The Insight Partners analysts forecasts the latest report on Global Gene Therapy Market (Covid-19) Impact and In-Depth Analysis by 2027, according to report; The Gene Therapy Market report covers the overall and all-inclusive analysis of Market with all its factors that have an impact on market growth. This report is anchored on the thorough qualitative and quantitative assessment of the Gene Therapy Market.

The study provides details such as the market share, Market Insights, Strategic Insights, Segmentation and key players in the Gene Therapy Market.

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Some of the Major Market Players Are:

Sangamo Therapeutics, Inc., bluebird bio, Inc., uniQure N.V., AveXis, Inc., Vineti, Solid Biosciences., Spark Therapeutics, Inc., CHIMERON BIO, RENOVA THERAPEUTICS, HORAMA S.A., and more.

MARKET INTRODUCTION

Gene therapy is the introduction of DNA into a patient to treat a genetic disease or a disorder. The newly inserted DNA contains a correcting gene to correct the effects of a disease, causing mutations. Gene therapy is a promising treatment for genetic diseases and also includes cystic fibrosis and muscular dystrophy. Gene therapy is a suitable treatment for infectious diseases, inherited disease and cancer.

Key Benefits

MARKET DYNAMICS

The growth of the gene therapy market is regulated due to various reason which includes the rapid involvement of synthetically modified gene to treat various diseases, it helps in designing the personalized medicine, rise in the research and development of the gene therapy among the others. The gene therapy requires less doses of medicines and is one time treatment, this factor is likely to show growth opportunity for gene therapy market in coming near future.

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

Note The Covid-19 (coronavirus) pandemic is impacting society and the overall economy across the world. The impact of this pandemic is growing day by day as well as affecting the supply chain. The COVID-19 crisis is creating uncertainty in the stock market, massive slowing of supply chain, falling business confidence, and increasing panic among the customer segments. The overall effect of the pandemic is impacting the production process of several industries. This report on Gene Therapy Market provides the analysis on impact on Covid-19 on various business segments and country markets. The reports also showcase market trends and forecast to 2027, factoring the impact of Covid -19 Situation.

Our 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 reports cover key developments in the Gene Therapy Market as organic and inorganic growth strategies. Various companies are focusing on organic growth strategies such as product launches, product approvals and others such as patents and events. Inorganic growth strategies activities witnessed in the market were acquisitions, and partnership & collaborations. These activities have paved way for the expansion of business and customer base of market players.

The report analyses factors affecting the Gene Therapy Market from further evaluates market dynamics affecting the market during the forecast period i.e., drivers, restraints, opportunities, and future trend. The report also provides exhaustive PEST analysis for all five regions namely; North America, Europe, APAC, MEA, and South America after evaluating political, economic, social and technological factors affecting the Gene Therapy Market in these regions.

Moreover, the report entails the estimate and analysis for the Gene Therapy Market on a global as well as regional level. The study provides historical data as well as the trending features and future predictions of the market growth. Further, the report encompasses drivers and restraints for the Gene Therapy Market growth along with its impact on the overall market development. In addition, the report provides an analysis of the accessible avenues in the market on a global level.

REGIONAL FRAMEWORK

The report provides a detailed overview of the industry including both qualitative and quantitative information. It provides an overview and forecast of the global Gene Therapy Market based on various segments. It also provides market size and forecast estimates from the year 2018 to 2027 with respect to five major regions. The Gene Therapy Market by each region is later sub-segmented by respective countries and segments. The report covers the analysis and forecast of 18 countries globally along with the current trend and opportunities prevailing in the region.

The Insight Partners dedicated research and analysis team consist of experienced professionals with advanced statistical expertise and offer various customization options in the existing study.

Purchase a copy of Gene Therapy Market research report @ https://www.theinsightpartners.com/buy/TIPHE100001165/

Major Features of Gene Therapy Market Report:

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Gene Therapy Market Growth Opportunities Created by Covid19 Outbreak. Prominent Players : Vineti, Solid Biosciences., Spark Therapeutics, Inc.,...

Recommendation and review posted by Bethany Smith

AUSTIN, Texas--(BUSINESS WIRE)--Genprex, Inc. (Genprex or the Company) (Nasdaq: GNPX), a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes, today announced that it is scheduled to join the U.S. broad-market Russell 3000 Index when FTSE Russell, a leading global index provider, reconstitutes its 2020 indices after the markets close on Friday, June 26, according to a preliminary list of additions posted on their website on June 5.

The Russell 3000 Index includes the 3,000 publicly traded companies on the Nasdaq and NYSE exchanges with the largest market capitalizations. FTSE Russell determines membership for its Russell indexes primarily by objective market-capitalization rankings and style attributes (i.e. growth or value). Each June, the Russell 3000 index is reconstituted to reflect market capitalization changes over the prior year. This closely watched market event impacts more than $9 trillion in investor assets benchmarked to or invested in products based on the Russell U.S. indices.

The selection of Genprex for the Russell 3000 Index will add to the awareness of our company among institutional investors, money managers and index funds, as well as highlight to them our suitability as an investment, said Rodney Varner, Genprexs Chairman and Chief Executive Officer. This inclusion indicates that our leadership in developing gene therapies is resonating with investors. It comes at a time when we are preparing to initiate our Phase I/II clinical trial to evaluate our lead drug candidate, Oncoprex, in combination with AstraZenecas Tagrisso for the treatment of non-small cell lung cancer (NSCLC) and preparing to file our IND to initiate a clinical trial of Oncoprex in combination with Mercks Keytruda in NSCLC. We believe our inclusion in the Russell 3000 Index is yet another significant milestone for us, as it will further increase our exposure with a broader group of institutional investors.

In January 2020, Genprex was awarded U.S. FDA Fast Track designation for use of Oncoprex combined with Tagrisso for the treatment of NSCLC patients with EGFR mutations whose tumors progressed after treatment with Tagrisso alone. Genprex also signed an exclusive license agreement earlier in 2020 with the University of Pittsburgh for a preclinical diabetes gene therapy candidate that has the potential to cure Type 1 and Type 2 diabetes. Additionally, the Company has significantly strengthened its balance sheet in 2020 and had more than $23 million in cash on its balance sheet at the end of the first quarter of 2020, providing a substantial runway for it to execute on its clinical plans, conduct additional research and development, and cover general corporate expenses.

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes. Genprexs technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to in-license and develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. The Companys lead product candidate, Oncoprex, is being evaluated as a treatment for non-small cell lung cancer (NSCLC). Oncoprex has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. Oncoprex has also been shown to block mechanisms that create drug resistance. In January 2020, the U.S. Food and Drug Administration granted Fast Track Designation for Oncoprex immunogene therapy for NSCLC in combination therapy with osimertinib (AstraZenecas Tagrisso). For more information, please visit the Companys web site at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effect of Genprexs product candidates, alone and in combination with other therapies, on cancer and diabetes, regarding potential, current and planned clinical trials, regarding the Companys future growth and financial status and regarding our commercial partnerships and intellectual property licenses. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of the effect of our product candidates, alone and in combination with other therapies, on cancer; the timing and success of our clinical trials and planned clinical trials of Oncoprex, alone and in combination with targeted therapies and/or immunotherapies, and whether our other potential product candidates, including our gene therapy in diabetes, advance into clinical trials; the success of our strategic partnerships; the timing and success of obtaining FDA approval of Oncoprex and our other potential product candidates including whether we receive fast track or similar regulatory designations; costs associated with developing our product candidates and whether patents will ever be issued under patent applications that are the subject of our license agreements. These and other risks and uncertainties are described more fully under the caption Risk Factors and elsewhere in our filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Read the rest here:
Genprex Scheduled to Join Russell 3000 Index - Business Wire

Recommendation and review posted by Bethany Smith

Expansions enable customers to bring transformative medicines to market faster

NEEDHAM, Mass., June 8, 2020 /PRNewswire/ --For biopharma companies seeking increased development and manufacturing capacity for vaccines and therapies, including new COVID-19-related programs, Thermo Fisher Scientific provided highlights of its new capabilities during the BIO International Convention, June 8-12, now a virtual event at http://www.bio.org/events/bio-digital.

"We can now provide an uninterrupted path from development to commercialization for biopharma companies, small to large, in geographies worldwide and across vaccines, antivirals and other therapies," said Mike Shafer, senior vice president and president, pharma services, Thermo Fisher Scientific. "Through our recent strategic initiatives, we are delivering to our customers a powerful combination of expertise, flexibility and scale that allows us to be the partner they start with and stay with."

Earlier this year, the company announced plans to invest in new capabilities and capacityfor biologics, cell and gene therapies and drug product development and commercialization. For example, to support demand for gene therapies, Thermo Fisher will be doubling its viral vector manufacturing capacity with a new manufacturing sitein Plainville, Mass. Construction of the 290,000-square-foot facility will be complete in 2022 and complements the company's recent expansions in Lexington and Cambridge, Mass., and Alachua, Fla. This week, STAT News will feature, "The STAT Guide to viral vectors, the linchpin of gene therapy," which covers the issues and considerations in engineering and manufacturing viral vectors.

In addition, through a new strategic partnership with CSLLimited,Thermo Fisher will support CSL's product portfolio through its pharma services network, including drug product development, biologics manufacturing, sterile fill-finish, packaging and clinical trials logistics. Thermo Fisher will also operate CSL's state-of-the-art biologics manufacturing facility in Lengnau, Switzerland, once construction is completed in 2021.

These strategic investments expand Thermo Fisher's pharma services capabilities for its biotech customers working on antiviral therapies, and for pharmaceutical manufacturers scaling up for novel coronavirus vaccines. Thermo Fisher's pharma services business is also supporting approximately 100 COVID-19 customer projects across its global pharma services network from producing promising therapeutics and treatments to ramping up manufacturing, distribution, packaging and logistics for clinical trials of new vaccine candidates.

To learn more, the company will host two webinars at BIO 2020 this week. The first, "Transforming Medicine with Expertise, Flexibility and Scale," will take place June 12 at 1:00 p.m. EDT. Register for the webinar here. In addition, an on-demand BPI Theater presentation, "Leveraging Infrastructure Investments and Innovation to Accelerate Biologics Development," will run throughout the week and can be accessed here.

About Thermo Fisher Scientific Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue exceeding $25 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, improving patient diagnostics and therapies or increasing productivity in their laboratories, we are here to support them. Our global team of more than 75,000 colleagues delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services and Patheon. For more information, please visit http://www.thermofisher.com.

Media Contact Information: Marcia Goff, Thermo Fisher Scientific +1.508.902.7041 marcia.goff@thermofisher.com

Rachel Robbins, Greenough +1.781.249.9490 rrobbins@greenough.biz

View original content:http://www.prnewswire.com/news-releases/thermo-fisher-scientific-highlights-growing-capacity-for-biologics-cell-and-gene-therapy-production-during-bio-2020-301071921.html

SOURCE Thermo Fisher Scientific

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Thermo Fisher Scientific Highlights Growing Capacity for Biologics, Cell and Gene Therapy Production During BIO 2020 - Yahoo Finance

Recommendation and review posted by Bethany Smith

Newswise NEW YORK CITY, NY, andHACKENSACK, NJ, JUNE 8, 2020 As part of the Memorial Sloan Kettering Hackensack Meridian Health Partnership, the two organizations have formed an Immunology Research Collaboration. Through this joint initiative, researchers can apply for funding to support innovative investigations to explore the power of the immune system and ways it may be harnessed to fight cancer.

The three researchers with projects selected in 2020 for funding support over one to two years are:

"Immunotherapy has become an essential pillar of cancer treatment, but much remains to be discovered about the immune system and new ways to take advantage of its power to treat cancer effectively," said Paul Sabbatini, MD, deputy physician-in-chief for clinical research at Memorial Sloan Kettering. "The Immunology Research Collaboration between Memorial Sloan Kettering and Hackensack Meridian Health gives researchers an opportunity to delve deeply into unexplored facets of the immune system, both in the lab and clinic, and speed discoveries that will ultimately contribute to reducing the burden of cancer on our patients, their families, and the world. We are enthusiastic about the potential of these three research projects and look forward to their results."

"While immunotherapy is revolutionizing cancer treatment, it benefits are not always sustainable over the long term," noted Andre Goy, MD, MS, chairman and executive director of John Theurer Cancer Center and physician-in-chief of the Hackensack Meridian Health Oncology Care Transformation Service. "The work of these investigators will expand our knowledge of the immune system and glean new insights which may lead to novel immunotherapeutics that are more powerful and more durable than those we are using today. These projects capture the collaborative spirit of this initiative and could have a significant impact on patient outcomes."

###

ABOUT MEMORIAL SLOAN KETTERING

As the worlds oldest and largest private cancer center, Memorial Sloan Kettering has devoted more than 135 years to exceptional patient care, influential educational programs, and innovative research to discover more effective strategies to prevent, control and, ultimately, cure cancer. MSK is home to more than 20,000 physicians, scientists, nurses, and staff united by a relentless dedication to conquering cancer. Today, we are one of 51 National Cancer Institute-designated Comprehensive Cancer Centers, with state-of-the-art science and technology supporting groundbreaking clinical studies, personalized treatment, and compassionate care for our patients. We also train the next generation of clinical and scientific leaders in oncology through our continually evolving educational programs, here and around the world. Year after year, we are ranked among the top two cancer hospitals in the country, consistently recognized for our expertise in adult and pediatric oncology specialties. http://www.mskcc.org.

ABOUTHACKENSACKMERIDIANHEALTH

Hackensack MeridianHealthis a leading not-for-profit health care organization that is the largest, most comprehensive and truly integrated health care network in New Jersey, offering a complete range of medical services, innovative research and life-enhancing care.

