Archive for the ‘Gene Therapy Research’ Category

Global Gene Therapy for Inherited Genetic Disorders Market: Trends Estimates High Demand by 2027

The Gene Therapy for Inherited Genetic Disorders Market 2020 report includes the market strategy, market orientation, expert opinion and knowledgeable information. The Gene Therapy for Inherited Genetic Disorders Industry Report is an in-depth study analyzing the current state of the Gene Therapy for Inherited Genetic Disorders Market. It provides a brief overview of the market focusing on definitions, classifications, product specifications, manufacturing processes, cost structures, market segmentation, end-use applications and industry chain analysis. The study on Gene Therapy for Inherited Genetic Disorders Market provides analysis of market covering the industry trends, recent developments in the market and competitive landscape.

It takes into account the CAGR, value, volume, revenue, production, consumption, sales, manufacturing cost, prices, and other key factors related to the global Gene Therapy for Inherited Genetic Disorders market. All findings and data on the global Gene Therapy for Inherited Genetic Disorders market provided in the report are calculated, gathered, and verified using advanced and reliable primary and secondary research sources. The regional analysis offered in the report will help you to identify key opportunities of the global Gene Therapy for Inherited Genetic Disorders market available in different regions and countries.

The final report will add the analysis of the Impact of Covid-19 in this report Gene Therapy for Inherited Genetic Disorders industry.

Some of The Companies Competing in The Gene Therapy for Inherited Genetic Disorders Market are: BioMarin Pharmaceutical Inc., bluebird bio Inc., Novartis AG, Orchard Therapeutics Plc, Spark Therapeutics Inc., and

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The report scrutinizes different business approaches and frameworks that pave the way for success in businesses. The report used Porters five techniques for analyzing the Gene Therapy for Inherited Genetic Disorders Market; it also offers the examination of the global market. To make the report more potent and easy to understand, it consists of info graphics and diagrams. Furthermore, it has different policies and improvement plans which are presented in summary. It analyzes the technical barriers, other issues, and cost-effectiveness affecting the market.

Global Gene Therapy for Inherited Genetic Disorders Market Research Report 2020 carries in-depth case studies on the various countries which are involved in the Gene Therapy for Inherited Genetic Disorders market. The report is segmented according to usage wherever applicable and the report offers all this information for all major countries and associations. It offers an analysis of the technical barriers, other issues, and cost-effectiveness affecting the market. Important contents analyzed and discussed in the report include market size, operation situation, and current & future development trends of the market, market segments, business development, and consumption tendencies. Moreover, the report includes the list of major companies/competitors and their competition data that helps the user to determine their current position in the market and take corrective measures to maintain or increase their share holds.

What questions does the Gene Therapy for Inherited Genetic Disorders market report answer pertaining to the regional reach of the industry?

The report claims to split the regional scope of the Gene Therapy for Inherited Genetic Disorders market into North America, Europe, Asia-Pacific, South America & Middle East and Africa. Which among these regions has been touted to amass the largest market share over the anticipated duration

How do the sales figures look at present how does the sales scenario look for the future?

Considering the present scenario, how much revenue will each region attain by the end of the forecast period?

How much is the market share that each of these regions has accumulated presently

How much is the growth rate that each topography will depict over the predicted timeline

A short overview of the Gene Therapy for Inherited Genetic Disorders market scope:

Global market remuneration

Overall projected growth rate

Industry trends

Competitive scope

Product range

Application landscape

Supplier analysis

Marketing channel trends Now and later

Sales channel evaluation

Market Competition Trend

Market Concentration Rate

Reasons to Read this Report

This report provides pin-point analysis for changing competitive dynamics

It provides a forward looking perspective on different factors driving or restraining market growth

It provides a six-year forecast assessed on the basis of how the market is predicted to grow

It helps in understanding the key product segments and their future

It provides pin point analysis of changing competition dynamics and keeps you ahead of competitors

It helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments

TABLE OF CONTENT:

Chapter 1:Gene Therapy for Inherited Genetic Disorders Market Overview

Chapter 2: Global Economic Impact on Industry

Chapter 3:Gene Therapy for Inherited Genetic Disorders Market Competition by Manufacturers

Chapter 4: Global Production, Revenue (Value) by Region

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

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

Chapter 7: Global Market Analysis by Application

Chapter 8: Manufacturing Cost Analysis

Chapter 9: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10: Marketing Strategy Analysis, Distributors/Traders

Chapter 11: Gene Therapy for Inherited Genetic Disorders Market Effect Factors Analysis

Chapter 12: GlobalGene Therapy for Inherited Genetic Disorders Market Forecast to 2027

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Research on Gene Therapy for Inherited Genetic Disorders Market (impact of COVID-19) with Top Players: BioMarin Pharmaceutical Inc., bluebird bio...

MINNEAPOLIS, June 10, 2020 /PRNewswire/ -- Be The Matchtoday announced the appointment of Amy Ronneberg as chief executive officer (CEO)the first female CEO for the organization. Ronneberg joined Be The Match in 2013 as chief financial officer (CFO) and chief of staff, and has served as acting CEO since February.

Be The Match is a non-profit organization that delivers cures to patients in need of life-saving blood stem cell therapy. The organization operates the national Be The Match Registry,the world's largest and most diverse listing of potential stem cell donors. As trusted leaders in advancing stem cell therapy, Be The Match also provides ground-breaking research, innovative technologies, patient support, and education so even more lives can be saved.

Around the same time Ronneberg joined Be The Match as CFO, she was diagnosed with breast cancer. Now cancer-free, she brings unique passion and perspective to her work.

"As a cancer survivor, I have personally felt what many other cancer patients and their loved ones encounter every day. My experience, the patients we serve, and our outstanding, dedicated employees are my motivation," Ronneberg said. "I am deeply honored to be selected to lead this organization and look forward to further advancing our commitment to providing equal outcomes for all. The recent pandemic has put a spotlight on healthcare inequalities, and we are uniquely positioned to address one glaring disparity that is affecting ethnically diverse patientsthat is the ability to find a matching donor. I look forward to leading our team in diversifying the registry and delivering more life-saving treatments to patients of all backgrounds."

After the Affordable Care Act was implemented, Ronneberg led a Be The Match budget restructure that netted a sustainable $50 million cost reduction and made marrow transplants more affordable. Those efforts and more earned her one of the Minneapolis/St. Paul Business Journal's 2016 CFO of the Year awards. Ronneberg also played a pivotal role in establishing Be The Match's operations in Mexico.

This spring, the Be The Match Board of Directors worked with an outside firm to identify the qualities needed in its next CEO and unanimously agreed that Ronneberg possesses all of them.

"Amy has the perfect blend of business experience, passion and health care expertise to lead Be The Match," said Chair of the Board David L. Porter, M.D. "She likes to say, 'patients first, employees always.' I applaud her commitment to carrying out our mission and putting people first."

