolled in college, is eligible for student coverage through Blue Cross Blue Shield of Massachusetts. The insurer, by policy, does not cover Exondys 51 for patients who can no longer walk. His mother appeals the insurance denial. Every six months, she sends a video of Austin in action, along with a letter from his doctor and so far, his medicines have been covered.

The payers made their coverage policies before the quality-adjusted life year analysis was published. Now, insurers who have been covering the Duchenne treatments have an independent analysis with which to rethink that decision.

For now, there is one thing that QALY supporters and critics agree on. "Very promising drugs are coming, and they're going to be very expensive," said Neumann, the health economist at Tufts. Increasingly, the QALY appears poised to influence how American health care money is spent.

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Lola Butcher is a health care business and policy writer based in Missouri.

This article was originally published on Undark. Read the original article.

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Is the medication you're taking worth its price? - Salon

In a second research report published this year so far, investigators found that the U.S. Food and Drug Administration (FDA) is approving drugs faster than ever. Unfortunately, it appears that the agency is also approving those drugs on less data and weaker evidence.

The first study published in the journal SSRN was by researchers at Harvard University, the University of Texas at Dallas, and the Massachusetts Institute of Technology (MIT). It questioned if the FDA and other regulatory agencies worldwide dont rush certain approvals, particularly at the end of the year in a kind of desk-clearing activity.

The report notes, In the United States, the number of December drug approvals is roughly 80% larger than in any other month. Similar approval spikes occur at the end of each calendar month. Additionally, approvals spike before holidays, such as before Thanksgiving in the United States and the Chinese New Year in China (but not vice versa).

And more troubling is that there appears to be a correlation with more problems with these drugs. Lauren Cohen, a professor of finance and entrepreneurial management at Harvard Business School and one of the authors, told the Wall Street Journal, We see about twice as many adverse effects.

The second study appeared in the journal JAMA Network and was conducted by researchers with Harvard Medical School. The lead author, Jonathan Darrow, a lawyer with the medical schools Program on Regulation, Therapeutics and Law, told NPR, There has been a gradual erosion of the evidence thats required for FDA approval. He points out that patients and physicians should not expect that new drugs will be dramatically better than older ones.

The study notes that about half of recent drug approvals were built on a single pivotal clinical trial. Typically, two pivotal, Phase III trials were the norm. In addition, the study says that surrogate measures, which are utilized as stand-ins for presumed patient benefits, has grown. For example, in oncology drugs, what most patients would want are improvements in survival after receiving treatment. But some cancer trials use a surrogate measure of tumor shrinkage. Ideally, both would be taken into consideration.

Darrow and his research associates studied FDA approvals, changes in the law and regulations, and how the industry funds agency reviews from 1983 through 2018. They found that the average number of new drug approvals annually grew from 34 in the 1990s to 41 in the 2010s. In the 2000s, it dropped to 25 a year. But now they are increasing. For example, in 2019, the FDA approved 48 new molecular entities and new therapeutic biological products. That doesnt include vaccines, allergenic products, blood and blood products, plasma derivatives, cellular and gene therapy products, or the numerous new indications approved for existing therapies.

Darrow, with Jerry Avorn and Aaron S. Kesselheim, both with the Division of Pharmacoepidemiology & Pharmacoeconomics at Brigham & Womens Hospital, found that faster approvals were related to legislative and regulatory modifications that started in the 1980s. Although there are probably several reasons for those changes beginning in that period, much of it is likely related to the beginning of the HIV epidemic and demands from patient populations and advocates to fund more research and get therapiesany therapiesto market faster.

Just some of those regulations include: the 21st Century Cures Act (2016), which authorized funds for the Precision Medicine Initiative and Cancer Moonshot; the Biologics Price Competition and Innovation Act (BPCIA, 2010), creating an abbreviated pathway for follow-on biologic products; Breakthrough Therapy designation (2012), for drugs that showed substantial improvement over existing therapies; the Hatch-Waxman Act (1984), which created an Abbreviated New Drug Application pathway for drugs approved after 1962; and the Pediatric Research Equity Act (2003), which required results from pediatric assessments to be submitted as part of New Drug Applications (NDAs).