Hackensack MeridianHealthcomprises 17 hospitals from Bergen to Ocean counties, which includes three academic medical centers Hackensack University Medical Center in Hackensack, Jersey Shore University Medical Center in Neptune, JFK Medical Center in Edison; two childrens hospitals - Joseph M. Sanzari Childrens Hospital in Hackensack, K. Hovnanian Childrens Hospital in Neptune; nine community hospitals Bayshore Medical Center in Holmdel, Mountainside Medical Center in Montclair, Ocean Medical Center in Brick, Palisades Medical Center in North Bergen, Pascack Valley Medical Center in Westwood, Raritan Bay Medical Center in Old Bridge, Raritan Bay Medical Center in Perth Amboy, Riverview Medical Center in Red Bank, and Southern Ocean Medical Center in Manahawkin; a behavioral health hospital Carrier Clinic in Belle Mead; and two rehabilitation hospitals - JFK Johnson Rehabilitation Institute in Edison and Shore Rehabilitation Institute in Brick.

Additionally, the network has more than 500 patient care locations throughout the state which include ambulatory care centers, surgery centers, home health services, long-term care and assisted living communities, ambulance services, lifesaving air medical transportation, fitness and wellness centers, rehabilitation centers, urgent care centers and physician practice locations. Hackensack MeridianHealthhas more than 35,000 team members, and 7,000 physicians and is a distinguished leader in health care philanthropy, committed to the health and well-being of the communities it serves.

The networks notable distinctions include having four hospitals among the top in New Jersey byU.S. News and World Report.Other honors include consistently achieving Magnet recognition for nursing excellence from the American Nurses Credentialing Center and being named toBeckers Healthcares150 Top Places to Work in Healthcare/2019 list.

The Hackensack Meridian School of Medicine at Seton Hall University opened in 2018, the first private medical school in New Jersey in more than 50 years, welcomed its second class of 96 students in 2019 to its ON3 campus in Nutley and Clifton. Additionally, the network partnered with Memorial Sloan Kettering Cancer Center to find more cures for cancer faster while ensuring that patients have access to the highest quality, most individualized cancer care when and where they need it.

Hackensack MeridianHealthis a member of AllSpire Health Partners, an interstate consortium of leading health systems, to focus on the sharing of best practices in clinical care and achieving efficiencies.

View original post here:
Memorial Sloan Kettering Hackensack Meridian Health Partnership Announces Funding for Inaugural Immunology Research Collaboration Projects - Newswise

Recommendation and review posted by Bethany Smith

By Gerald Ondrey | June 9, 2020

Model of a biopharmaceutical facility consisting of pre-configured ExyCell modules and an integrated power supply by Siemens (Source: Exyte)

Siemens Digital Industries (Nuremberg) and Exyte Management GmbH (Stuttgart, both Germany) join forces to offer end-to-end solutions for the biopharmaceutical industry by combining the digitalization expertise of Siemens with the innovation boost from Exyte.

Against the background of the current challenges associated with the Covid-19 pandemic, pharmaceutical companies are facing challenges in developing and mass-producing new vaccines and medicines in existing production facilities. Siemens and Exyte are committed to tackle those challenges. Together, they offer standardized, turnkey solutions compliant with cGMP (current Good Manufacturing Practice) and GAMP (Good Automated Manufacturing Practice) to biotechnology manufacturers as well as cell and gene therapy manufacturers.

Currently, the first buildings with Siemens technology and ExyCell modules are being designed for cell and gene therapy manufacturing and biologicals production in China and Europe.

We are excited to collaborate with Exyte to provide pre-fabricated, modular biotechnology solutions with our technology already embedded. As a market leader for the pharmaceutical industry, we offer know-how for process automation with our Simatic PCS 7 and WinCC automation platforms, as well as power supply and fire protection for modular cleanrooms, says Eckard Eberle, CEO Siemens Process Automation.

Luca Mussati, vice president Pharmaceuticals & Biotechnology at Exyte states: Our partnership with Siemens enables us to pre-integrate Siemens technology into ExyCell modules, thus offering clients end-to-end solutions for their facilities. Our collaboration allows our clients to reap the benefits of industry 4.0 without the necessity of engineering them from scratch each time, thus saving them time and money. ExyCell modules are suitable for new buildings as well as for the retrofit of existing buildings, and can be provided either in standard, off-the-shelf plant configurations, or modules that can be combined to meet specific customer requirements.

Go here to read the rest:
Siemens and Exyte join forces to deliver integrated solutions for fast-track construction of smart biotech facilities - ChemEngOnline

Recommendation and review posted by Bethany Smith

In its rather short life, the gene therapy field has been on a rollercoaster of experiences. While the initial hype was dampened by failures in clinical trials, the field is now experiencing a strong comeback. What was once seen as a hope, is now becoming a reality. But producing viral vectors, the essential delivery vehicles of gene therapies, remains challenging. CEVEC Pharmaceuticals has found a solution and developed a platform that can produce adeno-associated viral vectors (AAVs) as easily as if they were monoclonal antibodies.

As the gene therapy field grows, drug developers are confronted with the fact that most gene therapy products cant be produced at the scale needed to meet growing demands. The reason: lack of adequate viral vector production technologies. What gene therapy developers desperately need is a production platform that can produce viral vectors simply, with no variations, avoiding cumbersome processes, and at reasonable costs.

I have spoken to Nicole Faust, CEO at CEVEC Pharmaceuticals about the challenges in viral vector production, how these are addressed with the companys brand new production platform for AAVs, ELEVECTA, and what it has in common with standardized production platforms that already exist for monoclonal antibodies.

The great thing about gene therapy is that you can, in many cases, tackle the underlying cause of the disease. A lot of diseases today are just treated symptomatically, but with gene therapy, if the underlying cause is a gene defect, you can bring an intact copy of the gene into the patient or even repair the gene using genome editing tools like CRISPR-Cas.

To be able to do so, you need vehicles to deliver the gene. In most cases, although there are a number of non-viral approaches out there, the researchers use viral vectors. This makes a lot of sense because thats what a virus does it delivers genes to cells. Were exploiting that feature of the virus, replacing the viral genes with the therapeutic gene, and using that viral vector to deliver the therapeutic gene to the target cells.

At the moment, there are three different viral vector types mainly used for gene-therapy approaches. One of them is the lentiviral vector, which has the advantage of integrating the gene into the cells, so it will stay there permanently. But lentiviral vectors also bear some risks because they can integrate into an unwanted position in the genome.

At the moment, lentiviral vectors are mainly used for ex-vivo therapies, in particular, because they are very good for transducing hematopoietic cells. Novartis Kymriah, for example, is a CAR-T therapy that uses a lentiviral vector to deliver the Chimeric Antigen Receptor (CAR) to T-cells outside of the patient. Then the modified cells are given back to the patient.

Second, there are adenoviral vectors, which were basically used when gene therapy started more than 20 years ago. They are still being used, but mainly for vaccination approaches. For example, there are some SARS-CoV-2 vaccines being developed at the moment with adenoviral vectors.

When we talk about in vivo gene therapies actually delivering the therapeutic gene to the target cells inside the patient then nearly always AAVs are used. The reason is that AAVs are non-pathogenic and the virus always needs the presence of a helper virus to replicate, and this makes it a lot safer than other viral vectors.

AAV is also a very interesting virus because it comes in a lot of different serotypes different species of AAV. These serotypes correspond to distinctive structures on the surface of the virus, and that means that different tissues can be targeted. If you want to target neural tissue, for example, youll use a different AAV serotype than if you target the liver or muscles.

Another advantage of the AAV is that the particles themselves are very robust and very stable. They are easy to purify and once youve purified them, you can store them for a very long time without losing activity. All that makes them a nearly perfect tool for gene therapy.

Upscaling is one of the biggest challenges in AAV production. The reason is that most of these therapies come out of universities, which means the first viral vectors were produced in a research lab by a method that would yield just enough vector material to do lab experiments. These methods work very well at this level, but they are not really scalable.

Also, in many cases, the viral vector production is based on adherently growing cells, so the cells need a substrate to adhere to in order to survive and divide. That means you cant just use a huge 2000 L bioreactor, but you really have to provide the cells with a substrate and this is difficult at a large scale.Also, adherent cells are not a good solution for scalability because, very often, they still require animal-derived serum to grow, which presents a potential safety issue.

So instead, more and more suspension cell lines are being developed. But these cells still share one problem with adherent cells: Production of AAVs relies on a method called transient transfection.

What does that mean? To make an AAV, you have to bring into the cell different genetic elements. You need one plasmid that carries the rep and cap genes for the AAV life cycle and for producing the capsid; you need a second plasmid with the adenoviral helper gene; and a third plasmid with the therapeutic gene of interest, which is flanked by the recognition sequences that will allow the gene of interest to be packaged into the AAV vector.

So, you can imagine, transient transfection is convenient if you do it at a small scale in a lab,but its a challenge if you need to do it at several hundred liters. Its not only a challenge with respect to the complexity of the process, but it also means you have to provide a lot of plasmid material.

The common understanding is that plasmids used for transient transfection in vector manufacturing for use in humans need to fulfill good manufacturing practice (GMP) requirements, which makes them very expensive. The plasmid costs can make up one-third of the production cost of a batch. Thats obviously a huge cost factor. You also need a transfection reagent. Often, there are sourcing issues and it can sometimes take up to half a year until you finally get the plasmid you need for your GMP production round.

At the moment, were still talking about ultra-rare diseases where batch sizes arent large. But a lot of common diseases, like Alzheimers and Parkinsons, are currently in gene therapy trials. Once these trials are successful and they go into clinical phase III or even enter the market, then upscaling becomes a huge challenge. How will we produce sufficient amounts of the vectors in sufficient quantity and quality?

With ELEVECTA, our new scalable, stable producer cell line technology for AAV gene therapy vectors, we wanted to address all the challenges I just mentioned. First of all, we have eliminated the lengthy and complex transfection step. Our platform does not require any transfection for the actual production of the AAV vector, which also means it doesnt require any plasmid or transfection reagent. So, we dont need any of the expensive raw materials.

ELEVECTA is truly scalable because its basically made AAV production very similar to the well-established recombinant protein production methods. Using our platform, AAV production is very much like making a monoclonal antibody. With this, were addressing the major challenges that people are seeing for AAV production.

Weve been thinking about how people have mastered the production of other biotherapeutics like monoclonal antibodies and why their production is scalable, and the answer is relatively simple: because they are using true, stable producer cell lines. That means all the genetic information thats required to make the product is stably integrated into a producer cell.

Of course, we have to do cell line development in the beginning, but that only needs to be done once. Then, the producer cell line can be used for the production of the gene therapy vector for an unlimited period, as all the components are integrated into the cells genome.

I like making the comparison to monoclonal antibodies because making a monoclonal antibody used to be difficult, but its now become a common technology that can be outsourced to numerous service providers. We wanted to accomplish the same for AAVs. With our ELEVECTA producer cells, were now able to do so.

Developing the ELEVECTA technology was not trivial and we had to apply some tricks from the molecular biology toolbox, but we have been successful in the end. And we have very convincing data from true producer cells where we generated AAV vectors in large bioreactors with consistent productivity and quality.

One of the important quality measures is the full-versus-empty ratio because you always also generate empty particles that will not carry your gene of interest. Thats just inherent to AAVs. With our ELEVECTAplatform, we see a high ratio of full particles, which is what you are looking for, and its also consistent over different batches, making the process more robust and subsequent purification easier.

We want to make ELEVECTAwidely available and we want to see it in use for most of the future AAV gene therapy products. We will help interested parties make their specific producer cell lines with their specific gene of interest and their specific serotype

We are also supporting Pharma companies with a whole portfolio of products by offering a partner package and enabling them to do everything themselves, including cell line development.

But for most clients, we offer product-based projects. This means we generate stable producer cell lines as a service and then transfer those cells and the corresponding manufacturing processes to our clients who then use them under a technology license.

I believe that gene therapy has overcome the initial hurdles now and we will see many more products on the market in the future. But this also means that the therapy costs have to go down. At the moment, were talking about one million or two million for a treatment. Thats a big obstacle to making gene therapies commonly available. One important factor in addressing this issue is lowering production costs.

The standard size of a transient production run is about 200 L. With ELEVECTA, its not a problem to scale up to 2000 L and beyond. So, in addition to reducing the material costs, our clients can benefit significantly from the economy of scale while using standardized processes, equipment, and facilities as known from antibody production.

Are you struggling to find an upscaling solution for your gene therapy? Get in touch with the experts at CEVEC and visit their website for more information!

Images via Shutterstock.com and CEVEC

Read more from the original source:
How to Solve the Production Challenges of AAVs for Gene... - Labiotech.eu

Recommendation and review posted by Bethany Smith

A recent report added by Fior Markets entitledGlobal Gene Therapy Marketprovides a concise analysis of the industry size and review of the key influential factors in the market. The report studies the primary challenges and latest growth strategies embraced by key players. The report covers the initial and future assessment of the globalGene Therapymarket, considering market-relevant information. The markets drivers, restraints, opportunities, and trends, various segments are discussed in the report. It explains conclusions concerning growth factors and eventually influencing holistic growth and lucrative business models in the global market. The report is a comprehensive compilation of professional marketing cues that will help businesses make profitable decisions and drive their business in the correct direction.

NOTE: This report takes into account the current and future impacts of COVID-19 on this industry and offers you an in-depth analysis of GlobalGene TherapyMarket.

DOWNLOAD FREE SAMPLE REPORT:https://www.fiormarkets.com/report-detail/376052/request-sample

The research report covers competition trends, advantages, and disadvantages of the enterprise products as well as analysis of the raw material and industry downstream buyers among some of the parameters. Further, the report also provides data about the topographical reach which may prove useful for the buyers i.e., the production volume and valuation relatable to each region. The report offers data of previous years along with in-depth analysis from 2020 to 2025 on the basis of revenue (USD Billion). Lucrative opportunities available in the globalGene Therapymarket on a global level are highlighted in the report.

Leading companies reviewed in the report are:Spark Therapeutics LLC, Bluebird Bio, UniQure N.V., Juno Therapeutics, GlaxoSmithKline, Chiesi Farmaceutici S.p.A., Bristol Myers Squibb, Celgene Corporation, Human Stem Cell Institute, Voyager Therapeutics, Shire Plc, Sangamo Biosciences, Dimension Therapeutics and others.