Ronneberg is also responsible for Be The Match BioTherapies, which partners with global cell and gene therapy industry leaders to deliver high-quality, consistent and compliant therapies to patients in need. Under her guidance, Be The Match BioTherapies has invested in several therapeutics companies and launched an integrated supply chain platform called MatchSource.

Prior to joining Be The Match, Ronneberg spent 12 years at Capella Education Company where she served as chief accounting officer, vice president of finance and led enterprise-wide operations and customer service. She also worked for Ernst & Young as an audit manager.

Ronneberg has served on several boards and is currently on the board of Magenta Therapeutics (MGTA) and the Finance Committee for Allina Healthcare and the World Marrow Donor Association.

Ronneberg earned a Master of Business Administration from Capella University, Minneapolis, Minn. and a Bachelor of Business Administration in Accounting from University of Wisconsin-Eau Claire.

"Going through treatment with my husband and young children at my side made me a much stronger person, leader and decision maker," Ronneberg said. "Cancer is an unfortunate reality that millions of people have to face, but with support, hope and the right medical tools, it can be overcome."

About Be The MatchFor people with life-threatening blood cancerslike leukemia and lymphomaor other diseases, a cure exists. Be The Match connects patients with their donor match for a life-saving marrow or umbilical cord blood transplant. People can contribute to the cure as a member of the Be The Match Registry, financial contributor or volunteer. Be The Match provides patients and their families one-on-one support, education, and guidancebefore, during and after transplant.

Be The Match is operated by the National Marrow Donor Program (NMDP), a nonprofit organization that matches patients with donors, educates health care professionals and conducts research through its research program, CIBMTR (Center for International Blood and Marrow Transplant Research), so more lives can be saved. To learn more about the cure, visit BeTheMatch.orgor call 1 (800) MARROW-2.

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Be The Match Appoints Amy Ronneberg as Chief Executive Officer - Southernminn.com

Related ResearchSarepta Therapeutics Inc., a leader in precision genetic medicine for rare diseases, plans to invest over $30 million to expand its Gene Therapy Center of Excellence in Columbus, Ohio. The project is expected to create 100 new jobs.

Pending state approvals, Sarepta plans to open its new center in an 85,000-square-foot facility at 3435 Stelzer Road in Columbus. All employees currently operating out of the Companys Dublin, Ohio, office will transition to the new facility over time. Armed with the most advanced science in genetic medicine, the new center will allow Sarepta to lead the way in making Columbus an epicenter of gene therapy research and development. In addition to the new building, the companys investment will be used toward R&D equipment.

Sarepta is emerging as the world leader in gene therapy to treat and transform lives otherwise diminished and stolen by rare genetic disease. We are confident that gene therapy will revolutionize genetic medicine, and we chose Ohio for our Gene Therapy Center of Excellence because we believe Columbus will become a hub for genetic medicine innovation, said Sarepta President & CEO Doug Ingram. Our new center will strengthen the position of Columbus as a gene therapy leader, building on the advances of our long-standing partner, Nationwide Childrens Hospital.

We are proud to be the home of Sareptas new Gene Therapy Center of Excellence and to support their continued innovation within a critical field in medicine, said Columbus Mayor Andrew J. Ginther. The companys investment in Columbus is a testament to why our city and region are positioned to become the most important place in the world for gene therapy development.

From medical breakthroughs to cutting-edge technology, the Columbus Region is home to one of the top healthcare industries in the country. With several renowned healthcare systems and companies, including OhioHealth, Nationwide Childrens Hospital and Cardinal Health, the Columbus Region employs more than 45,000 in the industry. The Regions large pool of IT talent, combined with its well-established healthcare sector, makes it a hotbed for innovation.

Sarepta is one of the nations fastest growing biotechnology companies focused on the development of precision genetic medicines, said JobsOhio President and CEO J.P. Nauseef. With Ohio talent, Sarepta will advance its R&D of neuromuscular and central nervous system diseases at its new gene therapy R&D Center in the Columbus Region.

Founded in 1980, Sarepta develops genetic medicines to treat rare diseases, and currently has two approved products for Duchenne muscular dystrophy (DMD) and a pipeline of more than 40 treatments in development. The companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing.

Headquartered in Cambridge, Massachusetts, Sarepta has locations across four continents, and its current Central Ohio research facility in Dublin employs approximately 40 full-time workers.

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Sarepta Therapeutics Expands Gene Therapy Center of Excellence in Columbus, Ohio - Area Development Online

The report covers the forecast and analysis of the Cell and Gene Therapy Consumables market on a global and regional level. The study provides historical data from 2015 to 2018 along with a forecast from 2019 to 2027 based on revenue (USD Million). The study includes drivers and restraints of the Cell and Gene Therapy Consumables market along with the impact they have on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the Cell and Gene Therapy Consumables market on a global level.

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In order to give the users of this report a comprehensive view of the Cell and Gene Therapy Consumables market, we have included a competitive landscape and an analysis of Porters Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are bench marked based on their market size, growth rate, and general attractiveness.

The report provides company market share analysis to give a broader overview of the key players in the market. In addition, the report also covers key strategic developments of the market including acquisitions & mergers, new service & product launches, agreements, partnerships, collaborations & joint ventures, research & development, and regional expansion of major participants involved in the market on a global and regional basis.

The study provides a decisive view of the Cell and Gene Therapy Consumables market by segmenting the market based on product type, application/therapeutics, and regions. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2027. The regional segmentation includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

A rise in the awareness about the gene & cell therapies will propel the market growth during the period from 2019 to 2027. Nevertheless, conducting randomized control tests will inhibit the expansion of the market during the forecast timeline. However, the growing trend for treating neurodegenerative ailments through the use of gene treatment will proliferate the market growth over the forecast period.

The expansion of the market during the forecast timespan is owing to the high frequency of chronic ailments including cancer and heart disorders. Apart from this, inflation in the occurrence of these disorders produces lucrative demand for enhanced therapies and this will culminate in the market demand over the forecast timespan.

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Based on the product type, the market is sectored into Kits & Buffers, Diagnostic Assay, Culture Medium, and Cryopreservation Media. Application/ Therapeutics- wise, the market for cell and gene therapy consumables are classified into Cardiovascular, Urology, Dermatology, Critical Care, Respiratory, Endocrine & Metabolic, Neuroscience, Hematology & Oncology, Obstetrics, Immunology, and Gastroenterology.

Some of the key players in the market include Amgen Inc., ATLANTA BIOLOGICALS, bluebird bio, Inc., Cook, Dendreon Pharmaceuticals, LLC, Fibrocell Science, Inc., General Electric, Kolon TissueGene, Inc., Orchard Therapeutics plc., Pfizer, Inc., PromoCell GmbH, RENOVA THERAPEUTICS, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., Vericel, Helixmith Co., Ltd., and Vitrolife.

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Cell and Gene Therapy Consumables Market Top Key Players, Drivers and Trends to 2027 - Cole of Duty

The Global Gene Therapy Market was estimated to be valued at USD XX million in 2019 and is projected to reach USD XX million by 2026, at a CAGR of XX% during 2019 to 2026.

