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Archive for the ‘Gene Therapy Research’ Category

Maryland Labs Developing Vaccines And Therapeutics, Attracting Federal Dollars – Patch.com

By Bruce DePuytSeptember 10, 2020

A 2016 decision to link the research labs at the University of Maryland College Park with those at the University of Maryland at Baltimore is paying dividends in the competition for federal COVID-19 research dollars, a top education official told a science advisory panel on Wednesday.

The decision was a spinoff of the University of Maryland Strategic Partnership Act, said Laurie Ellen Locasio, vice president for research at the two institutions.

"Now we'll be ranked together by the [National Science Foundation] in the U.S. rankings of research universities and together this makes us [an] over $1 billion public research enterprise," she told the Maryland Life Sciences Advisory Board.

With a multi-billion dollar research powerhouse, the privately-funded Johns Hopkins University, also operating here, Maryland is unusually well-positioned, Locasio said.

"Very few states in the country have the distinction of having two research universities with over a billion dollars in research spending per year. We've really shown up as central to this pandemic."

Martin Rosendale, the CEO of the Maryland Tech Council, said there are so many therapeutics and vaccines in development that keeping up with them all is a daunting task.

Many firms adopted a "drop everything" approach when the scope of the coronavirus challenge started to become apparent, he said.

"We just saw a lot of amazing thing happening here in Maryland so many companies were pivoting immediately to support the response to the pandemic," he said. "They were basically dropping their other work and on their own dime spending their own money evaluating their technologies, their platforms, how they could apply them to COVID-19."

Many of the big players have grabbed global headlines for their work on a possible vaccine. But smaller companies are making potentially valuable strides as well, Commerce Secretary Kelly M. Schulz told the panel.

Other firms are working to develop COVID tests that are faster and more reliable.

The Tech Council formed the Maryland COVID-19 Coalition to bring companies together, "to make sure they knew who each other were and begin a conversation," Rosendale said. Twenty-five firms were part of the coalition's first call; 40 were on the second.

Chris P. Austin, director of the National Center for Advancing Translation Sciences at NIH, told the panel that the federal government has pumped more than $20 billion into COVID research.

"Of that, we know that over $3 billion is coming here to Maryland," Rosendale said. "That's a testament to the amazing vaccine and cell-and-gene therapy industries that have grown up here."

If foundation and other non-government funds are added to the public dollars, "the number is well over $4 billion," he added.

To play off the capital region's strengths in biotech, a group of industry leaders in Maryland, Washington, D.C., and Virginia is organizing a "pandemic and bio-defense center" to help countries around the globe guard against future pandemics.

"Because of the prominence of what's going on with COVID-19 development in our region especially within Maryland with vaccine, diagnostics, and therapeutic research we are sort of the epicenter in the world right now," said Richard A. Bendis, president and CEO of BioHealth Innovation, Inc. in Rockville.

"People are starting to take notice."

Schulz said the Department of Commerce paused its planned marketing efforts in the spring to focus on the pandemic, but now is ready to gear back up again with an "Innovation Uncovered" campaign.

"We're going back to where we wanted to be at the beginning of the year, which was to push the bio- and the life sciences worlds out, because we have heard from [company executives] that Maryland needs to be seen as a strong, tight ecosystem for this industry," she said.

bruce@maryandmatters.org

For more stories from Maryland Matters, visit http://www.marylandmatters.org.

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Maryland Labs Developing Vaccines And Therapeutics, Attracting Federal Dollars - Patch.com

Gene Therapy Market to Eyewitness Massive Growth by 2026: Leading Key Players Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc….

This report focuses on the Global Gene Therapy Market trends, future forecasts, growth opportunities, key end-user industries, and market players. The objectives of the study are to present the key developments of the market across the globe.

The latest research report on Gene Therapy market encompasses a detailed compilation of this industry, and a creditable overview of its segmentation. In short, the study incorporates a generic overview of the Gene Therapy market based on its current status and market size, in terms of volume and returns. The study also comprises a summary of important data considering the geographical terrain of the industry as well as the industry players that seem to have achieved a powerful status across the Gene Therapy market.