BROWSE COMPLETE REPORT AND TABLE OF CONTENTS:https://www.fiormarkets.com/report/global-gene-therapy-market-by-type-germline-gene-376052.html

Advantages of Buying This Report:

The report gives proper direction for important firms and individuals interested in strengthening their position in the globalGene Therapymarket globally. It also explains key elements such as revenue, business distribution, market share, shipment, gross profit. Several essential parameters related to the market studied include a competitive landscape, brief segmentation, and industrial infrastructure. The overall market report is classified by primitive players, applications, types, and geographical areas. The report focuses on the analysis of the new product, futuristic marketing trends, revenue share, demand/supply data, sales, and market growth during the predicted period.

According to the report, the market has set its essence through the locales of theNorth America, Europe, Asia Pacific, South America, and the Middle East and Africa.

The research report analyzes various elements and evaluation of the globalGene Therapymarket through core research methodologies such as PESTEL and SWOT analysis. The report gives a holistic global perspective, rendering conscious statistical analysis and a perspective of integral growth enablers prompting favorable growth across regions. Mainly this report aligns market-specific factors such as threats and challenges as well as opportunities that shape growth in the global market.

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team ([emailprotected]), who will ensure that you get a report that suits your needs.

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Global Gene Therapy Market 2020 Valuable Growth Prospects and Upcoming Trends till 2025 - Bulletin Line

Recommendation and review posted by Bethany Smith

SOUTH SAN FRANCISCO, Calif., June 08, 2020 (GLOBE NEWSWIRE) -- Catalyst Biosciences, Inc. (NASDAQ: CBIO), today announced oral and poster presentations at the upcoming World Foundation of Hemophilia Virtual Summit, taking place from June 14-19, 2020.

The poster abstract is now available online and can be accessed at https://www.wfh.org/virtual-summit/home.

Presentation details:

The open-label Phase 2b study of dalcinonacog alfa (DalcA), a next-generation subcutaneously (SQ) administered Factor IX (FIX) for the treatment of hemophilia B was designed to evaluate daily SQ dosing and the ability to maintain protective steady state FIX levels above 12% in six individuals with severe hemophilia B. Each subject received a single intravenous dose, followed by daily SQ doses of DalcA for 28 days whereby the pharmacokinetics, pharmacodynamics, safety, tolerability and anti-drug antibody formation were monitored. In February 2020, Catalyst Biosciences reported positive interim efficacy and safety data from its Phase 2b trial in an oral presentation at the 13th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD).

Dr. Blouse will present data from preclinical studies of Catalyst’s hemophilia B gene therapy CB 2679d-GT, a novel chimeric AAV capsid expressing the Company’s proprietary enhanced potency FIX variant that may reduce the vector dose required in gene therapy, while maintaining high FIX levels.

A copy of the presentation materials can be accessed on the Events and Presentations section of the Catalyst website once the presentations conclude.

About Catalyst Biosciences Catalyst is a research and clinical development biopharmaceutical company focused on addressing unmet needs in rare hematologic and systemic complement-mediated disorders. Our protease engineering platform includes development programs in hemophilia, a research program on subcutaneous (SQ) systemic complement inhibitors and a partnered preclinical development program with Biogen for dry age-related macular degeneration (AMD). One of our key competitive advantages is that the product candidates generated by our protease engineering platform have improved functionality and potency. These characteristics allow for improved dosing of our candidates including SQ systemic delivery of recombinant coagulation factors and complement inhibitors, low-dose high activity gene therapy constructs and less frequently dosed intravitreal therapeutics. Our most advanced asset, SQ MarzAA has successfully completed Phase 2 development in prophylaxis, significantly reducing the annualized bleed rate (ABR) in individuals with hemophilia A or B with inhibitors. Following regulatory guidance from the FDA and EMA, we recently announced the design of a Phase 3 registration study that is planned for late 2020. Subcutaneous dalcinonacog alfa (DalcA) is being developed for the treatment of hemophilia B and has demonstrated efficacy and safety in a Phase 2b clinical trial that has completed dosing and all participant activities. We have a discovery stage Factor IX gene therapy construct - CB 2679d-GT - for hemophilia B that has demonstrated superiority compared with the Padua variant in preclinical models. Finally, we have a global license and collaboration agreement with Biogen for the development and commercialization of anti-complement Factor 3 (C3) pegylated CB 2782 for the potential treatment of geographic atrophy-associated dry AMD. For more information, please visit http://www.catalystbiosciences.com.

Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. Forward-looking statements include statements about the potential uses and benefits of MarzAA and DalcA to address hemophilia indications and other rare bleeding disorders, including the potential benefits of SQ dosing, plans for the Phase 3 trial of MarzAA in late 2020, and about Catalyst’s collaboration with Biogen. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially, including, but not limited to, the risk that trials and studies may be delayed as a result of the COVID-19 virus and other factors, that trials may not have satisfactory outcomes, that complete data from the Phase 2b trial of DalcA may not replicate previously reported partial results or that additional human trials will not replicate the results from earlier trials, that potential adverse effects may arise from the testing or use of DalcA or MarzAA, including the generation of neutralizing antibodies, which has been observed in patients treated with DalcA, the risk that costs required to develop or manufacture the Company’s products will be higher than anticipated, including as a result of delays in development and manufacturing resulting from COVID-19 and other factors, the risk that Biogen will terminate Catalyst’s agreement with them, competition and other risks described in the Risk Factors” section of the Company’s quarterly report filed with the Securities and Exchange Commission on May 11, 2020, and in other filings with the Securities and Exchange Commission. The Company does not assume any obligation to update any forward-looking statements, except as required by law.

Contact: Ana Kapor Catalyst Biosciences, Inc. investors@catbio.com

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Catalyst Biosciences Announces Oral and Poster Presentations at the World Federation of Hemophilia Virtual Summit 2020 - Stockhouse

Recommendation and review posted by Bethany Smith

The strategy analysis on Global Gene Therapy for Rare Disease Market gives insights of market size, trends, share, growth, development plans, Investment Plan, cost structure and drivers analysis. With precise data covering all key aspects of the existing market, this report offers existing data of leading manufacturers. The Gene Therapy for Rare Disease market report covers marketing channels and market positioning to potential growth strategies, providing in-depth analysis for new competitors or exists competitors in the Gene Therapy for Rare Disease industry. The Report Gives Detail Analysis on Market concern Like Gene Therapy for Rare Disease Market share, CAGR Status, Market demand and up to date Market Trends with key Market segments. The report provides key statistics on the market status of the Gene Therapy for Rare Disease manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the industry. Overall, the report provides an in-depth insight of Gene Therapy for Rare Disease market covering all important parameters.

The PDF Research only provides Table of Contents (ToC), scope of the report and research framework of the report.

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Global Gene Therapy for Rare Disease market 2020-2026: Competitive AnalysisThe Gene Therapy for Rare Disease market report designed to provide entry support, customer profile and M&As as well as go-to-market strategy support. We provide a detailed analysis of key players operating in the global Gene Therapy for Rare Disease market, including key players such as Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc.

Scope of Gene Therapy for Rare Disease Market:The Gene Therapy for Rare Disease market was valued at XX Million US$ in 2019 and is projected to reach XX Million US$ by 2024, at a CAGR of XX% during the forecast period. In this study, 2019 has been considered as the base year and 2020 to 2024 as the forecast period to estimate the market size for Gene Therapy for Rare Disease.

Due to the pandemic, we have included a special section on the Impact of COVID 19 on the Gene Therapy for Rare Disease Market which would mention How the Covid-19 is Affecting the Gene Therapy for Rare Disease Industry, Market Trends and Potential Opportunities in the COVID-19 Landscape, Covid-19 Impact on Key Regions and Proposal for Gene Therapy for Rare Disease Players to fight Covid-19 Impact.

The report also focuses on global major leading industry players of Global Gene Therapy for Rare Disease market providing information such as company profiles, product picture and specification, price, capacity, cost, production, revenue and contact information. Upstream raw materials and equipment and downstream demand analysis are also carried out. With tables and figures helping analyze worldwide Global Gene Therapy for Rare Disease market, this research provides key statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market. In general, the research report is a compilation of key data with regards to the competitive landscape of this vertical and the multiple regions where the business has successfully established its position. The report provides detailed information regarding the major factors (drivers, restraints, opportunities, and challenges) influencing the growth of the Gene Therapy for Rare Disease market. The Gene Therapy for Rare Disease Market Report analyzes opportunities in the overall Gene Therapy for Rare Disease market for stakeholders by identifying the high growth segments.

The scope of the report is limited to the application of the type, and distribution channel. The regions considered in the scope of the report include North America Country (United States, Canada), South America, Asia Country (China, Japan, India, Korea), Europe Country (Germany, UK, France, Italy), Other Country (Middle East, Africa, GCC). This report presents the worldwide Gene Therapy for Rare Disease market size (value, production and consumption), splits the breakdown (data status 20152019 and forecast to 2024), by manufacturers, region, type and application.

Market segment by Type, the product can be split into:Product Type Segmentation: Epicel, IntegraIndustry Segmentation: Chemical, Cosmetic, Pharmaceutical

The Global Gene Therapy for Rare Disease Market report analyses the production of goods, supply, sales, and the current status of the market in a detailed manner. Furthermore, the report examines the production shares and market product sales, as well as the capacity, production capacity, trends in sales, cost analysis, and revenue generation. Several other factors such as import/export status, industrial statistics, demand and supply ratio, gross margin, and industry chain structure have also been studied in the Global Gene Therapy for Rare Disease Market report.

The report comprehends precise analytical information related to market forecasts for several upcoming years. The report also includes the particulars about the valuation of macro and microelements significant for the growth of already established Gene Therapy for Rare Disease Market contenders and emerging new companies. The report uses SWOT analysis for the growth assessment of the outstanding Gene Therapy for Rare Disease Market players. It also analyzes the most recent enhancements while estimating the expansion of the foremost Gene Therapy for Rare Disease Market players. Additionally, the key product category and segments along with sub-segments of the global Gene Therapy for Rare Disease Market are studied in the global Market research.

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This report sample includesBrief Introduction to the research report.Table of Contents (Scope covered as a part of the study)Top players in the marketResearch framework (presentation)Research methodology adopted by Coherent Market Insights

What Reports Provides Full in-depth analysis of the parent market Important changes in market dynamics Segmentation details of the market Former, on-going, and projected market analysis in terms of volume and value Assessment of niche industry developments Market share analysis Key strategies of major players Emerging segments and regional markets Testimonials to companies in order to fortify their foothold in the market.

Further, in the research report, the following points are included along with an in-depth study of each point:* Production Analysis Production is analyzed with respect to different regions, types, and applications. Here, the price analysis of various Market key players is also covered.* Sales and Revenue Analysis Both, sales and revenue are studied for the different regions of the global market. Another major aspect, price, which plays an important part in the revenue generation is also assessed in this section for the various regions.* Supply and Consumption In continuation of sales, this section studies the supply and consumption of the Market. This part also sheds light on the gap between supply and consumption. Import and export figures are also given in this part.* Other analyses Apart from the information, trade and distribution analysis for the Market, contact information of major manufacturers, suppliers and key consumers are also given. Also, SWOT analysis for new projects and feasibility analysis for new investment are included.

Reasons to Buy:* Obtain the most up to date information available on the Gene Therapy for Rare Disease projects globally.* Identify growth segments and opportunities in the industry.* Facilitate decision-making on the basis of strong historical and outlook of Gene Therapy for Rare Disease data.* Develop business strategies with the help of specific insights about the planned and announced Gene Therapy for Rare Disease projects globally.* Keep abreast of key new-build Gene Therapy for Rare Disease projects globally.* Assess your competitors planned and Gene Therapy for Rare Disease projects and capacities.

Additionally, the report is joined by a vital examination of the Gene Therapy for Rare Disease marketplace considering progress, part commitments, and future market forecasts. Furthermore, it offers detailed data of vendors including the profile, specifications of a product, sales, applications, annual performance in the industry, investments, acquisitions and mergers, market size, revenue, market share, and more. The report also studies individual regional market size along with country-wise and region-wise market size during the forecast period. The report also understands the export and import, production, and consumption of every particular region holding the highest market share, market size, or CAGR.

Conclusively, This report will provide you a clear view of each and every fact of the market without a need to refer to any other research report or a data source. Our report will provide you with all the facts about the past, present, and future of the concerned Market.

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Gene Therapy for Rare Disease Market Growth Statistics, Size Projection, Share Value, Top Key Players, Future Insights and Global Industry Trends By...

Recommendation and review posted by Bethany Smith

ORLANDO, Fla.--(BUSINESS WIRE)--Hesperos Inc., pioneers of the Human-on-a-Chip in vitro system, today announced the publication of a new peer-reviewed publication that describes how the companys technology can be used to investigate immune responses following treatment with biological therapeutics for multi-organ systemic diseases, including cancer, infectious diseases and inflammatory disorders. The study was part of a collaboration between Hesperos, Hoffman-La Roche Pharmaceuticals and the University of Central Florida. The manuscript, titled Differential Monocyte Actuation in a Three-Organ Functional Innate Immune System-on-a-Chip, was published today in the prestigious journal Advanced Science. Click here to view a multimedia version of the press release, including media-ready images, downloadable resources, and more.

The immune system plays an important role in coordinating with other organ systems to combat infection, eliminate damaged cells and repair tissue. However, modeling immune response following drug treatment in preclinical studies is challenging due to poor predictability, especially for the innate portion of the system. As the scientific community begins to turn more towards using multi-organ, human-on-a-chip systems as a cost-effective way to increase efficiency and lower toxicity, many of these models lack a systemic immune component.

Hesperos, in collaboration with Hoffmann-La Roche Pharmaceuticals, describe an in vitro, pumpless, three-organ system containing functional human cardiomyocytes, skeletal muscle and hepatocytes in a serum-free medium, along with recirculating human monocyte THP-1 immune cells. Monocytes are a vital immune system cells involved in wound healing, pathogen clearance and activation of the innate immune response, but are also responsible for the cytokine storm found in conditions such as sepsis.