The market is primarily driven by increase in funding for R&D activities pertaining to gene therapy and increase in awareness regarding gene therapy.

In addition, increase in government support, ethical acceptance of gene therapy for cancer treatment, and rise in prevalence of cancer is likely to fuel the market growth.

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Development policies and plans are discussed as well as manufacturing processes and cost structures are also analyzed. This report also states import/export consumption, supply and demand Figures, cost, price, revenue and gross margins.

Top Key Companies Analyzed inGlobal Gene Therapy Market are Novartis, Kite Pharma Inc., GlaxoSmithKline PLC, Spark Therapeutics Inc., Bluebird bio Inc., Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica and NewLink Genetics Corp

Key Benefit of This Report:

Global Gene Therapy Industry 2019 Market Research Report is spread across 121 pages and provides exclusive vital statistics, data, information, trends and competitive landscape details in this niche sector.

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Target Audience:

Research Methodology:

The market is derived through extensive use of secondary, primary, in-house research followed by expert validation and third party perspective, such as, analyst reports of investment banks. The secondary research is the primary base of our study wherein we conducted extensive data mining, referring to verified data sources, such as, white papers, government and regulatory published articles, technical journals, trade magazines, and paid data sources.

For forecasting, regional demand & supply factors, recent investments, market dynamics including technical growth scenario, consumer behavior, and end use trends and dynamics, and production capacity were taken into consideration.

Different weightages have been assigned to these parameters and quantified their market impacts using the weighted average analysis to derive the market growth rate.

The market estimates and forecasts have been verified through exhaustive primary research with the Key Industry Participants (KIPs), which typically include:

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Major Points Covered in Table of Contents:

1 Introduction

2 Research Methodology

3 Executive Summary

4 Global Gene Therapy Market Overview

5 Global Gene Therapy Market, by Vector

6 Global Gene Therapy Market, by Application

7 Global Gene Therapy Market by Region

8 Competitive Landscape

9 Company Profiles

10 Key Insights

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COVID-19 Impact on Global Gene Therapy Industry 2020: Market Trends, Size, Share, Growth Applications, SWOT Analysis by Top Key Players and Forecast...

- Six-month data from low-dose cohort expected in Q4 2020

- IND amended to include Type I (infantile onset) patients and to evaluate a higher dose - Expect to initiate high-dose cohort in 2H 2020

NEW YORK and BASEL, Switzerland, June 08, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (NASDAQ: AXGT), a clinical-stage company developing innovative gene therapies for neurological diseases, today announced that it has completed enrollment in the low-dose cohort of the Phase 1/2 (Stage 1”) study for Type II (late infantile and juvenile onset) GM1 patients evaluating safety, tolerability, and exploratory measures of efficacy at a dose of 1.5x1013 vg/kg delivered intravenously. Currently, the study is on track to report 6-month data (n=5) from the low-dose cohort of the AXO-AAV-GM1 clinical trial by Q4 2020. Additionally, the investigational new drug (IND) filing has been amended to expand the program to include Type I (infantile) patients and to evaluate a 3-fold higher dose (4.5x1013 vg/kg). The Company expects to initiate dosing in the high-dose cohort, which will include both Type I and Type II patients, in the second half of 2020.

The successful enrollment of the low-dose cohort of the ongoing Phase 1/2 study amidst the COVID-19 pandemic speaks to the dedication of all involved and the significant unmet need that exists for these children,” said Dr. Gavin Corcoran, chief R&D officer. With an expanded study protocol that now includes infantile-onset patients, AXO-AAV-GM1 is the only gene therapy in development to include both Type I and Type II GM1 patients, populations of children who suffer from a deficiency in the same enzyme, -galactosidase. We are grateful for the collaboration and perseverance of the National Institutes of Health (NIH), patients, and their families to advance efforts towards finding a treatment for this devastating pediatric disease.”

This study is being conducted at the NIH under the direction of Dr. Cynthia Tifft, Deputy Clinical Director at the National Human Genome Research Institute (NHGRI) in collaboration with Axovant Gene Therapies. In late 2019, the Company presented an update from the first GM1 Type II child dosed with AXO-AAV-GM1 under an expanded access protocol who was observed to have clinically significant improvements from baseline gene transfer to six-month follow-up based on neurological exam, the Vineland-3 scale, Clinical Global Impression assessments, and nutritional status. In addition, AXO-AAV-GM1 was observed to be generally well-tolerated with no reports of serious adverse events related to the investigational gene therapy or intravenous administration of the vector.

GM1 gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene leading to impaired production of the -galactosidase enzyme. There are currently no approved treatments for GM1 gangliosidosis.

AXO-AAV-GM1 was granted orphan drug designation (ODD) by the U.S. Food and Drug Administration (FDA) in November 2019.

About AXO-AAV-GM1

AXO-AAV-GM1 is an investigational gene therapy that delivers a functional copy of the GLB1 gene via an adeno-associated viral (AAV) vector, with the goal of restoring -galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1’s ability to improve -galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), and Parkinson’s disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

In 2018, Axovant licensed exclusive worldwide rights from the University of Massachusetts Medical School (UMMS) for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases. A three-way Cooperative Research and Development Agreement (CRADA) among Axovant, the NHGRI, and the University of Massachusetts was established in 2019 to support the conduct of the clinical program.

About the University of Massachusetts Medical School

The mission of the University of Massachusetts Medical School is to advance the health and well-being of the people of the commonwealth and the world through pioneering education, research, public service and health care delivery.

Research into potential therapies for lysosomal storage diseases such as Tay-Sachs, Sandhoff disease and GM1 gangliosidosis at UMass Medical School and Auburn University has led to significant advances in the field. Miguel Sena-Esteves, PhD, associate professor of neurology at UMass Medical School; Heather Gray-Edwards, PhD, DVM, formerly of Auburn and currently assistant professor of radiology at UMass Medical School; and Douglas Martin, PhD, professor of anatomy, physiology and pharmacology in the College of Veterinary Medicine and the Scott-Ritchey Research Center at Auburn University, have worked collaboratively for more than a decade on animal models and therapeutic approaches for these and similar disorders. For more information, visit http://www.umassmed.edu.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "anticipate," "will," "would," "should," "expect," "believe," "estimate," and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding the initiation, timing, progress, and reporting of clinical data for its clinical programs, are forward-looking. All forward-looking statements are based on estimates and assumptions by Axovant’s management that, although Axovant believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Axovant expected. Such risks and uncertainties include, among others, the initiation and conduct of the AXO-AAV-GM1 program and the availability of data for disclosures; Axovant’s scientific approach and general development, manufacturing and regulatory progress; Axovant’s ability to perform under its existing clinical and business collaborations; and risks of unforeseen operational delays and other uncertainties caused by the COVID-19 pandemic. These statements are also subject to a number of material risks and uncertainties that are described in Axovant’s most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on February 10, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Axovant undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Media and Investor Contact: Parag Meswani Axovant Gene Therapies (212) 547-2523 investors@axovant.com media@axovant.com

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Axovant Completes Enrollment of Low-Dose Cohort in Phase 1/2 Study of AXO-AAV-GM1 and Expands Study to Include Type I (Infantile Onset) Patients with...