Congress also passed the Prescription Drug User Fee Act in 1993, and that first year, FDA collected $29 million in fees. In 2018, the agency brought in $908 million in PDUFA fees. Or, as the study notes, industry fees were responsible for about 80% of the money spent on FDA employee salaries for drug reviews.

There is some concern about the incentives that this created within the FDA, Darrow told NPR. And whether it has created a culture in the FDA where the primary client is no longer viewed as the patient, but as the industry.

Another factor that is related, is the concept of me-too drugs. These are basically drugs that are very structurally similar to approved drugs, with only minor differences. Thats not necessarily a bad thing, because they need to be at least as good as the drugs already on the market, and generally need to be betteralthough not necessarily by much. Which does mean a number of companies spend time on developing drugs that are only incrementally better than others on the market.

Joshua Sharfstein, former FDA Principal Deputy Commissioner, told NPR that there are more changes needed to ensure drugs are worthwhile for patients. Some of them are really great, and some of them [are] not so great. And a lot of them are very expensive.

Sharfstein is currently a professor at the Johns Hopkins Bloomberg School of Public Health. He wrote an editorial in JAMA that accompanied the newer study. In it, he suggests its time to reevaluate the FDAs expedited approval programs to determine which ones are working and which ones are increasing healthcare costs.

Weve kind of reached a point where it makes sense to pause and see whether we can do things better, Sharfstein said. And I think we can.

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The FDA Is Approving Drugs Faster, But That May Not Be A Good Thing - BioSpace

For Immediate Release: January 28, 2020

This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA has approved four gene therapy products, which insert new genetic material into a patients cells. The agency anticipates many more approvals in the coming years, as evidenced by the more than 900 investigational new drug (IND) applications for ongoing clinical studies in this area. The FDA believes this will provide patients and providers with increased therapeutic choices.

In that spirit, today, the FDA is announcing the release of a number of important policies: six final guidances on gene therapy manufacturing and clinical development of products and a draft guidance, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.

The growth of innovative research and product development in the field of gene therapy is exciting to us as physicians, scientists and regulators, said FDA Commissioner Stephen M. Hahn, M.D. We understand and appreciate the tremendous impact that gene therapies can have on patients by potentially reversing the debilitating trajectory of diseases. These therapies, once only conceptual, are rapidly becoming a therapeutic reality for an increasing number of patients with a wide range of diseases, including rare genetic disorders and autoimmune diseases.

As the regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs, said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research. Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review; including how these products work, how to administer them safely, and whether they will continue to achieve a therapeutic effect in the body without causing adverse side effects over a long period of time.

One of the most important steps the FDA can take to support safe innovation in this field is to create policies that provide product developers with meaningful guidance to answer critical questions as they research and design their gene therapy products.

The six final guidances issued today provide the agencys recommendations for product developers on manufacturing issues and recommendations for those focusing on gene therapy products to address specific disease areas. The six guidance documents incorporate input from many stakeholders and take a significant step toward helping to shape the modern structure for the development and manufacture of gene therapies. The agency is issuing this suite of documents to help advance the field of gene therapy while providing recommendations to help ensure that these innovative products meet the FDAs standards for safety and effectiveness.

The scientific review of gene therapies includes the need to evaluate highly complex information on product manufacturing and quality. In addition, the clinical review of these products frequently poses more challenging questions to regulators than reviews of more conventional drugs, such as questions about the durability of response, and these questions often cant be fully answered in pre-market trials of reasonable size and duration. For some gene therapy products, therefore, although they have met the FDAs standards for approval, we may need to accept some level of uncertainty around questions of the duration of the response at the time of marketing authorization. Effective tools for reliable post-market follow up, such as post-market clinical trials, are going to be key to advancing this field and helping to ensure that our approach fosters safe and innovative treatments.