One application where the immune-system-on-a-chip can be immediately useful is for uncovering how severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) directly affects multi-organ systems by activating the cytokine storm from inflammatory macrophages and to support the rapid development of therapeutics. As the global pandemic of COVID-19 continues to grow, this system has the potential to quickly evaluate antiviral and repurposed drugs to help combat this devastating disease, said Michael L. Shuler, Ph.D., Chief Executive Officer of Hesperos.

In the study, the researchers evaluated two different innate immune responses: 1) targeted immune response following tissue-specific damage, which simulates indirect activation of THP-1 cells and, 2) pro-inflammatory immune response following direct activation of immune cells, mimicking acute inflammation and the cytokine storm. Though not reported in this study, Hesperos has also shown that peripheral blood mononuclear cells (PBMCs) and T-cells are sustainable in these multi-organs systems, which would allow some aspects of adaptive immunity to also be modeled.

In the targeted immune response experiments, the cardiotoxic compound amiodarone was used to selectively damage cardiac cells to evaluate how THP-1 immune cells affect the three-organ system. The presence of both amiodarone and THP-1 immune cells led to a more pronounced reduction in cardiac force, conduction velocity and beat frequency compared to amiodarone alone. THP-1 cells were also found to infiltrate the damaged cardiomyocytes and induce significantly increased cytokine IL-6 expression, indicating an M2 macrophage phenotype. No immune-activated damage was reported in the skeletal muscle or liver cells.

The most striking features of our immune-system-on-a-chip is that it emulates different immune reactions for direct tissue-damage and acute inflammation, as well as distinguishes between M1 vs. M2 macrophage phenotypes, said James Hickman, Ph.D., Chief Scientist at Hesperos and Professor at the University of Central Florida.

The study was initially funded by Roche Pharmaceuticals and completed under an NIH grant from National Center for Advancing Translational Sciences (NCATS) Small Business Innovation Research program, which supports studies to advance tissue chip technology toward commercialization.

Tissue chips are a promising technology for accelerating the preclinical timeline and getting treatments to patients more efficiently, said Danilo A. Tagle, Ph.D., associate director for special initiatives at NCATS. Finding improved ways to study immune responses has tremendous implications for drug discovery and the development of more effective personalized medicines in diseases that affect multiple organ systems.

In the pro-inflammatory response experiments, the three-organ system was exposed to lipopolysaccharide (LPS) and interferon gamma (IFN-) to stimulate acute inflammation/cytokine storm and provoke monocyte differentiation and activation. In the absence of THP-1 immune cells, LPS/IFN- treatment had no significant effect on function of the three-organ system. However, with the addition of THP-1 immune cells, LPS/IFN- treatment caused cellular damage to all three-organ components, including THP-1 cell infiltration in liver tissue, and led to significant alterations in cardiac force and beat frequency, as well as skeletal muscle force. Additionally, there was an upregulation of pro-inflammatory cytokines, including TNF-, IL-6 and IL-10, indicating an M1 macrophage phenotype, which is analogous to the cytokine storm found during certain reactions to biologic therapeutics and emulates what occurs during sepsis.

To read the full manuscript, please visit https://doi.org/10.1002/advs.202000323.

About Hesperos

Hesperos, Inc. is a leader in efforts to characterize an individuals biology with Human-on-a-Chip microfluidic systems. Founders Michael L. Shuler and James J. Hickman have been at the forefront of every major scientific discovery in this realm, from individual organ-on-a-chip constructs to fully functional, interconnected multi-organ systems. With a mission to revolutionize toxicology testing as well as efficacy evaluation for drug discovery, the company has created pumpless platforms with serum-free cellular mediums that allow multi-organ system communication and integrated computational PKPD modeling of live physiological responses utilizing functional readouts from neurons, cardiac, muscle, barrier tissues and neuromuscular junctions as well as responses from liver, pancreas and barrier tissues. Created from human stem cells, the fully human systems are the first in vitro solutions that accurately utilize these platforms to predict in vivo functions without the use of animal models, as featured in Science. More information is available at https://hesperosinc.com

Hesperos and Human-on-a-Chip are trademarks of Hesperos Inc. All other brands may be trademarks of their respective holders.

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Hesperos demonstrates innovative Human-on-a-Chip approach to modeling innate immune system response following tissue damage and acute inflammation -...

Recommendation and review posted by Bethany Smith

Simply stated, cancer develops from an accumulation of harmful gene changes (mutations) in a cell, which makes the cell abnormal. The abnormal cancer cell begins to divide without stopping, and may spread to nearby or distant areas of the body.

An inherited gene mutation is passed down by a parent and is present at birth. This occurs in only 5% to 10% of all cancers, and genetic testing is needed to know for sure if someone was born with an inherited cancer gene mutation. Acquired gene mutations are more common and develop over time as people age. A formal cancer risk assessment for individuals with a personal or family history of cancer can be helpful in developing a plan for cancer prevention and early detection.

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Jennifer Van Horn is an oncology clinical nurse specialist. She joined the cancer program at Cheyenne Regional Medical Center in 2011. She received her graduate training at the University of Northern Colorado, and recently completed a post-masters nurse practitioner program through the University of Colorado.

View original post here:
Van Horn: Myths vs. facts about inherited cancer risk and testing - Wyoming Tribune

Recommendation and review posted by Bethany Smith

A new national study aims to answer that question, and women are being recruited to participate at no cost in a comparison of the effectiveness of the two screening methods.

Its called the WISDOM study Women Informed to Screen Depending On Measures of risk and its sponsors include Blue Cross Blue Shield of North Dakota and Sanford Health.

The study has a goal of recruiting 100,000 women who have not had breast cancer between the ages of 40 and 74.

So far, 27,000 women have enrolled, including 6,000 from Sanford Health in North Dakota, South Dakota and Minnesota.

Study participation through Sanford currently leads among health systems, said Dr. Andrea Kaster, a doctor at the Edith Sanford Breast Center and one of 10 investigators conducting the study.

Mammogram screening recommendations vary, but generally doctors recommend that women between the ages of 40 and 74 get yearly scans.

Thats what Sanford still recommends, Kaster said. The age to stop screening is based on a discussion between the patient and provider, she said, taking risk factors into consideration.

With so many recommendations for breast cancer screening out there, it can be hard for patients and providers to know what the right approach is for them, she said.

Clinical studies like WISDOM help us use the latest in imaging and genetics along with a patients personal history to determine how best to screen women for breast cancer," she added. "Through this study, I hope that we will be able to contribute to developing a more personalized approach to breast cancer screening.

Dr. Greg Glasner, chief medical officer at Blue Cross Blue Shield of North Dakota, said if personalized screening proves effective, it could spare many women from the anxiety caused by false positives.

The results of the study have the potential to impact existing standards of practice on screening and prevention, diagnosis and treatment of breast cancer, he said. Supporting the WISDOM study by encouraging eligible members to participate in the study is an important step in its success.

Blue Cross Blue Shield of North Dakota will cover the cost of services a member may receive through the study, with no cost sharing or impact to the members preventive benefit accumulations.

Similarly, the Sanford Health Plan covers the cost of genetic testing, assessments and other expenses associated with the WISDOM study for its members who are enrolled in the study and are asked to be tested.

Women who are interested in enrolling can go online to learn more by visiting http://www.thewisdomstudy.org or edith.sanfordhealth.org/research/wisdom.

The study will last five years, but partial results will be released before the studys conclusion.

Read more from the original source:
Is personalized screening more effective than annual mammograms in detecting breast cancer? This study aims to find out - Duluth News Tribune

Recommendation and review posted by Bethany Smith

A new market report by Verified Market Research on the Prenatal and New-born Genetic Testing Market has been released with reliable information and accurate forecasts for a better understanding of the current and future market scenarios. The report offers an in-depth analysis of the global market, including qualitative and quantitative insights, historical data, and estimated projections about the market size and share in the forecast period. The forecasts mentioned in the report have been acquired by using proven research assumptions and methodologies. Hence, this research study serves as an important depository of the information for every market landscape. The report is segmented on the basis of types, end-users, applications, and regional markets.

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The report also emphasizes the initiatives undertaken by the companies operating in the market including product innovation, product launches, and technological development to help their organization offer more effective products in the market. It also studies notable business events, including corporate deals, mergers and acquisitions, joint ventures, partnerships, product launches, and brand promotions.

Leading Prenatal and New-born Genetic Testing manufacturers/companies operating at both regional and global levels:

The report also inspects the financial standing of the leading companies, which includes gross profit, revenue generation, sales volume, sales revenue, manufacturing cost, individual growth rate, and other financial ratios.

The report also focuses on the global industry trends, development patterns of industries, governing factors, growth rate, and competitive analysis of the market, growth opportunities, challenges, investment strategies, and forecasts till 2026. The Prenatal and New-born Genetic Testing Market was estimated at USD XX Million/Billion in 2016 and is estimated to reach USD XX Million/Billion by 2026, expanding at a rate of XX% over the forecast period. To calculate the market size, the report provides a thorough analysis of the market by accumulating, studying, and synthesizing primary and secondary data from multiple sources.

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The market is predicted to witness significant growth over the forecast period, owing to the growing consumer awareness about the benefits of Prenatal and New-born Genetic Testing. The increase in disposable income across the key geographies has also impacted the market positively. Moreover, factors like urbanization, high population growth, and a growing middle-class population with higher disposable income are also forecasted to drive market growth.

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Key factors influencing market growth:

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Prenatal and New-born Genetic Testing Market 2019 Break Down by Top Companies, Countries, Applications, Challenges, Trends, Opportunities and Forecast...

Recommendation and review posted by Bethany Smith

From 7% to nearly 9% of patients with advanced cancer were found to harbor a germline variant with targeted therapeutic actionability in the first study of its kind.

The study involved 11,974patients with various tumor types. All the patients underwent germline genetic testing from 2015 to 2019 at the Memorial Sloan Kettering Cancer Center (MSKCC) in New York City, using the next-generation sequencing panel MSK-IMPACT.

This testing identified 2043 patients (17.1%) with variants in cancer predisposition genes, including 849 patients (7.1%) who had targetable genes by strict criteria and 1003 patients (8.6%) by less strict criteria.

"Of course, these numbers are not static," commented lead author Zsofia K. Stadler, MD, a medical oncologist at MSKCC. "And with the emergence of novel targeted treatments with new FDA indications, the therapeutic actionability of germline variants is likely to increase over time.

"Our study demonstrates the first comprehensive assessment of the clinical utility of germline alterations for therapeutic actionability in a population of patients with advanced cancer," she added.

Stadler presented the study results during a virtual scientific program of the American Society of Clinical Oncology (ASCO) 2020.

Testing for somatic mutations is evolving as the standard of care in many cancer types, and somatic genomic testing is rapidly becoming an integral part of the regimen for patients with advanced disease. Some studies suggest that 9% to 11% of patients harbor actionable genetic alterations, as determined on the basis of tumor profiling.

"The take-home message from this is that now, more than ever before, germline testing is indicated for the selection of cancer treatment," said Erin Wysong Hofstatter, MD, from the Yale School of Medicine, New Haven, Connecticut, in a Highlights of the Day session.

Now, more than ever before, germline testing is indicated for the selection of cancer treatment. Dr Erin Wysong Hofstatter

An emerging indication for germline testing is the selection of treatment in the advanced setting, she noted. "And it is important to know your test. Remember that tumor sequencing is not a substitute for comprehensive germline testing."

For their study, Stadler and colleagues reviewed the medical records of patients with likely pathogenic/pathogenic germline (LP/P) alterations in genes that had known therapeutic targets so as to identify germline-targeted treatment either in a clinical or research setting.

"Since 2015, patients undergoing MSK-IMPACT may also choose to provide additional consent for secondary germline genetic analysis, wherein up to 88 genes known to be associated with cancer predisposition are analyzed," she said. "Likely pathogenic and pathogenic germline alterations identified are disclosed to the patient and treating physician via the Clinical Genetic Service."

A total of 2043 (17.1%) patients who harbored LP/P variants in a cancer predisposition gene were identified. Of these, 11% of patients harbored pathogenic alterations in high or moderate penetrance cancer predisposition genes. When the analysis was limited to genes with targeted therapeutic actionability, or what the authors defined as tier 1 and tier 2 genes, 7.1% of patients harbored a targetable pathogenic germline alteration.

BRCA alterations accounted for half (52%) of the findings, and 20% were associated with Lynch syndrome.

The tier 2 genes, which included PALB2, ATM, RAD51C, and RAD51D, accounted for about a quarter of the findings. Hofstatter noted that, using strict criteria, 7.1% of patients were found to harbor a pathogenic alteration and a targetable gene. Using less stringent criteria, additional tier 3 genes and additional genes associated with DNA homologous recombination repair brought the number up to 8.6%.

For determining therapeutic actionability, the strict criteria were used; 593 patients (4.95%) with recurrent or metastatic disease were identified. For these patients, consideration of a targeted therapy, either as part of standard care or as part of an investigation or research protocol, was important.

Of this group, 44% received therapy targeting the germline alteration. Regarding specific genes, 50% of BRCA1/2 carriers and 58% of Lynch syndrome patients received targeted treatment. With respect to tier 2 genes, 40% of patients with PALB2, 19% with ATM, and 37% with RAD51C or 51D received a PARP inhibitor.

Among patients with a BRCA1/2 mutation who received a PARP inhibitor, 55.1% had breast or ovarian cancer, and 44.8% had other tumor types, including pancreas, prostate, bile duct, gastric cancers. These patients received the drug in a research setting.

For patients with PALB2 alterations who received PARP inhibitors, 53.3% had breast or pancreas cancer, and 46.7% had cancer of the prostate, ovary, or an unknown primary.