DUBLIN--(BUSINESS WIRE)--The "Bioreactors Market Forecast to 2027 - COVID-19 Impact and Global Analysis by Cell; Molecule; Technology; End User, and Geography" report has been added to ResearchAndMarkets.com's offering.

The bioreactors market was valued at US$ 2,958.50 million in 2019 and is projected to reach US$ 5,169.01 million by 2027; it is expected to grow at a CAGR of 7.3% from 2020 to 2027. The increasing adoption of single-use bioreactors, and rapid growth of the pharmaceuticals and biotechnology industries are among the prime factors driving the bioreactors market. However, stringent regulatory frameworks hinder the growth of the market.

The use of single-use bioreactors has increased in the modern biopharmaceutical processes in the last few years. This can be attributed to their unique ability to allow enhanced process flexibility, reduce investment requirements, and limit operational costs. Also, many companies have developed single-use bioreactors for the production of a wide range of therapeutics. For instance, Distek Inc., has developed a benchtop single-use bioreactor system for recombinant protein production. Single-use bioreactors reduce the risks of contamination and decrease production turnaround times. Moreover, the reduction in validation time has been one of the prime benefits of single-use bioreactors. The rising adoption of single-use bioreactors for upstream bioprocessing is driving the growth of the market.

The global bioreactors market is segmented on the basis of cell, molecule, technology, and end user. The bioreactors market, by molecule, is segmented into monoclonal antibodies, vaccines, recombinant proteins, stem cells, gene therapy, and others. The monoclonal antibodies segment held the largest share of the market in 2019. However, the stem cell segment is projected to register the highest CAGR in the market during the forecast period. Based on cell, the bioreactors market is segmented into mammalian cells, bacterial cells, yeast cells, and others. Based on technology, the market is segmented into wave-induced motion sub, stirred sub, single-use bubble column, and others. Based on end user, the market is segmented into research and development organizations, biopharma manufacturers, contract manufacturing organizations (CMOs).

Key report benefits:

Key Topics Covered:

1. Introduction

2. Bioreactors Market - Key Takeaways

3. Research Methodology

4. Global Bioreactors Market- Market Landscape

4.1 Overview

4.2 PEST Analysis

4.3 Expert Opinion

5. Global Bioreactors Market - Key Market Dynamics

5.1 Market Drivers

5.1.1 Increasing Adoption of Single-Use Bioreactors

5.1.2 Rapid Growth of Pharmaceutical and Biotechnology Industry

5.2 Market Restraints

5.2.1 Stringent Regulatory Framework

5.3 Market Opportunities

5.3.1 Rising Demand of Personalized Medicine

5.4 Future Trends

5.4.1 Technological Advancements in Bioreactors

5.5 Impact analysis

6. Bioreactors Market- Global Analysis

7. Bioreactors Market- By Cell

7.1 Overview

7.2 Bioreactors Market Share, by Cell, 2019 and 2027 (%)

7.3 Mammalian Cells

7.4 Bacterial Cells

7.5 Yeast Cells

7.6 Others

8. Bioreactors Market- By Molecule

8.1 Overview

8.2 Bioreactors Market Share, by Molecule, 2019 and 2027 (%)

8.3 Monoclonal Antibodies

8.4 Vaccines

8.5 Recombinant Proteins

8.6 Stem Cells

8.7 Gene Therapy

8.8 Others

9. Global Bioreactors Market Analysis- By Technology

9.1 Overview

9.2 Global Bioreactors Market, By Technology 2019 & 2027 (%)

9.3 Wave-Induced Motion SUB

9.4 Stirred SUB

9.5 Single-Use Bubble Column

9.6 Other Technologies

10. Global Bioreactors Market Analysis- By End User

10.1 Overview

10.2 Global Bioreactors Market, By End User 2019 & 2027 (%)

10.3 Research And Development Organizations

10.4 Biopharma Manufacturers

10.5 Contract Manufacturing Organisations (CMOs)

11. Bioreactors Market - Geographical Analysis

12. Bioreactors Market - Industry Landscape

12.1 Overview

12.2 Growth Strategies Done by the Companies in the Market, (%)

12.3 Organic Developments

12.3.1 Overview

12.4 Inorganic Developments

12.4.1 Overview

13. Company Profiles

14. Appendix

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Global Bioreactors Market Forecast to 2027 - COVID-19 Impact and Analysis by Cell, Molecule, Technology, End User and Geography -...

The latest report pertaining to The Future of Gene Therapy Market collated by Market Study Report, LLC, provides a detailed analysis regarding market size, revenue estimations and growth rate of the industry. In addition, the report illustrates the major obstacles and newest growth strategies adopted by leading manufacturers who are a part of the competitive landscape of this market.

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According to a new report, the global gene therapy market is anticipated to reach USD 4,300 million by 2021. The demand for gene therapy is primarily driven by continuous technological advancements and successful progression of several clinical trials targeting treatments with strong unmet need. Moreover, rising R&D spend on platform technologies by large and emerging biopharmaceutical companies and favorable regulatory environment will accelerate the clinical development and the commercial approval of gene therapies in the foreseeable future. Despite promise, the high cost of gene therapy represents a significant challenge for commercial adoption in the forecast period.

Gene therapy offers promise in the treatment of range of indications in cancer and genetic disorders. Large Pharmaceuticals and Biotechnology companies exhibit strong interest in this field and key among them include Allergan, Shire, Biomarin, Pfizer and GSK. The gene therapy space is witnessing a wave of partnerships and alliances. Pfizer has recently expanded its presence in gene therapy with the acquisition of Bamboo Therapeutics and Allergan entered the field, with the acquisition of RetroSense and its Phase I/II optogenetic program.

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North America holds a dominating position in the global gene therapy market which is followed by Europe and the Asia Pacific. The U.S. has maximum number of clinical trials ongoing followed by Europe. Moreover, the field of gene therapy in the U.S. and Europe continues to gain investor attention driven by success of high visible clinical programs and the potential of gene therapy to address strong unmet need with meaningful commercial opportunity. Moreover, the increasing partnerships and alliances and the disruptive potential of gene therapy bodes well for the sector through the forecast period.

Gene therapy involves inactivating a mutated gene that is not functioning properly and introducing a new gene to assist in fighting a disease. Overall, the field of gene therapy continues to mature and advance with many products in development and nearing commercialization. For instance, Spark Therapeutics received approval of Luxturna, a rare form inherited blindness in December 2017. Gene therapy market in late 2017 also witnessed the approvals of Gilead/Kite Pharmas Yescarta and Novartis Kymriah in the cancer therapeutic area.