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Gene Therapy Market Segmentation

Reports include the following segmentation: By Disease Indication Cancer Genetic disorders Cardiovascular diseases Ophthalmology Neurological conditions OthersBy Type of Vectors Viral vectors Non-viral vectorsBy Type of Cells Somatic cells Germline cellsBy Region North Americao U.S.o Canadao Mexico Europeo UKo Franceo Germanyo Russiao Rest of Europe Asia-Pacifico Chinao South Koreao Indiao Japano Rest of Asia-Pacific LAMEAo Latin Americao Middle Easto Africa

The report has been curated after observing and studying various factors that determine regional growth such as economic, environmental, social, technological, and political status of the particular region. Analysts have studied the data of revenue, production, and manufacturers of each region. This section analyses region-wise revenue and volume for the forecast period of 2015 to 2026. These analyses will help the reader to understand the potential worth of investment in a particular region.

Global Gene Therapy Market: Competitive LandscapeThis section of the report identifies various key manufacturers of the market. It helps the reader understand the strategies and collaborations that players are focusing on combat competition in the market. The comprehensive report provides a significant microscopic look at the market. The reader can identify the footprints of the manufacturers by knowing about the global revenue of manufacturers, the global price of manufacturers, and production by manufacturers during the forecast period of 2015 to 2020.

The major players in the market Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc. Boehringer Ingelheim International GmbH uniQure N.V. bluebird bio, Inc. Celgene Corporation Others

Global Gene Therapy MarketThis research report providesCOVID-19 Outbreakstudy accumulated to offer Latest insights about acute features of the Gene Therapy Market. The report contains different market predictions related to marketsize, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market. It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary andSWOT analysis.It presents the360-degreeoverview of the competitive landscape of the industries. Gene Therapy Market is showing steadygrowthandCAGRis expected to improve during the forecast period.

The main sources are industry experts from the global Gene Therapy industry, including management organizations, processing organizations, and analytical services providers that address the value chain of industry organizations. We interviewed all major sources to collect and certify qualitative and quantitative information and to determine future prospects. The qualities of this study in the industry experts industry, such as CEO, vice president, marketing director, technology and innovation director, founder and key executives of key core companies and institutions in major biomass waste containers around the world in the extensive primary research conducted for this study We interviewed to acquire and verify both sides and quantitative aspects.

Global Gene Therapy Market: Regional AnalysisThe report offers in-depth assessment of the growth and other aspects of the Gene Therapy market in important regions, including the U.S., Canada, Germany, France, U.K., Italy, Russia, China, Japan, South Korea, Taiwan, Southeast Asia, Mexico, and Brazil, etc. Key regions covered in the report are North America, Europe, Asia-Pacific and Latin America.

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Complete Analysis of the Gene Therapy Market:

Comprehensive assessable analysis of the industry is provided for the period of 2020-2025 to help investors to capitalize on the essential market opportunities.

The key findings and recommendations highlight vital progressive industry trends in the global Gene Therapy market, thereby allowing players to improve effective long term policies

A complete analysis of the factors that drive market evolution is provided in the report.

To analyze opportunities in the market for stakeholders by categorizing the high-growth segments of the market

The numerous opportunities in the Gene Therapy market are also given.

Report Answers Following Questions:

What are the factors driving the growth of the market?

What factors are inhibiting market growth?

What are the future opportunities in the market?

Which are the most dynamic companies and what are their recent developments within the Gene Therapy Market?

What key developments can be expected in the coming years?

What are the key trends observed in the market?

TABLE OF CONTENT

1 Report Overview

2 Global Growth Trends

3 Market Share by Key Players

4 Breakdown Data by Type and Application

5 United States

6 Europe

7 China

8 Japan

9 Southeast Asia

10 India

11 Central & South America

12 International Players Profiles

13 Market Forecast 2020-2025

14 Analysts Viewpoints/Conclusions

15 Appendix

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Gene Therapy Market to Eyewitness Massive Growth by 2026: Leading Key Players Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc....

Cancer Gene Therapy Market Size Will Expand Post COVID-19 Covered in Forecast Report 2020-2026 – NJ MMA News

[190+ Pages PDF Report] Facts & Factors (FnF) published a market research report onCancer Gene Therapy Market: By Size, Share, Growth, Analysis Covering COVID-19 Impact and Forecast to 2020-2026that includes a research report with TOC including a list of tables and figures in its research offerings.