The discussant for the paper, Funda Meric-Bernstam, MD, chair of the Department of Investigational Cancer Therapeutics at the University of Texas MD Anderson Cancer Center, Houston, pointed out that most of the BRCA-positive patients had cancers traditionally associated with the mutation. "There were no patients with PTEN mutations treated, and interestingly, no patients with NF1 were treated," she said. "But actionability is evolving, as the MEK inhibitor selumitinib was recently approved for NF1."

Some questions remain unanswered, she noted, such as, "What percentage of patients undergoing tumor-normal testing signed a germline protocol?" and, "Does the population introduce a bias such as younger patients, family history, and so on?"

It is also unknown what percentage of germline alterations were known in comparison with those identified through tumor/normal testing. Also of importance is the fact that in this study, the results of germline testing were delivered in an academic setting, she emphasized. "What if they were delivered elsewhere? What would be the impact of identifying these alterations in an environment with less access to trials?

"But to be fair, it is not easy to seek the germline mutations," Meric-Bernstam continued. "These studies were done under institutional review board protocols, and it is important to note that most profiling is done as standard of care without consenting and soliciting patient preference on the return of germline results."

An infrastructure is needed to return/counsel/offer cascade testing, and "analyses need to be facilitated to ensure that findings can be acted upon in a timely fashion," she added.

The study was supported by MSKCC internal funding. Stadler reports relationships (institutional) with Adverum, Alimera Sciences, Allergan, Biomarin, Fortress Biotech, Genentech/Roche, Novartis, Optos, Regeneron, Regenxbio, and Spark Therapeutics. Meric-Bernstram reports relationships with numerous pharmaceutical companies.

American Society of Clinical Oncology (ASCO) 2020: Abstract 1500, presented May 30, 2020.

Follow Medscape Oncology on Twitter for more cancer news: @MedscapeOnc.

Read more here:
Germline Testing In Advanced Cancer Can Lead to Targeted Tx - Medscape

Recommendation and review posted by Bethany Smith

The post-Covid world will be a new and changed one. And this new world will give way to new jobs and careers. The pandemic has left no sector or industry untouched; especially hit are entertainment, aviation, real estate, hospitality and tourism. However, there are those that have, in some ways, benefitted--healthcare, edutech, information technology and consulting, among others. Does that mean that these sectors are poised to soar further once this global crisis is over?

TECH JOBS

Opportunities in the field of digital technology, with an emphasis on enterprise management systems which deal with controlling, monitoring and managing information technology (IT), infrastructure and its applications, will be the way forward. Logistics, marketing and secondary value chains, such as human resources, finance, and customer relationship management (CRM), may also witness a rise within organisations. Everything is rapidly going digital, and candidates equipped with know-how in cloud computing, machine learning, artificial intelligence, data analytics, robotic process automation and digital marketing will have high-growth career prospects, says Sandeep Pachpande, chairman, ASM Group of Institutes, Pune.

GENETIC TESTING

The pandemic has made it clear that there is a severe shortage of doctors, medical equipment, devices and raw materials in the country, which are needed to deal with a crisis of this magnitude. Now, more than ever, jobs in pharmaceutical companies and healthcare will soar and, along with it, requirement for online counseling, psychological aid and telemedicine, since many may avoid visiting hospitals for a long time to come. People have become vigilant about health concerns and general well-being as a consequence of Covid. Therefore, genetic testing, a field that identifies mutations in genes, proteins and chromosomes, could emerge as an advantageous career. Our genes contain all the information we need to maintain optimum health and fitness to reach correct and early diagnostics. At a time where we are struggling with a single virus, research in this field has grown immensely, says Chandni Luthra, director, FutureMed, a company that helps patients with proactive and preventive health planning.

ALSO READ | Online transition

CONTENT CREATORS

It is a golden time for careers in the area of OTT (over-the-top) platformsNetflix, Amazon Prime and Disney+ Hotstar, among many othersdelivering content over the internet. Software engineers, project managers, UX designers and creative producers are currently seeing a lot of opportunities. Content curators can make the most of this time as consumption of online content has gone up since the lockdown. Studying the current trends and interests related to online streaming will come in handy for scriptwriters, lyricists and screenplay writers to gain a newer understanding of the struggles of people in a state of crisis.

FOOD SCIENTISTS

The E-grocery sector has certainly been on an upswing since the lockdown. Making daily food essentials available to customers is a Herculean task, and any disruption in supply chains can lead to loss of perishable products. Hence, the need for greater workforce, better planning and last-mile connectivity across areas will lead to a surge in recruitment of agricultural scientists, environmental engineers and supply chain officials in agriculture-technology companies to help farmers manage crops, plan harvest, deal with water shortage and reach a greater consumer base. Food scientists will be all the more indispensable with consumers becoming extra cautious about their consumption habits and switching to nutrition-focused diets.

A shift to online payments becoming more popular is encouraging fin-tech companies to come up with contactless payment solutions. People should also keep an eye out for roles such as financial analysts, strategists and business developers.

ONLINE FACULTY

The new trend in education is to learn at a click. This has given edutech companies a massive user base since many have opted to utilise their time upskilling and reskilling. As remote learning grows, demand for faculty members, teachers, education consultants and social media specialists grows with it. We have received tremendous response from learners across the nation since the lockdown, says Nikhil Barshikar, founder and managing director of Imarticus Learning, an edutech platform. The accessibility of online learning modules from any device is making it easier for aspirants to learn from home. This new phase in learning is here to stay.

The Covid crisis has made it clear that businesses can run in the absence of workers in traditional office set-ups, and that people will have to become more efficient and innovative with their work for long-term survival. Keeping this in mind, students will have to review and re-align their career focus to join the workforce of tomorrow. The future is all about multi-specialties, resilience and adaptability.

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Recommendation and review posted by Bethany Smith

CINCINNATI, June 08, 2020 (GLOBE NEWSWIRE) -- Aerpio Pharmaceuticals, Inc. (Aerpio) (Nasdaq: ARPO), a biopharmaceutical company focused on developing compounds that activate Tie2 to treat ocular diseases and diabetic complications, today announced that it is hosting a key opinion leader (KOL) call on a novel mechanism for the treatment of glaucoma on Friday, June 12, 2020 at 11:30am Eastern Time.

The call will feature presentations by Dr. Paul Kaufman M.D. (University of Wisconsin) and Dr. Janey Wiggs, M.D., Ph.D. (Massachusetts Eye and Ear Infirmary and Harvard Medical School), who will discuss the current glaucoma treatment landscape and unmet medical needs, as well as the role of the Tie2 receptor in maintaining intraocular pressure. Drs. Kaufman and Wiggs will be available to answer questions at the conclusion of the event.

Aerpio's management team will also discuss its pipeline candidate, razuprotafib (formerly AKB-9778), for treating patients with glaucoma. Razuprotafib is a small molecule inhibitor that restores Tie2 activation in Schlemms canal and lowers intraocular eye pressure (IOP) via decreasing resistance to outflow from the eye. Razuprotafib has been formulated as a once or twice-daily topical eye drop and is entering a Phase 2 clinical trial in Q3:20, with top line data expected in Q1:21.

Aerpio recently announced positive and statistically significant intraocular eye pressure (IOP) reduction in a Phase 1b trial of 43 glaucoma patients, when razuprotafib was added to prostaglandin treatment. This data set is summarized here.

Paul Kaufman, M.D. is the Ernst H. Brny Emeritus Professor of Ocular Pharmacology and past Chair of the Department of Ophthalmology & Visual Sciences at the University of Wisconsin School of Medicine and Public Health, in Madison, Wisconsin. He is a physician-scientist, specializing in glaucoma and studying the mechanisms of aqueous humor formation and drainage, and the age-related loss of near vision. Dr Kaufman is a past President and past Executive Vice President of the Association for Research in Vision and Ophthalmology (ARVO), past President of the International Society for Eye Research (ISER), and has served on the US National Advisory Eye Council and numerous foundation and corporate scientific advisory boards. He has had continuous research funding from the US National Eye Institute for 40 years and from numerous private foundations, has authored over 375 original scientific articles and 75 book chapters, co-edited several textbooks including the most recent editions of Adlers Physiology of the Eye, and received numerous honors and awards including the Friedenwald Award from ARVO and the Balazs Prize from ISER. He was Editor-in-Chief of Investigative Ophthalmology & Visual Science from 2008 through 2012. Dr. Kaufman also holds an honorary Doctor of Medicine degree from Uppsala University in Sweden, where he was a post-doctoral research fellow.

Janey L. Wiggs, M.D., Ph.D. is a physician-scientist at the Massachusetts Eye and Ear Infirmary and Harvard Medical School. She is currently the Paul Austin Chandler Professor of Ophthalmology and is the Vice Chair for Clinical Research in Ophthalmology at Harvard Medical School. She also directs the CLIA-certified genetic testing laboratory at the Massachusetts Eye and Ear Infirmary and is a co-director of the Ocular Genomics Institute and co-director of the Glaucoma Center of Excellence. Dr. Wiggs received her B.A. and Ph.D. degrees in biochemistry from the University of California at Berkeley and her M.D. degree from Harvard Medical School. She did post-doctoral training in molecular genetics under the direction of Dr. Ted Dryja. Dr. Wiggs completed the ophthalmology residency at the Massachusetts Eye and Ear Infirmary and received fellowship training in glaucoma and also in medical genetics and is certified by the both the American Board of Ophthalmology and the American Board of Medical Genetics. Dr. Wiggs research program is focused on the discovery and characterization of genetic factors that contribute to the blinding eye disease glaucoma and is funded by the National Eye Institute (NEI) as well as other nonprofit foundations. She is investigating the genetic etiologies of both early-onset and adult forms of glaucoma and is the PI of the NEIGHBORHOOD consortium for gene discovery in primary open angle glaucoma and is a founding member of the International Glaucoma Genetics Consortium (IGGC). She has also participated in research programs funded by the US-INDO joint working group (NEI) and the NEI eyeGENE consortium. Dr. Wiggs was the inaugural chair of the Genetics Group for ARVO and is an ARVO gold fellow. She currently serves on the editorial boards of IOVS, JAMA Ophthalmology, Molecular Vision, Journal of Glaucoma, and Annual Reviews in Vision Science. She is a member of the scientific advisory boards for the Glaucoma Research Foundation, Research to Prevent Blindness and the Glaucoma Foundation, and is a past member of the Advisory Council of the National Eye Institute. She has received the Heed Award, the Heed/Knapp Award, the Research to Prevent Blindness Scholar Award, the AAO Honor Award, the Lew Wasserman Merit Award, the Alcon Research Award, the David L. Epstein award from the ARVO Foundation and was a winner of the NEI Audacious Goal competition. She is an elected member of the Glaucoma Research Society, the American Ophthalmological Society, the Academia Ophthalmologica Internationalis and the National Academy of Medicine.

About RazuprotafibRazuprotafib binds to and inhibits vascular endothelial protein tyrosine phosphatase (VE-PTP), an important negative regulator of Tie2. Decreased Tie2 activity contributes to vascular instability in many diseases including diabetes and more recently has been shown to contribute to the development of increased IOP and glaucoma. Razuprotafib activates the Tie2 receptor irrespective of extracellular levels of its binding ligands, angiopoietin-1 (agonist) or angiopoietin-2 (antagonist) and may be the most efficient pharmacologic approach to maintain normal Tie2 activation. Aerpio is studying a topical ocular formulation of razuprotafib in open angle glaucoma and exploring the utility of subcutaneous razuprotafib for diabetic complications, including diabetic nephropathy.

About Aerpio PharmaceuticalsAerpio Pharmaceuticals, Inc. is a biopharmaceutical company focused on developing compounds that activate Tie2 to treat ocular diseases and diabetic complications. Recently published mouse and human genetic data implicate the Angpt/Tie2 pathway in maintenance of Schlemms canal, a critical component of the conventional outflow tract. The Companys lead compound, razuprotafib (formerly AKB-9778), a first-in-class small molecule inhibitor of vascular endothelial protein tyrosine phosphatase (VE-PTP), is being developed as a potential treatment for open angle glaucoma, and the Company intends to investigate the therapeutic potential of razuprotafib in other indications. The Company is also evaluating development options for ARP-1536, a humanized monoclonal antibody, for its therapeutic potential in the treatment of diabetic vascular complications including nephropathy and diabetic macular edema (DME). The Companys third asset is a bispecific antibody that binds both VEGF and VE-PTP which is designed to inhibit VEGF activation and activate Tie2. This bispecific antibody has the potential to be an improved treatment for wet age-related macular degeneration and DME via intravitreal injection. Finally, the Company has exclusively out-licensed AKB-4924 (now called GB004), a first-in-class small molecule inhibitor of hypoxia-inducible factor-1 (HIF). GB004 is being developed by AKB-4924s exclusive licensor, Gossamer Bio, Inc. (Nasdaq: GOSS). For more information, please visit http://www.aerpio.com.

Forward Looking StatementsThis press release contains forward-looking statements. Statements in this press release that are not purely historical are forward-looking statements. Such forward-looking statements include, among other things, the Companys product candidates, including razuprotafib, ARP-1536 and the bispecific antibody asset, the clinical development plan therefor and the therapeutic potential thereof, the Companys plans and expectations with respect to razuprotafib and the development therefor and therapeutic potential thereof in addressing COVID-19 and the intended benefits from the Companys collaboration with Gossamer Bio for GB004, including the continued development of GB004 and the milestone and royalty payments related to the collaboration. Actual results could differ from those projected in any forward-looking statements due to several risk factors. Such factors include, among others, the continued development of GB004 and maintaining and deriving the intended benefits of the Companys collaboration with Gossamer Bio; ability to continue to develop razuprotafib or other product candidates, including in indications related to COVID-19; the inherent uncertainties associated with the drug development process, including uncertainties in regulatory interactions, the design of planned or future clinical trials, commencing clinical trials and enrollment of patients in clinical trials; obtaining any necessary regulatory clearances in order to commence and conduct planned or future clinical trials; the impact of the ongoing COVID-19 pandemic on the Companys business operations, including research and development efforts and the ability of the Company to commence, conduct and complete its planned clinical activities; and competition in the industry in which the Company operates and overall market conditions; and the additional factors set forth in our Annual Report on Form 10-K for the year ended December 31, 2019, as updated by our subsequent Quarterly Reports on Form 10-Q and our other subsequent filings with the SEC.