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Key Findings from the study suggest products accessible in the market are much competitive and manufacturers are progressively concentrating on advancements to pick up an aggressive edge. Companies are in a stage of development of new items in order to guarantee simple implementation and connection with the current gene. The hospatility segment is anticipated to grow at a high growth rate over the forecast period with the expanding utilization of smart locks inferable from expanding security-related worries among clients amid their stay at the hotels. North America is presumed to dominate the global smart locks market over the forecast years and Asia Pacific region shows signs of high growth owing to the booming economies of India, and China.

The Future of Gene Therapy Market share byMajor regions included:

United StatesNorth AmericaAsia PacificEuropeMiddle East & Africa

Table of Contents

1.Gene Therapy Overview1.1.History and Evolution of Gene Therapies1.2.What is Gene Therapy1.3.Types of Gene Therapy1.4.Ex vivo and in vivo Approaches of Gene Therapy1.5.RNAi Therapeutics1.6.CAR-T Technology based Gene Therapy1.7.Types of Vectors used for Gene Therapy1.7.1.Viral1.7.2.Non-Viral2.Historical Marketed Gene Therapies [2003-2012]2.1.Rexin-G (Epeius Biotechnologies Corporation)2.2.Gendicine (SiBiono GeneTech Co., Ltd)2.3.Neovasculgen [Human Stem Cells Institute (HSCI))2.4.Glybera (UniQure Biopharma B.V.)3.First Countries to get an access to Gene Therapies3.1.Philippines for Rexin-G [2003]3.2.China for Gendicine [2003]3.3.Russia for Neovasculgen [2011]3.4.Selected European Countries for Glybera [2012]4.Marketed Gene Therapies [Approved in Recent Years]4.1.KYMRIAH (tisagenlecleucel)4.1.1.Therapy Description4.1.2.Therapy Profile4.1.2.1.Company4.1.2.2.Approval Date4.1.2.3.Mechanism of Action4.1.2.4.Researched Indication4.1.2.5.Vector Used4.1.2.6.Vector Type4.1.2.7.Technology4.1.2.8.Others Development Activities4.1.3.KYMRIAH Revenue Forecasted till 20214.2.YESCARTA (axicabtagene ciloleucel)4.2.1.Therapy Description4.2.2.Therapy Profile4.2.2.1.Company4.2.2.2.Approval Date4.2.2.3.Mechanism of Action

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Global and Regional The Future of Gene Therapy Market Research 2018 Report | Growth Forecast 2026 - Medic Insider

XconomyBoston

Hearing loss can stem from a variety of reasons, but in rare cases its caused by genetic defects. Akouos is developing a gene therapy to address these problems and its planning an IPO to finance the first tests of its approach in humans.

In paperwork filed with regulators late Friday, Boston-based Akouos set a preliminary $100 million goal for its IPO.

The first target for Akouos is hearing loss due to mutations in the OTOF gene, which encodes a protein called otoferlin. Hearing happens when tiny hair cells in the inner ear pick up vibrations and turn them into signals that the brain interprets as sound. Otoferlin is key to this process, as it enables the sensory cells of the ear to release tiny membranes carrying neurotransmitters, Akouos says in its filing. These neurotransmitters activate auditory neurons, which in turn relay the information to the brain where it is recognized as sound.

Those who have OTOF gene mutations are typically born deaf. There are currently no FDA-approved therapies for this form of hearing loss. The experimental Akouos gene therapy, AK-OTOF, is intended to deliver a functioning version of the OTOF gene with the goal of restoring proper otoferlin expression.

The Akouos therapy is delivered using an engineered version of adeno-associated virus (AAV), an approach used by some FDA-approved gene therapies marketed by other companies for other diseases, as well as for some gene therapies still in development. But the anatomy of the inner ear presents challenges for AAV gene therapy delivery, the company says in its filing. Also, AAV has a limited capacity for carrying a genetic payload.

To overcome those limitations, Akouos uses synthetic AAVs that recreate naturally occurring viruses called ancestral AAVs, which the company says can reach the target in the ear. Furthermore, the company uses a dual vector approach that employs two engineered viruses, each carrying a fragment of the OTOF gene. The Akouos approach to gene therapy is based on the research of Luk Vandenberge, director of the Grousbeck Gene Therapy Center in Boston and a professor at Harvard Medical School. In mouse studies, Akouos says AK-OTOF delivered its genetic payload to the inner ear hair cells, restoring auditory function. Now the company wants to test its technology in humans.

Paperwork seeking FDA permission to start a clinical trial is on track to be submitted in 2021, Akouos says in its filing. If all goes as planned, the company expects to report preliminary data from the clinical trial the following year.

Akouos isnt the only company developing new treatments for inherited forms of hearing loss.Decibel Therapeutics is also developing a gene therapy to address the same genetic protein deficiency that Akouos is targeting. The Boston-based companys experimental gene therapy, DB-OTO, is in preclinical development with partner Regeneron Pharmaceuticals (NASDAQ: REGN). Otonomy (NASDAQ: OTIC), in San Diego, is advancing a preclinical gene therapy program intended to address hearing loss caused by mutations in another key gene, GJB2. Frequency Therapeutics (NASDAQ: FREQ) is addressing hearing loss caused by the loss of hair cells in the inner ear. The Woburn, MA-based company is developing a regenerative medicine approach that coaxes the regrowth of these hairs.

Akouos was founded in 2016 and unveiled $50 million in financing two years later. In March, the company closed a $105 million Series B round of financing that added crossover investors. The biggest shareholders in Akouos prior to the IPO are 5AM Ventures and New Enterprise Associates, which own about 21.6 and 18.5 percent respectively, according to the filing.

Akouos has applied for a listing on the Nasdaq under the stock symbol AKUS. If the company successfully completes the IPO, it says it will apply the capital toward clinical development of AK-OTOF, as well as the preclinical development of other programs in its pipeline.

Image: iStock/iLexx

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

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Akouos Aims for an IPO to Advance Hearing Loss Gene Therapy to Clinic - Xconomy

Knowing what cancer will do next could lessen the likelihood of it becoming resistant to treatment. A new U of T study investigates how cancer adapts its metabolism to potentially overcome therapies still in development.

Several clinical trials have failed because metabolism is such an adaptive process by which cancer cells gain drug resistance, saysMichael Aregger, a co-lead author and Research Associate working withJason Moffat, Professor of molecular genetics in the Donnelly Centre for Cellular and Biomolecular Research, who co-led the work. If you know how cells are able to adapt to perturbations, maybe we can target them more specifically to avoid resistance from developing.

The research was also led byBrenda AndrewsandCharles Boone, University Professor and Professor of molecular genetics at the Donnelly Centre, respectively, andChad Myers, a Professor of computer science at the University of Minnesota-Twin Cities.

Thestudy, published this week in the journalNature Metabolism, is the first to investigate global changes in cancerous cells as they adapt to a shortfall of critical nutrients such as fat molecules, or lipids, which make up the cells outer envelope.