Cancer Gene Therapy Market Market Report: Industry Insights, 2020-2026

This multi-client research study on theCancer Gene Therapy Market marketprovides in-depth research and analysis into Cancer Gene Therapy Market industry trends, market developments and technological insights. The report provides data and analysis of Cancer Gene Therapy Market penetration across application segments across countries and regions. The report presents a strategic analysis of the Cancer Gene Therapy Markety market through key drivers, challenges, opportunities, and growth contributors.

The global Cancer Gene Therapy Market market delivers value to customers through reliable market size for 2020 on the basis of demand and price analysis. The report presents near term and long term forecast of the addressable Cancer Gene Therapy Market market size to 2026.

The report presents an introduction to the Cancer Gene Therapy Market market in 2020, analyzing the COVID-19 impact both quantitatively and qualitatively.

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(The sample of this report is readily available on request).

The FREE PDF Sample Report Includes:

(Note: The sample of this report is updated with COVID-19 impact analysis before delivery)

Some of Following Top Market Players Profile Included in This Report:

2020 Cancer Gene Therapy Market Market: COVID-19 Impact Analysis

The global rise of COVID-19 has many businesses struggling and confused about what steps to take to minimize the economic impact. A simple look at the stock market will tell you that coronavirus has led to a volatile economy, but there are numerous other factors at play.

The long term COVID-19 impact on the business industry is largely still unknown; hence,Facts and Factors market researchanalysts have already covered the effects of COVID-19 on the business industry at a large level, as well as global and regional levels. Through our coverage below, get a better understanding of the business and economic implications coronavirus has on trends like remote working, consumer shopping behavior, global advertising spend, and essential industries like food, medical, travel, and transportation.

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Cancer Gene Therapy Market Market 2020: Research Scope & Coverage

The report coversCancer Gene Therapy Market market sizeand growth, characteristics, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the markets historic and forecast market growth by geography. The market size analysis gives the market size covering both the historic growth of the market, the impact of the COVID 19 virus, and forecasting its recovery. The report also provides a comprehensive analysis of current & future trends and emerging avenues for the growth of this market along with this research also offers an insight into the competitive landscape in terms of new technological developments, untapped segments, and value chain analysis.

The report, Cancer Gene Therapy Market Market, provides up-to-date information about market size, share shifts, and potential risks, as well as in-depth knowledge of products and services, which can help in planning and in executing category management activities. It focuses on the cost-saving aspects of procurement and on providing insights that can lead to the optimization of category spend.

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Competitive Landscape

The market appears to be fragmented and with the presence of several. Thissize of Cancer Gene Therapy Market marketresearch report will help clients identify new growth opportunities and design unique growth strategies by providing a comprehensive analysis of the markets competitive landscape and offering information on the products offered by companies.

Key Takeaways from this Cancer Gene Therapy Market Market Report

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Cancer Gene Therapy Market Size Will Expand Post COVID-19 Covered in Forecast Report 2020-2026 - NJ MMA News

Global and US Gene Therapy for Ovarian Cancer Market to Witness Huge Growth by abc Major Players included in report Takara Bio, VBL Therapeutics,…

Global Coronavirus pandemic has impacted all industries across the globe, Gene Therapy for Ovarian Cancer market being no exception. As Global economy heads towards major recession post 2009 crisis, Cognitive Market Research has published a recent study which meticulously studies impact of this crisis on Global Gene Therapy for Ovarian Cancer market and suggests possible measures to curtail them. This press release is a snapshot of research study and further information can be gathered by accessing complete report. To Contact Research Advisor Mail us @ [emailprotected] or call us on +1-312-376-8303.

The research report on global Gene Therapy for Ovarian Cancer market as well as industry is a detailed study that provides detailed information of major key players, product types & applications/end-users; historical figures, region analysis, market drivers/opportunities & restraints forecast scenarios, strategic planning, and a precise section for the effect of Covid-19 on the market. Our research analysts intensively determine the significant outlook of the global Gene Therapy for Ovarian Cancer market study with regard to primary & secondary data and they have represented it in the form of graphs, pie charts, tables & other pictorial representations for better understanding.