The draft guidance on interpreting sameness of gene therapy products under the orphan drug regulations provides the FDAs proposed current thinking on an interpretation of sameness between gene therapy products for the purposes of obtaining orphan-drug designation and eligibility for orphan-drug exclusivity. The draft guidance focuses on how the FDA will evaluate differences between gene therapy products when they are intended to treat the same disease. As laid out in the draft guidance and our regulations, the agencys determination will consider the principal molecular structural features of the gene therapy products, which includes transgenes (the transferred gene) and vectors (the vehicle for delivering the transgene to a cell).

With the large volume of products currently being studied, gene therapy product developers have asked the agency important questions about orphan-drug designation incentives to develop products for rare diseases with very small patient populations. The draft guidance has potential positive implications both for product developers and patients by providing insight into the agencys most current thinking on the sameness of products, and thus, not discourage the development of multiple gene therapy products to treat the same disease or condition. For patients, this policy could help lead to the development and approval of multiple treatments, creating a more competitive market with choices. We encourage stakeholders to provide their comments.

In sum, these policy documents are representative of efforts to help advance product development in the field of gene therapy. We will continue to work with product innovators, sponsors, researchers, patients, and other stakeholders to help make the development and review of these products more efficient, while putting in place the regulatory controls needed to ensure that the resulting therapies are both safe and effective. We also encourage developers of new gene therapy products to make full use of our expedited programs available for products intended to address unmet medical needs in the treatment of serious or life-threatening conditions. These programs include breakthrough therapy designation, regenerative medicine advanced therapy designation, and fast track designation, as well as priority review and accelerated approval. Developers should pursue these programs whenever possible to help bring the benefits of important advances to patients as soon as possible. We believe our work will help advance innovations in a way that assures their safety and effectiveness, provides new therapeutic choices to patients and providers and continues to build confidence in this novel and emerging area of medicine.

The FDA is an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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FDA Continues Strong Support of Innovation in Development of Gene Therapy Products - FDA.gov

Aperio Clinical Outcomes, a leading clinical research organization (CRO), announced today that Jonathan Yusi, an expert in the coordination and management of cell and gene therapy clinical trials, has joined the company as Senior Clinical Trials Manager to support their biotechnology clients in the immuno-oncology space.

Yusi has been managing immune-based therapy trials for over seven years. Prior to joining Aperio, he was a program manager for CAR-T studies and oversaw the first CAR-T program at a large CRO. He has provided independent trial management consulting for CAR-T trials, and his expertise has resulted in lasting KOL relationships within the immuno-oncology space. In addition to his adoptive cell therapy knowledge, Yusi brings over 20 years of clinical research experience to Aperio, with a focus on trial logistics, management, and monitoring of targeted and immune therapies in oncology trials.

Says Suzanne Kincaid, Aperios COO and an oncology industry veteran herself, "FDA expects to see over 200 INDs for cell and gene therapies in 2020, so it is imperative that our biotech clients have expertise like Jonathans to manage their trials. He has a strong understanding of the complexities of cell and gene therapy studies and can break down the components for ideal study set-up. We are so excited to have Jonathan help our immuno-oncology clients as they explore these groundbreaking treatments."

"Cell and gene therapy trials are a logistical maze, and one missed endpoint can be catastrophic to the study," says Yusi. "These programs allow me to utilize everything Ive learned about clinical research and oncology, and my medical and scientific background brings an understanding to the science behind the treatments. The bulk of my career has involved oncology trials, so as the treatments have evolved and become more personalized, my experience has evolved as well. These are life-saving breakthroughs, and Im happy to bring this experience to Aperio and our immune-based therapy clients."

About Aperio Clinical Outcomes

In a data driven industry, Aperio remains dedicated to transparency with clients and focused on the most important part of the process: people. Aperio provides full, customizable clinical research services across multiple therapeutic areas, as well as consulting services in Quality Assurance, Strategic Resourcing and Clinical Trial Technology. For more information: http://www.aperioclinical.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200131005540/en/

Contacts

Heather Newbold: +1 919-604-5704heather.newbold@aperioclinical.com

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Aperio Hires CAR-T Trials Expert Jonathan Yusi to Support Cell and Gene Therapy Studies - Yahoo Finance