These forward-looking statements are made as of the date of this press release, and the Company assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law. Investors should consult all the information set forth herein and should also refer to the risk factor disclosure set forth in the reports and other documents the Company files with the SEC available at http://www.sec.gov.

Investors & Media:Gina MarekVP Financegmarek@aerpio.comOrInvestors:Irina KofflerLifeSci Advisorsikoffler@lifesciadvisors.com

The rest is here:
Aerpio Hosting Key Opinion Leader Call on a Novel Mechanism for the Treatment of Glaucoma - GlobeNewswire

Recommendation and review posted by Bethany Smith

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A FEW SIMPLE STEPS: Discover your DNA story with our easy-to-follow instructions. Simply activate your DNA kit online and return your saliva sample in the prepaid package to our state-of-the-art lab. In roughly six to eight weeks, your results will be ready online.DNA + TRAITS: AncestryDNA + Traits lets you discover 25+ of your most interesting traits, allowing you to explore how your genes might have influenced a range of appearance, sensory, and other personal characteristicslike eye color, freckles, and more.PRECISE ETHNICITY AND LIVING RELATIVES: A more precise ethnicity estimate with greater geographic detail and in-depth historical insights connects you to the places in the world where your story startedfrom unique regions to living relatives.BUILD A FAMILY TREE: Combine what you learn from your DNA with over 100 million family trees and billions of records for more insight into your genealogy and origins.PROTECTING PRIVACY: When you send in your DNA test kit, we employ industry-standard security measures in all stages of our process, work to protect your data, and give you control over itwith data encryption, secure databases, and easy-to-use controls.

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Trusted Business Insights answers what are the scenarios for growth and recovery and whether there will be any lasting structural impact from the unfolding crisis for the Clinical Oncology Next Generation Sequencing market.

Trusted Business Insights presents an updated and Latest Study on Clinical Oncology Next Generation Sequencing Market 2019-2026. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market.The report further elaborates on the micro and macroeconomic aspects including the socio-political landscape that is anticipated to shape the demand of the Clinical Oncology Next Generation Sequencing market during the forecast period (2019-2029).It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

Get Sample Copy of this Report @ Clinical Oncology Next Generation Sequencing Market Size Report 2025 (Includes Business Impact of COVID-19)

Industry Insights, Market Size, CAGR, High-Level Analysis: Clinical Oncology Next Generation Sequencing Market

The global clinical oncology next generation sequencing market size was estimated at USD 627.2 million in 2018. Significant increase in the adoption of genome-focused pharmacology for cancer treatment is expected to provide growth avenues for the market. NGS serves as one of the highly advanced genomic techniques for the analysis of mutations that lead to cancer progression.The growth of the market is supplemented by decrease in the cost of genetic sequencing, advancements in the field of personalized medicine and oncology, rise in prevalence of cancer, globally, and increase in adoption of this technique over single-gene testing. Since this technique is more reliable than Sanger sequencing for diagnosis, the market is expected to witness substantial growth over the forecast period.

Breakthrough advancements in the development of genetic-based tests for oncology and other disorders during the past few years have significantly propelled market growth. Next generation sequencing technology offers a better understanding of tumor mechanism, thus enabling rational drug design. As a result, more products are expected to get commercialized in the near future.A rise in oncology-related spending is further expected to fuel market growth in the coming years. Government organizations are focused on encouraging patients to regularly undergo diagnostic examinations to reduce oncology-related healthcare expenditures. As per data estimates, healthcare expenditure is anticipated to rise significantly, thereby influencing the adoption of NGS diagnostics platforms.The biotechnology industry is marked by high competition and so is the NGS market for clinical as well as research applications. Prominent participants are actively involved in R&D to develop novel rapid, small, and less expensive platforms. Service providers are embracing the trend of increasing the amount of constructed sequence reads for each cycle run.Commercially available sequencing platforms analyze both DNA and RNA samples. Key players aim to increase the utility of high throughput technologies for clinical applications. The acquisition of smaller entities operating in the market is also one of the strategic initiatives adopted by players to maintain a competitive position. For instance, in May 2018, Illumina acquired Edico Genome to accelerate data analysis for its next generation sequencing platforms.

Technology Insights of Clinical Oncology Next Generation Sequencing Market

Targeted sequencing in clinical oncology accounted for the largest revenue share in 2018 and is expected to maintain its share over the forecast period. Targeted sequencing and resequencing encompasses shortest read lengths and is one of the conventional forms of commercially available sequencing technology. Moreover, focused panels for targeted cancer sequencing aid in the reduction of cost burdens owing to reduction in the amount of data to be analyzed. As targeted cancer sequencing evaluates a limited gene-set, it can be used to analyze variations in rare tumor sub-clones with greater sensitivity and deep coverage.The whole-genome sequencing segment is expected to grow at the fastest rate through 2025. Application of whole genome sequencing technology for comparisons between tumor tissue and normal tissue in clinical oncology is attributive to segment growth. Developed rapid, large-capacity sequencers such as HiSeq and MiSeq series find application in largescale whole genome sequencing.Workflow InsightsNGS workflow includes library preparation, sample enrichment, sample quality assurance, DNA tagging, sequencing, DNA quantification, genetic data assemble, data storage and analysis, and interpretation. Sequencing accounted for the largest share in the clinical oncology NGS market as it is the most important and crucial step in the entire process and involves the usage of sophisticated platforms. Continuous R&D has led to the introduction of novel, benchtop, or portable NGS sequencing platforms, thereby complimenting market growth. Moreover, NGS platforms are expected to witness lucrative growth throughout the forecast period owing to the expected increase in adoption and usage rates of whole-genome sequencing.

Application Insights of Clinical Oncology Next Generation Sequencing Market

Next generation sequencing finds application in the field of clinical oncology for screening, companion diagnostics, and others. The adoption of NGS-based molecular diagnosis for oncology has increased as this technology allows concurrent sequencing of several target genes and provides rich diagnostic markers for the development of molecular diagnostic assays.Application of NGS technology in the field of oncology ensures early diagnosis so that treatment measures can be taken in time. Therefore, the arrival of NGS for cancer diagnosis has opened many new frontiers in the field. For instance, large-scale projects such as International Cancer Genome Consortium (ICGC) and The Cancer Genome Atlas (TCGA) have issued data from thousands of tumors across major cancer types. This data can be used to have a comprehensive overview of cancerous cells and help enhance prognostic and therapeutic applications.The market for companion diagnostics is expected to grow significantly in the coming years owing to numerous benefits offered by the technique in the field of oncology. Currently, available companion diagnostic products are applicable for non-small cell lung cancer, melanoma, colorectal, ovarian, and breast cancers.End-use InsightsBy way of end use, the clinical oncology next generation sequencingmarket has been segmented into hospitals, clinics, and laboratories. Laboratories held a prominent share in the market for clinical oncology NGS and this trend is likely to continue over the forecast period. The hospitals segment is expected to register a significant CAGR through 2025. Rise in adoption of NGS services in hospitals to determine the rate of disease progression and scale-up the appropriate treatment regimen for cancer is anticipated to influence growth in this segment.

Provision of clinical services by leading players in the market such as Illumina, which includes CLIA certified tests for predisposition screening, is anticipated to boost revenue in the coming years. Clinics utilize NGS technology to identify novel cancer mutations, for genetic testing for hereditary cancer syndrome, for personalized oncology treatment, and for detection of circulating DNA for cancer.

Regional Insights of Clinical Oncology Next Generation Sequencing Market

North America occupied the largest share in the global market in 2018 owing to increased focus of government bodies on cancer treatment, rise in healthcare expenditure, and presence of adequate healthcare infrastructure. Moreover, high prevalence of cancer, growing demand for personalized medicine, well-developed healthcare facilities, and availability of novel diagnostics techniques, especially in the field of oncology, can be attributed to the estimated market share of the region. Additionally, growing morbidity and mortality due to cancer has increased the need for development of novel therapies, thereby driving this regional market.Asia Pacific is expected to be the fastest-growing region throughout the forecast period majorly due to continuous reforms in the healthcare and pharmaceutical industries in the region. Other factors contributing to this growth are untapped markets in emerging economies. The market is continuously driven by rise in cancer incidence across Asia Pacific. China dominates the cancer burden in the region, followed by India, Japan, Indonesia, and the Republic of Korea. The top three types of cancer with his incidences in the region are breast, lung, and prostate cancer.

Market Share Insights of Clinical Oncology Next Generation Sequencing Market

The global market is marked by the presence of high competition among market players. Illumina and Roche account for a substantial share owing to wide product offerings in the field of next-generation sequencing for cancer. Other prominent players operating in this market space include Agilent; Thermo Fisher Scientific; Myriad Genetics; Beijing Genomics Institute (BGI); Perkin Elmer, Inc.; Qiagen NV; Partek, Inc.; Pacific Bioscience, Inc.; Foundation Medicine; Oxford Nanopore Technologies Ltd.; Paradigm Diagnostics, Inc.; Caris Life Sciences; and Eurofins Scientific.

Segmentations, Sub Segmentations, CAGR, & High-Level Analysis overview of Clinical Oncology Next Generation Sequencing Market Research ReportThis report forecasts revenue growth at global, regional, and country levels and provides an analysis of latest industry trends in each of the sub-segments from 2014 to 2025. For the purpose of this study, this market research report has segmented the globalclinical oncology next generation sequencing market report on the basis of technology, workflow, application, end use, and region:

Technology Outlook (Revenue, USD Million, 2019 2030)

Whole Genome Sequencing

Whole Exome Sequencing

Targeted Sequencing & Resequencing

Workflow Outlook (Revenue, USD Million, 2019 2030)

NGS Pre-Sequencing

NGS Sequencing

NGS Data Analysis

Application Outlook (Revenue, USD Million, 2019 2030)

Screening

Sporadic Cancer

Inherited Cancer

Companion Diagnostics

Others

End-use Outlook (Revenue, USD Million, 2019 2030)

Hospitals

Clinics

Laboratories

Quick Read Table of Contents of this Report @ Clinical Oncology Next Generation Sequencing Market Size Report 2025 (Includes Business Impact of COVID-19)

Trusted Business InsightsShelly ArnoldMedia & Marketing ExecutiveEmail Me For Any ClarificationsConnect on LinkedInClick to follow Trusted Business Insights LinkedIn for Market Data and Updates.US: +1 646 568 9797UK: +44 330 808 0580

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How COVID-19 Pandemic Will Impact Clinical Oncology Next Generation Sequencing Market Business Opportunity, And Growth 2020-2026 - Jewish Life News

Recommendation and review posted by Bethany Smith

-- In post-treatment muscle biopsies, clinical trial participants in the high-dose cohort showed a dose-dependent increase in transduction and expression when compared with the low-dose cohort, with a mean of 72% beta-sarcoglycan (beta-SG) positive fibers, as measured by immunohistochemistry (IHC), substantially exceeding the pre-defined 50% measure for success ---- A mean signal intensity of 73% in the high-dose group was observed compared to normal control ---- A mean beta-sarcoglycan expression of 62% as measured by Western blot was observed in the high-dose cohort compared to normal control ---- An 89% mean reduction of creatine kinase (CK) from baseline was observed in the high-dose cohort ---- Continued functional improvement was observed in the low-dose cohort at one year --

CAMBRIDGE, Mass., June 08, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc.(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive results from a study of SRP-9003, its investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). Results included safety and expression results from three clinical trial participants in the high-dose cohort measured at 60 days, and one-year functional data from three clinical trial participants in the low-dose cohort. SRP-9003 is in development for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4), a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan (beta-SG) proteins. SRP-9003 is a gene construct that transduces skeletal and cardiac muscle, delivering a gene that codes for the full-length beta-sarcoglycan protein, the absence of which is the sole cause of progressive degeneration and a shortened lifespan characterized by the disease.

We were very encouraged by the previously reported results from our first cohort of patients treated with a lower dose of SRP-9003, including impressive expression, good tolerability, and positive functional signals, which continue impressively at one year. We are excited to have been able to achieve even more impressive expression and other biomarkers in our higher-dose cohort for SRP-9003, along with good tolerability. The SRP-9003 gene construct, vector and promoter were designed with the goal of robustly delivering to skeletal and cardiac muscles a gene coding for the missing beta-sarcoglycan protein that causes LGMD2E. These data support the conclusion that the therapy is achieving its intended purpose, driving robust expression in the muscles where it is needed, said Doug Ingram, President and CEO, Sarepta. SRP-9003 employs the same vector, AAVrh74, and same promoter, MHCK7, as SRP-9001, our therapy in development to treat Duchenne muscular dystrophy. And Cohort 2 received a similar dose as our ongoing SRP-9001 studies for Duchenne. The safety and efficacy results with these two doses of SRP-9003 provide us with additional experience and confidence with the rh74 vector and the MHCK7 promoter as we select the dose for the pivotal trial of SRP-9003 and work to quickly develop this therapy for patients who currently have no treatment options.

The SRP-9003 study has two cohorts, each studying a different dose-per-kilogram based on the weight of the patient. Three participants in the low-dose cohort (Cohort 1) were treated with a one-time infusion of SRP-9003 dosed at 5x1013vg/kg and an additional three participants in the high-dose cohort (Cohort 2) received a one-time infusion dosed at 2x1014vg/kg. The six participants were between the ages of 4 and 13. Post-treatment biopsies were taken at 60 days. Sarepta previously shared data from Cohort 1 in 2019, including positive and robust expression and biomarker data and positive 9-month functional results.

Preliminary results from Cohort 2 (n=3) are as follows:

In Cohort 1 (low dose), at one year all three participants continued to show improvements from baseline across all functional measures, including the North Star Assessment for Limb-Girdle Muscular Dystrophies, time-to-rise, four-stair climb, 100-meter walk test and 10-meter walk test. These results are distinctly different from what an age-matched, natural history group would predict. There have been no new drug-related safety signals observed since the 9-month update, and no decreases in platelet counts outside of the normal range or signs of complement activation were observed.