When cancer cells are unable to make their own lipids, they gobble them up from their environment to ensure a steady supply of these essential building blocks, the study found. Lipids also serve as fuel and chemical signals for communication between cells, among other roles.

"If you know how cells are able to adapt to perturbations, maybe we can target them more specifically to avoid resistance from developing" - Michael Aregger, Research Associate

The switch in metabolism could be bad news for drugmakers seeking to target cancer by reducing its lipid reserves. In particular, drugs that inhibit an enzyme called FASN, forfattyacidsynthase, involved in an early step of lipid synthesis, are being explored in patient trials. Fatty acids are precursors of larger lipid molecules and their production is increased in many cancers thanks to elevated FASN levels, which are also associated with poor patient prognosis.

The U of T study suggests that the effectiveness of FASN inhibitors could be short-lived owing to cancers ability to find another way to procure lipids.

Because FASN is upregulated in many cancers, fatty acid synthesis is one of the most promising metabolic pathways to target saysKeith Lawson, a co-lead author and PhD student in Moffats lab enrolled in the Surgeon-Scientist Program at the Faculty of Medicine. Given that we know there is a lot of plasticity in metabolic processes, we wanted to identify and predict ways in which cancer cells can potentially overcome the inhibition of lipid synthesis.

To block fatty acid synthesis, the researchers employed a human cell line from which the FASN coding gene was removed. Using the genome editing tool CRISPR, they deleted from these cells all ~18,000 or so human genes, one by one, to find those that can compensate for the halt in lipid production. Such functional relationships are also referred to as genetic interactions.

Data analysis, performed byMaximilian Billmann, a co-lead author and a postdoctoral fellow in Myers lab at Minnesota-Twin Cities, revealed hundreds of genes that become essential when cells are starved of fat. Their protein products clustered into well-known metabolic pathways through which cells hoover up dietary cholesterol and other lipids from their surroundings.

Cells intake of cholesterol has become textbook knowledge since it was discovered half a century ago, winning aNobel Prizeand inspiring the blockbuster drug statin and many others. But the new study found that one component of this process remained overlooked all this time.

The gene encoding it was only known as C12orf49, named after its location on chromosome 12. The researchers re-named the gene LUR1, forlipiduptakeregulator 1, and showed that it helps switch on a set of genes directly involved in lipid import.

This was a big surprise to us that we were able to identify a new component of the process we thought we knew everything about, says Aregger. It really highlights the power of our global genetic interaction approach that allowed us to identify a new player in lipid uptake in a completely unbiased way.

By a remarkable coincidence, two groups working independently in New York and Amsterdam also linked C12orf49 to lipid metabolism, lending further support for the genes role in this process. The New York team published their findings in the same journal issue as Moffat and colleagues.

Inhibiting LUR1, or other components of lipid import, along with FASN could lead to more effective cancer treatments. Such combination therapies are thought to be less susceptible to emerging drug resistance because the cells would have to simultaneously overcome two obstaclesblocked lipid production and importwhich has a lower probability of occurring.

Therapeutic context that comes out of our work is that you should be targeting lipid uptake in addition to targeting lipid synthesis and our work highlights some specific genes that could be candidates, says Lawson.

The research was supported by the Canadian Institutes for Health Research, Ontario Research Fund, Canada Research Chairs Program and the U.S. National Institutes of Health.

Reference:Aregger, M., Lawson, K.A., Billmann, M. et al. (2020) Systematic mapping of genetic interactions for de novo fatty acid synthesis identifies C12orf49 as a regulator of lipid metabolism. Nat Metab. DOI: https://doi.org/10.1038/s42255-020-0211-z

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

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When Cancer Cells Cant Produce Their Own Fat, They Import More of It - Technology Networks

By Charles PillerJun. 8, 2020 , 7:00 PM

Sciences COVID-19 reporting is supported by the Pulitzer Center.

Three unlikely collaborators are at the heart of the fast-moving COVID-19 research scandal, which led to retractions last week by The Lancet and The New England Journal of Medicine (NEJM), and the withdrawal of an online preprint, after the trove of patient data they all relied on was challenged. The three physician-scientists never were at the same institution nor had they ever before written together, but they are the only authors in common on the disputed papers, and the other co-authors all have ties to at least one of them. Their partnership, which seized a high-impact role during a global public health crisis, has now ended disastrously.

The first author for both retracted papers was cardiac surgeon Mandeep Mehra, an eminent Harvard University professor who works at Brigham and Womens Hospital (BWH) and is known internationally for cardiovascular medicine and heart transplants. He provided the kind of gravitas that can fast-track papers to leading journals. In a statement provided by BWH, Mehra said he had met another of the trio, cardiac surgeon Amit Patel, in academic and medical circles, and that Patel had introduced him to Sapan Desai, a vascular surgeon and founder of Surgisphere, the tiny company that supplied the data. Journal disclosures, however, also indicate Mehra received compensation from Triple-Gene, a gene therapy company Patel co-founded to develop cardiovascular treatments.

Desai publicly aspired to combine big data and artificial intelligence (AI) in ways that he said can replace randomized controlled clinical trials. For a brief moment, it seemed that Surgispheres enticing data set, said to include nearly 100,000 detailed patient records from about 700 hospitals on six continents, would settle questions about the possible benefits of various drugsincluding the controversial antimalarial hydroxychloroquinefor COVID-19 patients.

Patel once apparently headed cardiac surgery at the University of Miami Miller School of Medicine. A university press release announcing his arrival in 2016 is no longer posted on the university website, however, and the school has not confirmed his job duties there. More recently, he has been a volunteer adjunct professor at the University of Utah. But, as STAT first reported yesterday, Patel tweeted on Friday that he had severed his relationship with the university, which a school spokesperson confirmed. In recent years Patel has developed and commercialized experimental stem cell therapies purported to cure heart problems, reverse aging, or treat sexual dysfunction. He is also part of a network of physicians that just launched a trial to use stem cells from umbilical cord blood to treat COVID-19 patients.

Normally co-authors of high-profile papers share subject area expertise or have clear professional ties, says Jerome Kassirer, chief editor ofNEJMduring the 1990s. He calls the collaboration of the apparently disparate individuals completely bizarre, and a red flag that the studies warranted intensive scrutiny that the journals failed to provide.

None of the three co-authors responded to requests for comment. Patel spoke with aSciencereporter initially but said he wanted to wait for audits of the Surgisphere data to comment, and Desais spokesperson stopped communicating after the retractions. Still, interviews with former colleagues and a long paper trail shed some light on each of them.

Desai had a history of convincing respected researchers of his skill and integrity. One of them, Gilbert Upchurch, department of surgery chair at the University of Florida, wrote last year in a journal commentary that he had never met Desai but had nonetheless mentored him remotely and developed an online friendship with him. Upchurch placed the scientist in a group of amazing and talented young vascular surgeons.

Illinois court records show Desai is facing two medical malpractice lawsuits filed last year. He told The Scientist that he deems any lawsuit naming him to be unfounded.