LGMD2E is a devastating neuromuscular disease that causes significant disability in the children we see and currently lacks treatment options beyond tailored physical therapy, said Jerry Mendell, M.D., principal investigator at the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Childrens Hospital and lead investigator for the study. We are pleased that these data show robust expression, similar to what we observed in the micro-dystrophin program, for the protein that is missing in children with LGMD2E, and remain hopeful that this brings us one step closer to a therapy that can help improve both prognosis and quality of life.

About SRP-9003 and the studySRP-9003 uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat peripheral neuromuscular diseases. AAVrh74 has lower immunogenicity rates than reported with other human AAV vectors. The MHCK7 promoter has been chosen for its ability to robustly express in the heart, which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications.

This first-in-human study is evaluating a single intravenous infusion of SRP-9003 among children with LGMD2E between the ages of four and 15 years with significant symptoms of disease. Sarepta has exclusive rights to the LGMD2E gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

About Limb-Girdle Muscular DystrophyLimb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begin in muscles around the hips and shoulders before progressing to muscles in the arms and legs.

Patients with limb-girdle muscular dystrophy Type 2E (LGMD2E) begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, progresses to loss of ambulation in the teen years and often leads to early mortality. There is currently no treatment or cure for LGMD2E.

Sarepta has five LGMD gene therapy programs in development, including subtypes for LGMD2E, LGMD2D, LGMD2C, LGMD2B and LGMD2L, and holds an option for a sixth program for LGMD2A.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.comor follow us onTwitter,LinkedIn,InstagramandFacebook.

Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the goal of the SRP-9003 gene construct, vector and promoter of robustly delivering to skeletal and cardiac muscles a gene coding for the missing beta-sarcoglycan protein that cause LGMD2E; SRP-9003s potential to improve both prognosis and quality of life; the potential read through of the SRP-9003 trial results to SRP-9001; our plans to select the dose for the pivotal trial of SRP-9003 and work to quickly develop this therapy for patients; and the potential market opportunities with respect to SRP-9003 and SRP-9001.

These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: success in preclinical trials and early clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; the data presented in this release may not be consistent with the final data set and analysis thereof or result in a safe or effective treatment benefit; different methodologies, assumptions and applications we utilize to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by theFDAor foreign regulatory authorities; we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, due to a variety of reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates and the COVID-19 pandemic; and even if Sareptas programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year endedDecember 31, 2019, and most recent Quarterly Report on Form 10-Q filed with theSecurities and Exchange Commission(SEC) as well as otherSECfilings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review theSECfilings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

InternetPosting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com.Weencourageinvestorsandpotentialinvestorsto consult our website regularly for important information about us.

Source:Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics Announces Positive Expression and Functional Data From the SRP-9003 Gene Therapy Trial to Treat Limb-Girdle Muscular Dystrophy...

Recommendation and review posted by Bethany Smith

Several projects that push the boundaries of knowledge and have high potential for impact have been awarded support through Princeton'sDean for Research Innovation Fund.

The funding makes possible explorations in the natural sciences and social sciences, collaborations with industry, and collaborations between artists and scientists or engineers. Several of the projects have the potential for direct benefits to human health while others explore themes in history and the arts. The projects were chosen by faculty-led committees based on the quality, originality and potential of the research.

"Princeton faculty are pioneers across the range of human inquiry, and these innovation funds enable our researchers and their teams to explore paths that they might not otherwise take," said Dean for ResearchPablo Debenedetti, the Class of 1950 Professor in Engineering and Applied Science and professor of chemical and biological engineering. "Through this funding program, the University shows support for the innovation mindset that leads to truly profound advances and can also lead to societal benefits."

A project to study antimicrobial agents found in the pouches of marsupials such as this sugar glider is one of three to be awarded Dean for Research Innovation Funds for New Ideas in the Natural Sciences.

Photo by Patrick Kavanagh

The fund supports the exploration of new concepts that require additional study or experiments before they are ready to become the basis of a competitive proposal to a funding agency.

Ricardo Mallarino, assistant professor of molecular biology, will study the naturally occurring antimicrobial agents that marsupials which carry their offspring to term in an external pouch employ to kill toxic microbes. Marsupials are born without a working immune system and rely for survival entirely on factors secreted by glands found in the pouch or transferred through maternal milk. The researchers will explore the genetic sequences of these molecules, known as antimicrobial peptides, to discover how the genes have evolved over time in ways that contribute to their efficacy against microbes, with the eventual goal of understanding natural defense mechanisms and informing the design of new antimicrobial drugs.

Charles Fefferman and Amit Singer

Photos by Julia Fefferman and provided by the Department of Mathematics

Amit Singer, professor of mathematics and the Program in Applied and Computational Mathematics, and Charles Fefferman, the Herbert E. Jones, Jr. '43 University Professor of Mathematics, will develop algorithms to enhance the capability of a powerful microscope to capture images of protein structures as they move and change shape. The team will adapt mathematical approaches for use with cryogenic electron microscopy (cryo-EM) to take pictures of not just fixed molecules but also ones that are flexing and morphing into differing shapes, as happens for example when a drug molecule interacts with its target receptor.

Lisa Boulanger, associate professor of neuroscience, will conduct research to improve the safety and efficacy of viruses that are used for gene therapy to treat nervous system disorders including spinal motor atrophy and certain kinds of blindness. The most commonly used viruses are forms of adeno-associated virus, or AAV, in which the viral genes were removed and replaced with therapeutic genes. Boulanger found that these viruses can unexpectedly change the structure of circuits in the brain, changes that are associated with increased exploratory behavior in mice. She and her team will determine how AAV alters neural circuits, screen for approaches to prevent these changes, and facilitate gene therapy in the nervous system.

The use of individual-level historical data for studies of long-term impacts of history, including precolonial and colonial societies in Africa, is the focus of a new projected awarded the Dean for Research Innovation Fund for New Ideas in the Social Sciences.

Image by Franois-Edmond Fortier

This fund encourages scholarship on new and enduring questions. The selected projects are ones that will result in the advancement of a discipline through the development of new directions, working groups, conferences, technologies, or expanded access to research resources, or lead to a major piece of scholarly work.

Katja Guenther, associate professor of history, will explore the concept of resilience, which has emerged over the last few decades as one of the most prized traits of the modern age, the key to the future success of our businesses, financial systems, cities, the environment, and even humanity. Guenther will trace the history of the concept across a range of disciplines ranging from psychology to ecology while incorporating environmental humanities, the history of science and the study of systems. In examining the way resilience has been used in the past, she will trace its ethical ramifications, its translations into policy, and its future implications.

Leonard Wantchekon,professor ofpolitics andinternationalaffairs, will lead a team of undergraduate and graduate studentsfrom Princeton University as well as researchers from the African School of Economics and the Universidad de los Andesto explore how to rigorously collect individual-level historical data for studies of long-term impacts of historicalevents.Through studies of precolonial and colonial societies in Africa and the Americas, Wantchekon will develop a novel approach that combines econometrics and advanced statistical methods with more traditional historical and social research techniques to pioneer the emerging field of historical applied microeconometrics.

This fund supports research collaborations that address societal challenges of interest both to industry and to academic scientists and engineers. Industry often plays an essential role in bringing the innovations of University researchers to fruition and making them available to society at large. The program requires an agreement from the industry collaborator to provide matching funds in the second year of the project.

Gerard Wysocki, associate professor of electrical engineering, and his team will develop a highly sensitive laser-based sensor in collaboration with Hamamatsu Photonics, a leading instrumentation company. During critical care of patients in respiratory distress, measuring the consumption of oxygen in the breath can provide important information about the patient's health. The resulting prototype, capable of detecting not only oxygen concentration but the types (or isotopes) of oxygen, will be evaluated by collaborators in a hospital setting.

Howard Stone, Princeton's Donald R. Dixon '69 and Elizabeth W. Dixon Professor of Mechanical and Aerospace Engineering, will team with Princeton, NJ, medical device firm NovaFlux Inc. to explore the ability of a new cleaning fluid composed of nano-sized fibers and micro-sized particles suspended in a liquid. Stone and colleagues at NovaFlux will explore the fluid's ability to remove bacterial coatings, or biofilms, which form on surfaces such as medical tubing and devices. The fluid can also be used to remove a broad range of pathogens including bacteria, spores and viruses.

Left: Dancer Cori Kresge in photo by Sigrid Adriaenssens. Right: Cori Kresge in net, with dancer Chris Ralph standing in background.

Photo by Barry Onouye Studio

This fund encourages collaborations between faculty scholars in the arts and those in the natural sciences or engineering to promote synergistic innovations, allowing experts in seemingly unrelated fields to unify and expand their respective knowledge in ways that benefit both disciplines.

Sigrid Adriaenssens and Rebecca Lazier

Photos by Sameer A. Khan/Fotobuddy and Bentley Drezner

Combining dance and structural engineering, Rebecca Lazier, senior lecturer in dance in the Lewis Center for the Arts, and Sigrid Adriaenssens, associate professor of civil and environmental engineering, will collaborate on a new project involving the movements of dancers as a strategy to explore nets as resilient building facades and impact barriers. Nets are attractive for uses that require flexibility to withstand waves, winds and earthquakes, but they stiffen with usage, making for potentially disastrous outcomes. This project aims to create choreographic works that generate a new understanding of how nets turn rigid when loaded and soften when unloaded. At the same time, dancers will explore the interaction between net and human. The net will be a full collaborative partner in the dance rather than a setting or a prop, enabling creativity that moves beyond how people have used nets previously.

The project involves first designing and fabricating different types of nets and then hosting intensive research sessions with dancers in Princeton's Lewis Center for the Arts. The team will experiment with choreography and collect movement in a digital framework gathered from reflective beads mounted on the nets. The project will include collaboration with Adam Finkelstein, professor of computer science, to transform these data into insights on net structure and properties. The project will include a public performance.

Clockwise from top left: Eduardo Cadava, Fazal Sheikh, Mark Zondlo and John Higgins.

Photos by Eduardo Cadava, Alexandra Beck, Andlinger Center for Energy and the Environment, and Laura Pedrick

A team of artists, scientists and engineers led by Eduardo Cadava, professor of English,John Higgins, associate professor of geosciences, and Mark Zondlo, associate professor of civil and environmental engineering, will embark on a field measurement and outreach project centered on environmental justice issues in the Red Rock Wilderness surrounding Bears Ears and Grand Staircase-Escalante National Monuments in southern Utah. The project, which builds on work by artist and photographer Fazal Sheikh, the 2018-19 Barron Visiting Professor in the Environment and the Humanities and current artist-in-residence at the Princeton Environmental Institute (PEI), will document the effects of extractive industries such as mining on public and Native lands. Expected outcomes include collaborations with indigenous communities and native engineers and scientists, methane and water testing, geological research, an interactive mapping system and website, and online syllabi. The project will contribute to an exhibition at the Smart Art Museum and the Yale University Gallery of Art.

The team includes Michael Celia, the Theodora Shelton Pitney Professor of Environmental Studies and professor of civil and environmental engineering and director of PEI; Peter Jaffe, the William L. Knapp '47 Professor of Civil Engineering and professor of civil and environmental engineering; Zia Mian, research scientist at Princeton's Woodrow Wilson School of Public and International Affairs and co-director of the Program on Science and Global Security; and Emily Wild, Princeton's chemistry, geosciences and environmental studies librarian.

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Funding the next big idea: New projects receive Dean for Research Innovation awards - Princeton University

Recommendation and review posted by Bethany Smith

NEEDHAM, Mass., June 8, 2020 /PRNewswire/ --For biopharma companies seeking increased development and manufacturing capacity for vaccines and therapies, including new COVID-19-related programs, Thermo Fisher Scientific provided highlights of its new capabilities during the BIO International Convention, June 8-12, now a virtual event at http://www.bio.org/events/bio-digital.

"We can now provide an uninterrupted path from development to commercialization for biopharma companies, small to large, in geographies worldwide and across vaccines, antivirals and other therapies," said Mike Shafer, senior vice president and president, pharma services, Thermo Fisher Scientific. "Through our recent strategic initiatives, we are delivering to our customers a powerful combination of expertise, flexibility and scale that allows us to be the partner they start with and stay with."

Earlier this year, the company announced plans to invest in new capabilities and capacityfor biologics, cell and gene therapies and drug product development and commercialization. For example, to support demand for gene therapies, Thermo Fisher will be doubling its viral vector manufacturing capacity with a new manufacturing sitein Plainville, Mass. Construction of the 290,000-square-foot facility will be complete in 2022 and complements the company's recent expansions in Lexington and Cambridge, Mass., and Alachua, Fla. This week, STAT News will feature, "The STAT Guide to viral vectors, the linchpin of gene therapy," which covers the issues and considerations in engineering and manufacturing viral vectors.

In addition, through a new strategic partnership with CSLLimited,Thermo Fisher will support CSL's product portfolio through its pharma services network, including drug product development, biologics manufacturing, sterile fill-finish, packaging and clinical trials logistics. Thermo Fisher will also operate CSL's state-of-the-art biologics manufacturing facility in Lengnau, Switzerland, once construction is completed in 2021.

These strategic investments expand Thermo Fisher's pharma services capabilities for its biotech customers working on antiviral therapies, and for pharmaceutical manufacturers scaling up for novel coronavirus vaccines. Thermo Fisher's pharma services business is also supporting approximately 100 COVID-19 customer projects across its global pharma services network from producing promising therapeutics and treatments to ramping up manufacturing, distribution, packaging and logistics for clinical trials of new vaccine candidates.

To learn more, the company will host two webinars at BIO 2020 this week. The first, "Transforming Medicine with Expertise, Flexibility and Scale," will take place June 12 at 1:00 p.m. EDT. Register for the webinar here. In addition, an on-demand BPI Theater presentation, "Leveraging Infrastructure Investments and Innovation to Accelerate Biologics Development," will run throughout the week and can be accessed here.

About Thermo Fisher Scientific Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue exceeding $25 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, improving patient diagnostics and therapies or increasing productivity in their laboratories, we are here to support them. Our global team of more than 75,000 colleagues delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services and Patheon. For more information, please visit http://www.thermofisher.com.

Media Contact Information: Marcia Goff, Thermo Fisher Scientific +1.508.902.7041 marcia.goff@thermofisher.com

Rachel Robbins, Greenough +1.781.249.9490 rrobbins@greenough.biz

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SOURCE Thermo Fisher Scientific

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Thermo Fisher Scientific Highlights Growing Capacity for Biologics, Cell and Gene Therapy Production During BIO 2020 - P&T Community

Recommendation and review posted by Bethany Smith

Big name partnerships were critical for Nimbus Therapeutics first decade. With a head-turning $1.2 billion $600 million of which were paid within months deal from Gilead and a returning customer in Celgene, the biotech emerged as a prolific pioneer of computational chemistry and structure-based drug discovery while the industry went through a seismic shift in its thinking of the role that algorithms play in engineering new therapies.

As the second of the initial batch of programs enter the clinic, Nimbus is unveiling the headings that will define what it calls its second chapter.

Their team of 20-plus scientists has identified four new targets AMPK2, CTPS1, Cbl-b and WRN which they have been probing with academic collaborators and experts at Schrdinger. And this time around, they plan to keep all four in-house for at least a little longer, focusing on recruiting new staffers and friendly researchers rather than buyers.

At the same time, Nimbus has dropped its STING efforts after a slew of biotechs reached for it and came up empty.

One of the compliments weve been paid by our peers in the broader drug discovery and development community has been in our ability to select really interesting targets that are quite compelling, Jeb Keiper the former BD chief who took over as CEO from Don Nicholson less than two years ago told Endpoints News. We care a lot about being able to do that.

Aside from the usual suspects in target selection, such as genetic validation and medical need, Nimbus zoomed into ones for which a selective, structure-based approach is particularly helpful, CSO Peter Tummino said.

In AMPK (AMP-activated protein kinase), that means finding activators selective for the 2 subunit, which could translate into a better safety profile as metabolic drugs. Similarly, the challenge in CTP is to find compounds selective for the S-1 isoform. Cbl-b (Cbl proto-oncogene B) is an E3 ubiquitin ligase a natural protein degrader thats garnered attention from both small companies like Nurix and big ones like Roche. Finally, the goal with WRN (Werner syndrome ATP-dependent helicase) is to come up with a new treatment option for tumors vulnerable to disruptions in DNA repair.

What isnt new is all 4 are were looking for small molecule agents, he said.

Although these are targets of high interest, he added, much is still unknown about their structures, and Nimbus is working with leading biologists to elucidate them with techniques like cryo-EM and crystallography.

These are not things you can simply outsource to contract research groups, Keiper added. You really are doing fundamental academic discovery work. Thats the kind of special sauce mixing dyed in the wool drug discovery veterans with computational experts that Nimbus believes will keep it going for many years more.

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Nimbus picks 4 preclinical targets for the next chapter of its pioneering computational drug discovery work - Endpoints News

Recommendation and review posted by Bethany Smith

The collaboration will see Achilles Therapeutics expand their manufacturing footprint at the CGT Catapult manufacturing centre to supply clinical trials of their novel T cell therapy products.

Onboarding has begun into one of the newly operational modules at the state-of-the-art facility.

London UK, 8 June 2020 The Cell and Gene Therapy Catapult (CGT Catapult) announced today that Achilles Therapeutics (Achilles) will be manufacturing their advanced therapy products for clinical trials in one of the newly operational modules at the CGT Catapult manufacturing centre.

The CGT Catapult manufacturing centre has remained operational throughout the COVID-19 pandemic to prioritise the production of patient batches manufactured for clinical supply, and the facility is continuing to support companies as they accelerate their operations.

Achilles is a clinical-stage biopharmaceutical company developing novel cancer immunotherapies targeting clonal neoantigens: protein markers unique to each individual that are expressed on the surface of every cancer cell. Achilles uses DNA sequencing data from each patient, together with a proprietary bioinformatics platform, to identify clonal neoantigens specific to that patient, enabling the development of personalised cell therapies. Targeting multiple clonal neoantigens that are present on all cancer cells, but absent on healthy cells, allows individualised treatments to target and destroy tumours, without harming healthy tissues and minimising tumour escape. Achilles has two ongoing clinical studies, in advanced non-small cell lung cancer and recurrent metastatic melanoma.

Achilles is the first company to occupy one of the new modules, part of an expansion of the CGT Catapult manufacturing centre completed in September 2019 which has doubled its capacity. The additional six segregated modules are now fully operational, helping to meet exceptional demand for collaboration at the centre and support the growing UK cell and gene therapy industry.

By establishing their manufacturing process at the CGT Catapult manufacturing centre, Achilles will have access to the GMP facilities, supply chain and expertise to enable them to expand their manufacturing capabilities and support clinical studies of their innovative cell therapy products.

Dr Edward Samuel, SVP Manufacturing, Achilles Therapeutics said:

We are excited that this collaboration with CGT Catapult will enable us to increase our GMP manufacturing capacity as we progress our ongoing clinical trials. The opportunity to occupy a dedicated module with access to key support services and expertise will enable Achilles to establish a manufacturing platform to drive our growing pipeline.

Matthew Durdy, CEO, Cell and Gene Therapy Catapult commented:

We are delighted to announce this collaboration with Achilles Therapeutics, the latest pioneering company to choose the CGT Catapult manufacturing centre and surrounding ecosystem to develop their products. By working with Achilles, we hope to further the development of their highly promising pipeline products, and in doing so help to address an unmet medical need for patients.

This collaboration also represents the first company to occupy one of the new modules at the centre, an expansion which has been driven by strong Government and European Regional Development Fund support and will enable us to continue to play a pivotal role for growth in the UK industry moving forward.

The CGT Catapult manufacturing centre has been backed by over 75m of funding, including investment from the UK governments Industrial Strategy Challenge Fund, the Department for Business Energy and Industrial Strategy, Innovate UK and from the European Regional Development Fund.

About the Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 200 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

About the European Regional Development Fund

This project has received 3.36m of funding from the England European Regional Development Fund as part of the European Structural and Investment Funds Growth Programme 2014-2020. The Ministry of Housing, Communities and Local Government (and in London the intermediate body Greater London Authority) is the Managing Authority for European Regional Development Fund. Established by the European Union, the European Regional Development Fund helps local areas stimulate their economic development by investing in projects which will support innovation, businesses, create jobs and local community regenerations. For more information visit https://www.gov.uk/european-growth-funding.

About the Industrial Strategy Challenge Fund

This project has received 12m of funding from the Industrial Strategy Challenge Fund, part of the governments modern Industrial Strategy. The Industrial Strategy Challenge Fund is a four-year, 1 billion investment in cutting-edge technology designed to create jobs and improve living standards, built on guidance from business and the academic community. Healthcare and Medicine is one of three core areas for investment under the programme.

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Achilles Therapeutics to set up manufacturing operations at the CGT Catapult manufacturing centre - PharmiWeb.com

Recommendation and review posted by Bethany Smith

SOUTH SAN FRANCISCO, Calif., June 08, 2020 (GLOBE NEWSWIRE) -- Catalyst Biosciences, Inc. (NASDAQ: CBIO), today announced oral and poster presentations at the upcoming World Foundation of Hemophilia Virtual Summit, taking place from June 14-19, 2020.

The poster abstract is now available online and can be accessed at https://www.wfh.org/virtual-summit/home.

Presentation details:

The open-label Phase 2b study of dalcinonacog alfa (DalcA), a next-generation subcutaneously (SQ) administered Factor IX (FIX) for the treatment of hemophilia B was designed to evaluate daily SQ dosing and the ability to maintain protective steady state FIX levels above 12% in six individuals with severe hemophilia B. Each subject received a single intravenous dose, followed by daily SQ doses of DalcA for 28 days whereby the pharmacokinetics, pharmacodynamics, safety, tolerability and anti-drug antibody formation were monitored. In February 2020, Catalyst Biosciences reported positive interim efficacy and safety data from its Phase 2b trial in an oral presentation at the 13th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD).

Dr. Blouse will present data from preclinical studies of Catalysts hemophilia B gene therapy CB 2679d-GT, a novel chimeric AAV capsid expressing the Companys proprietary enhanced potency FIX variant that may reduce the vector dose required in gene therapy, while maintaining high FIX levels.

A copy of the presentation materials can be accessed on the Events and Presentations section of the Catalyst website once the presentations conclude.

About Catalyst BiosciencesCatalyst is a research and clinical development biopharmaceutical company focused on addressing unmet needs in rare hematologic and systemic complement-mediated disorders. Our protease engineering platform includes development programs in hemophilia, a research program on subcutaneous (SQ) systemic complement inhibitors and a partnered preclinical development program with Biogen for dry age-related macular degeneration (AMD). One of our key competitive advantages is that the product candidates generated by our protease engineering platform have improved functionality and potency. These characteristics allow for improved dosing of our candidates including SQ systemic delivery of recombinant coagulation factors and complement inhibitors, low-dose high activity gene therapy constructs and less frequently dosed intravitreal therapeutics. Our most advanced asset, SQ MarzAA has successfully completed Phase 2 development in prophylaxis, significantly reducing the annualized bleed rate (ABR) in individuals with hemophilia A or B with inhibitors. Following regulatory guidance from the FDA and EMA, we recently announced the design of a Phase 3 registration study that is planned for late 2020. Subcutaneous dalcinonacog alfa (DalcA) is being developed for the treatment of hemophilia B and has demonstrated efficacy and safety in a Phase 2b clinical trial that has completed dosing and all participant activities. We have a discovery stage Factor IX gene therapy construct - CB 2679d-GT - for hemophilia B that has demonstrated superiority compared with the Padua variant in preclinical models. Finally, we have a global license and collaboration agreement with Biogen for the development and commercialization of anti-complement Factor 3 (C3) pegylated CB 2782 for the potential treatment of geographic atrophy-associated dry AMD. For more information, please visit http://www.catalystbiosciences.com.

Forward-Looking StatementsThis press release contains forward-looking statements that involve substantial risks and uncertainties. Forward-looking statements include statements about the potential uses and benefits of MarzAA and DalcA to address hemophilia indications and other rare bleeding disorders, including the potential benefits of SQ dosing, plans for the Phase 3 trial of MarzAA in late 2020, and about Catalysts collaboration with Biogen. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially, including, but not limited to, the risk that trials and studies may be delayed as a result of the COVID-19 virus and other factors, that trials may not have satisfactory outcomes, that complete data from the Phase 2b trial of DalcA may not replicate previously reported partial results or that additional human trials will not replicate the results from earlier trials, that potential adverse effects may arise from the testing or use of DalcA or MarzAA, including the generation of neutralizing antibodies, which has been observed in patients treated with DalcA, the risk that costs required to develop or manufacture the Companys products will be higher than anticipated, including as a result of delays in development and manufacturing resulting from COVID-19 and other factors, the risk that Biogen will terminate Catalysts agreement with them, competition and other risks described in the Risk Factors section of the Companys quarterly report filed with the Securities and Exchange Commission on May 11, 2020, and in other filings with the Securities and Exchange Commission. The Company does not assume any obligation to update any forward-looking statements, except as required by law.

Contact:Ana KaporCatalyst Biosciences, Inc.investors@catbio.com

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Catalyst Biosciences Announces Oral and Poster Presentations at the World Federation of Hemophilia Virtual Summit 2020 - GlobeNewswire

Recommendation and review posted by Bethany Smith

LEIDEN, Netherlands & CAMBRIDGE, Mass., June 08, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V.(Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announced a virtual presentation of data from the Companys Phase 1/2 trial of sepofarsen. The data will be shared via a video presentation through the Association for Research in Vision and Ophthalmology (ARVO). It is the first time this data, previously announced in a press release and conference call in October 2019, will be presented in association with an ophthalmology medical congress.

Due to COVID-19, the ARVO 2020 Annual Meeting was cancelled and instead video-recorded presentations will be available on ARVOLearn, ARVOs online learning platform.

Details of ProQRs presentation are as follows:

Presenter: Stephen R. Russell, MD, Professor and Director of Vitreoretinal Diseases and Surgery Service, Department of Ophthalmology and Visual Sciences, University of Iowa

Presentation Title: Results of a phase 1b/2 trial of intravitreal (IVT) sepofarsen (QR-110) antisense oligonucleotide in Leber congenital amaurosis 10 (LCA10) due to p.Cys998X mutation in the CEP290 gene

Date: The video presentation will be available on ARVOLearn starting June 15, 2020.

About SepofarsenSepofarsen (QR-110) is being evaluated in the pivotal Phase 2/3 Illuminate trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of Leber congenital amaurosis 10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene. The p.Cys998X mutation leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to enable normal splicing, resulting in restoration of normal (wild type) CEP290 mRNA and subsequent production of functional CEP290 protein. Sepofarsen is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation and rare pediatric disease designation from the FDA as well as access to the PRIME scheme by the EMA.

About ProQRProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare diseases such as Leber congenital amaurosis 10, Usher syndrome and autosomal dominant retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.*Since 2012*

FORWARD-LOOKING STATEMENTSThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such statements include those relating to our presentation of data through ARVO. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

ProQR Therapeutics N.V.

Investor Contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@proqr.comorHans VitzthumLifeSci AdvisorsT: +1 617 535 7743hans@lifesciadvisors.com

Media Contact:Sara ZelkovicLifeSci Public RelationsT: +1 646 876 4933sara@lifescipublicrelations.com

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ProQR Announces Virtual Presentation of Phase 1/2 Sepofarsen Data Through the Association for Research in Vision and Ophthalmology (ARVO) -...

Recommendation and review posted by Bethany Smith