Desai has a history of big aspirations and entrepreneurial venturessome short-lived. His science-fiction blog, corewardfront.com, was meant to find the most parsimonious route for mankind to establish a meaningful presence in space. In 2009, he wrote that the site would publish fiction grounded in facts and reality, adding, the scientific method must be followed religiously. The blog is no longer published.

As a student, Desai won several small National Institutes of Health (NIH) grants for studies of the vestibular system. He started Surgisphere in 2007, when he was a medical resident at Duke University. Surgispheres initial products were medical guides and textbooks, although Desai has said he was working on big data projects for the company from its birth. In 2010, under the firms auspices, he founded the Journal of Surgical Radiologywhose editors included researchers with well-established publishing records. It folded in January 2013. Articles from the journal were cited only 29 times in its history, according to Scimago, a journal rating service. Yet an undated Surgisphere web page, no longer accessible online, said the online-only publication had 50,000 subscribers and nearly 1 million page views monthlywhich would have placed it in elite company in academic publishing.

Surgisphere appears over time to have shifted its efforts into developing a database of hospital records that could be used for research. When the pandemic erupted, Desai declared that his data set could answer key questions about the efficacy and safety of treatments. Speaking about the finding that hydroxychloroquine increases mortality in COVID-19 patients, the main finding from the now retracted Lancet paper, he told a Turkish TV reporter, with data like this, do we even need a randomized controlled trial? Soon after, the World Health Organization temporarily suspended enrolling patients for its COVID-19 trial of the drug.

Immediately after the Lancet and NEJM studies appeared, however, critics identified anomalies in the data. And they doubted that a tiny firmwith a scant public track record in AI, few employees, and no publicly named scientific boardcould convince hundreds of unidentified hospitals in dozens of nations to share complex, protected, and legally fraught patient data. Ultimately, despite Desai promising repeatedly to allow an independent audit of Surgisphere, the firm refused to release the raw patient data and agreements with hospitals for an audit, so no one could validate the authenticity of its database.

No hospitals have come forward to acknowledge working with Surgisphere. Indeed, NHS Scotland, which is mentioned as a case study on the companys website, says none of its hospitals worked with Surgisphere and that it would ask the firm to remove an image of a Glasgow hospital from its website.

Science contacted several of Desais current or former employees or colleagues. Most would not comment. But Fred Rahimi, an Illinois podiatrist and co-author of a paper with Desai, praises the surgeon as highly capable for salvaging limbs, and easy to work with. Through his publicist, Desai cited Mark Melin, a University of Minnesota, Twin Cities, vascular surgeon, as a supporter. Before the retractions, Melin called Desai a gentleman of the highest integrity who has nothing to cover up.

But one physician-scientist who worked closely with Desai several years ago, says, Just about everyone who knew him would say: I just didnt have a good feeling about him. After theyd been with him, most people dissociated themselves from him, the scientist says, declining to be named to avoid personal and institutional embarrassment.

In the decade since completing his medical residency, Desai moved from job to jobat Duke, the University of Texas, Southern Illinois University, and two private Illinois hospitals, according to his LinkedIn profile. You might say we should have stopped him, which now seems obvious, Desais former colleague says. We should have found a way to get together and say, Whats going on here? rather than allowing him to move from place to place. We should have done better as a medical community. We looked the other way.

Before and after his stint at the University of Miami, which appears to have started in late 2016 or early 2017, Patels academic home was the University of Utah. He started as a full-time faculty member at Utah in 2008 and kept that position until he left for Miami. The website for Foldax, a heart valve company that he serves as medical adviser, describes him as a Tenured Professor of Surgery in the Division of Cardiothoracic Surgery at the University of Utah School of Medicine and Director of Clinical Regenerative Medicine and Tissue Engineering at the University of Utah.

The university confirmed Patel had tenure there, but says the directorship was an unofficial title. And among more than 100 publications listed on his University of Utah profile, nearly two-thirds were actually co-authored by other scientists who share the same surname. The page was removed from the university website after inquiries from Science.

According to the NIH database, Patel has never received funding from the agency. Before the recent COVID-19 papers, one of his most notable publications was a 2016 paper in The Lancet, which reported that extracting stem cells from the bone marrow of a person with end-stage heart failure and then reinjecting them could reduce the number of cardiac events that produced deaths or hospital admissions by 37%. The 126 patient, 31-site, phase II trial was billed in a press release, now not available on the University of Utah website but stored elsewhere, as the largest cell therapy trial for heart failure to date. Despite the apparent positive results, the sponsoring company Vericel no longer is developing stem cells for heart disease and, according to its webpage, is focused on advanced cell therapies for the sports medicine and severe burn care markets.

Patel left Miami under unclear circumstances, but has retained ties with Camillo Ricordi, an influential stem cell researcher at the University of Miami School of Medicine who is also the founder of a nonprofit called the Cure Alliance. The alliance previously focused on testing whether stem cells derived from umbilical cord blood could treat diabetes or Alzheimers, but has now pivoted to fighting COVID-19, according to its website. Ricordi is the principal investigator on a multisite trial to see whether the stem cells can treat lung inflammation in severe COVID-19 patients and Patel is listed in various references to the trial as a key contributor or coprincipal investigator. Ricordi did not reply to requests for comments on his relationship with Patel.

Patel recently tweeted that he is related to Dr. Desai by marriage but called that old news and added, Despite this I still do not have the information of what happened at Surgisphere. In addition to apparently connecting Mehra and Desai, Patel had prior connections with other authors of the NEJM paper and the preprint. David Grainger, co-author of the preprint, is a professor of biomedical engineering at the University of Utah and also works with Foldax. Grainger declined to comment.

Timothy Henry, a cardiovascular clinician and scientist at the Christ Hospital in Cincinnati and a co-author on the NEJM article, has written several scholarly articles with Patel, including the 2016 Lancet paper. Henry, who also declined to comment, advises Patels Triple-Gene, which develops cardiovascular gene therapy treatments. Henry and Patel adviseand Patel is a board member ofCreative Medical Technology Holdings, a Phoenix company that develops and markets stem cell therapies, including treatments purported to reverse aging and cure sexual disfunction.

Creative Medicals CaverStem and FemCelz kits are distributed to physicians who use them to extract stem cells from a patients bone marrow, then inject the cells into the penis or clitoral area to stimulate blood flow, according to a statement filed with the U.S. Securities and Exchange Commission. (As of the market close Friday, the publicly traded firms shares were valued at one-third of 1 cent.) The CaverStem treatments are advertised by the company as successful in more than 80% of patients, based on a 40-person phase I clinical trial that was not randomized or controlled, and on observations of 100 other patients. Phase I trials typically measure safety, not health benefits of a potential treatment.

Science contacted multiple colleagues or co-authors of Patel. None would comment. Before the retractions, two high-profile researchersDeepak Bhatt, who directs interventional cardiovascular programs at BWH; and Peter Gruber, a pediatric cardiothoracic surgeon at Yale Universityendorsed Patel on his LinkedIn page. Bhatt says he doesnt know Patel and attempted to remove his endorsement after being contacted by Science. Gruber says he overlapped with Patel at the University of Utah about a decade ago, but doesnt know his work in detail.

In contrast, Mehraauthor of more than 200 scholarly articles, editor of The Journal of Heart and Lung Transplantation, and head of the cardiology division of theUniversity of Maryland before moving to BWH in 2012enjoys considerable support even after the unraveling of the recent studies. Obviously, you dont rise to the position hes risen to without being ambitious, but Ive never had any indication whatsoever that he would do anything unethical, says Keith Aaronson, a cardiologist at the University of Michigan, Ann Arbor, who collaborated with Mehra on several studies, including a clinical trial of a mechanical pump for heart failure patients.

Mehra, the first author on both retracted papers, was the only one to issue a personal statement of apology, for failing to ensure that the data source was appropriate for this use. BWH and Harvard declined to say whether further investigation of Mehras roles in the papers would occur. (Mehra has written papers recently with another co-author of the Lancet paper, Frank Ruschitzka of University Hospital Zrich.)

I think he just fell into thisperhaps a little navely, says another former collaborator, cardiothoracic surgeon Daniel Goldstein of the Albert Einstein College of Medicine. Given the amount of data that was in the [Surgisphere] database, its just hard to believe someone would [fabricate] something like this.

Kassirer offers a harsher view: If youre a scientist and youre going to sign on to a project, by God you should know what the data are.

With reporting by Kelly Servick and John Travis.

This story was supported by theScienceFundforInvestigativeReporting.

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Who's to blame? These three scientists are at the heart of the Surgisphere COVID-19 scandal - Science Magazine

CSL Behring, based in King of Prussia, Pennsylvania, is acquiring Vancouver, British Columbia-based Vitaeris. The two companies inked a strategic partnership in 2017 to accelerate the development of Vitaeriss clazakizumab. At that time, CLS Behring also had an option to acquire Vitaeris, the therapeutic, and the rest of Vitaeriss assets.

No financial details were disclosed. In the 2017 deal, Vitaeris retained control of projects through the end of Phase III development. There was an upfront cash payment of $15 million, with research-and-development milestone payments over that period, which included future sales-related payments, as well as a royalty to Alder BioPharmaceuticals, who originated clazakizumab.

Clazakizumab is an anti-interleukin-6 (IL-6) monoclonal antibody for the treatment of chronic active antibody-mediated rejection (AMR), which is the top cause of long-term rejection in kidney transplant patients. There are currently no treatments approved for transplant recipients who develop antibody-mediated rejection.

Clazakizumab will join CLS842 and CSL964 as part of CSL Behrings late-stage program related to transplants.

Clazakizumab has been a promising monoclonal antibody in the Transplant therapeutic area since we started working with Vitaeris several years ago, said Bill Mezzanotte, executive vice president, Head of R&D, CSL Behring. Acquiring Vitaeris and their associate expertise helps us to continue to grow our strategic scientific platform of recombinant proteins and antibodies. We look forward to continuing to advance this treatment candidate as a potential option for people experiencing rejectionan area where current treatment options for transplant recipients are limited, at best.

Also today, CSL Behring announced results from a Phase II clinical trial of garadacimab (formerly CSL312), an investigational novel Factor XIIa-inhibitory monoclonal antibody to prevent hereditary angioedema (HAE). The company presented results at the European Academy of Allergy and Immunology (EAACI) Digital Congress 2020.

The trial met the primary endpoint, showing a decreased number of attacks compared to placebo in patients with HAE. HAE is a rare, genetic and potentially life-threatening disease. HAE is one of two types of bradykinin-mediated angioedema, with the other being nonhereditary or acquired angioedema. HAE is the result of deficient or dysfunctional C1-INH, a blood protein that helps control inflammation.

Last month, the U.S. Food and Drug Administration (FDA) granted garadacimab orphan drug designation for bradykinin-mediated angioedema.

The attacks that HAE patients experience can be very frightening, and clinicians want to do anything in their power to reduce the frequency of these attacks, lessen the need for rescue medicine and simplify treatment, said Timothy Craig, lead study investigator with Allergy, Asthma and Immunology, Department of Medicine and Pediatrics, Penn State Hershey, Hershey, Pennsylvania. The findings of this study are very encouraging and we look forward to further research assessing the safety and efficacy of garadacimab.

CSL Behring has been quite busy recently. On June 2 the company announced a strategic alliance with Seattle Childrens Research Institute to develop stem cell gene therapies for primary immunodeficiency diseases. The initial focus will be on Wiskott-Aldrich Syndrome and X-linked Agammaglobulinemia.

The company is also working on several fronts to develop treatments and preventions for COVID-19. The company is part of the CoVIg-19 Plasma Alliance, which is working to develop an unbranded anti-SARS-CoV-2 polyclonal hyperimmune immunoglobulin therapy. The Alliance is also working with the U.S. National Institute of Allergy and Infectious Diseases (NIAID) to test the hyperimmune therapy in adult patients with COVID-19. CSL Behring Australia is developing an anti-SARS-COV-2 plasma product for the Australian market. CSL Behring is also partnered with the Coalition for Epidemic Preparedness Innovations (CEPI) and The University of Queensland (UQ) to speed the development, manufacture and distribution of a COVID-19 vaccine, as well as other initiatives in this space.

And on May 27, 2020, CSL Limited entered into a strategic partnership with Thermo Fisher Scientific to meet the growing demand for biologic therapies while also accelerating CSLs broader manufacturing objectives. Thermo Fisher will leverage its pharma services network to support CSLs product portfolio, and via a long-term lease deal, will operate a new state-of-the-art biologics manufacturing facility in Lengnau, Switzerland, which is being built now and is expected to be completed in mid-2021.

Of the acquisition by CSL Behring, Kevin Chow, president and chief executive officer of Vitaeris, stated, Were pleased to become part of CSL Behring, a well-established, global industry leader, and know that the future of clazakizumab is in excellent hands. Together, we have already achieved much progress through our partnership over the past few years and are now in an even stronger position to realize our collective goal of addressing one of the greatest unmet needs in the organ transplant community.

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CSL Behring Buys Vitaeris, Picks Up Transplant Rejection Therapeutic - BioSpace

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.

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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.,...

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.

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Genprex Scheduled to Join Russell 3000 Index - Business Wire

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

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."

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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.

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Memorial Sloan Kettering Hackensack Meridian Health Partnership Announces Funding for Inaugural Immunology Research Collaboration Projects - Newswise

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.

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Siemens and Exyte join forces to deliver integrated solutions for fast-track construction of smart biotech facilities - ChemEngOnline

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

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How to Solve the Production Challenges of AAVs for Gene... - Labiotech.eu

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

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

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.

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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.

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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

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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...

-- 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...

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

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 - P&T Community

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