Intravenous, Intratumoral, Intraperitoneal are some of the key types of market. All the type segments have been analyzed based on present and future trends and the market is estimated from 2020 to 2027. Based on the application segment, the global market can be classified into Ovarian Cancer (unspecified), Recurrent Ovarian Epithelial Cancer, Platinum Resistant Ovarian Cancer . The analysis of application segment will help to analyze the demand for market across different end-use industries.

Request Free Sample Copy of Gene Therapy for Ovarian Cancer Market Research [emailprotected] https://cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-ovarian-cancer-market-report#download_report

Amid the COVID-19 pandemic, the industry is witnessing a major change in operations.Some of the key players include Takara Bio, VBL Therapeutics, CELSION, Targovax . key players are changing their recruitment practices to comply with the social distancing norms enforced across several regions to mitigate the risk of infection. Additionally, companies are emphasizing on using advanced recruiting solutions and digital assets to avoid in-person meetings. Advanced technologies and manufacturing process are expected to play a decisive role in influencing the competitiveness of the market players.

Regional Analysis for Gene Therapy for Ovarian Cancer Market:North America (United States, Canada)Europe (Germany, Spain, France, UK, Russia, and Italy)Asia-Pacific (China, Japan, India, Australia, and South Korea)Latin America (Brazil, Mexico, etc.)The Middle East and Africa (GCC and South Africa)

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NOTE: Whole world is experiencing the impact of Covid-19 pandemic due to its increasing spread hence, the report comprises of an up to date scenario of the Gene Therapy for Ovarian Cancer market report. Research analyst team of our company is understanding & reviewing the Covid19 Impact on Market and all the necessary areas of the market that have been altered due to the change caused by Covid19 impact. Get in touch with us for more precise/in-depth information of the Gene Therapy for Ovarian Cancer market.

Any query? Enquire Here For Discount (COVID-19 Impact Analysis Updated Sample): Click Here>Download Sample Report of Gene Therapy for Ovarian Cancer Market Report 2020 (Coronavirus Impact Analysis on Gene Therapy for Ovarian Cancer Market)

At the end of May, many states began lifting lockdown restrictions and reopening in order to revive their economies, despite warnings that it was still too early. As a result, by mid-July, around 33 states were reporting higher rates of new cases compared to the previous week with only three states reporting declining rates. Due to this Covid-19 pandemic, there has been disruptions in the supply chain which have made end-use businesses realize destructive in the manufacturing and business process. During this lockdown period, the plastic packaging helps the products to have longer shelf life as the public would not be able to buy new replacements for the expired products because most of the production units are closed.

About Us:Cognitive Market Research is one of the finest and most efficient Market Research and Consulting firm. The company strives to provide research studies which include syndicate research, customized research, round the clock assistance service, monthly subscription services, and consulting services to our clients. We focus on making sure that based on our reports, our clients are enabled to make most vital business decisions in easiest and yet effective way. Hence, we are committed to delivering them outcomes from market intelligence studies which are based on relevant and fact-based research across the global market.Contact Us: +1-312-376-8303Email: [emailprotected]Web: https://www.cognitivemarketresearch.com/

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Global and US Gene Therapy for Ovarian Cancer Market to Witness Huge Growth by abc Major Players included in report Takara Bio, VBL Therapeutics,...

VBL Presents Human Proof-of-Concept Data That Show the Potential of its Novel anti-MOSPD2 Monoclonal Antibodies for Multiple Sclerosis at the MS…

TEL AVIV, Israel, Sept. 11, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT), today presents for the first time new data demonstrating ex-vivo activity of its proprietary investigational anti-MOSPD2 mAbs in patients with relapsing-remitting and progressive multiple sclerosis (MS). VBL's study is being presented at the MS Virtual 2020, the virtual 8th Joint ACTRIMS-ECTRIMS Meeting.

Data show that VBL's anti-MOSPD2 mAbs significantly inhibited migration of monocytes isolated from all MS patients included in the study (n=33) by up to 97%, regardless of disease severity, gender or active treatment. Notably, the activity was seen not only in the monocytes from relapsing-remitting, but also those from primary progressive and secondary progressive patients with high Expanded Disability Status Scale (EDSS) scores of 5.5-6.5.

We believe our antibodies open up a completely novel mechanism for potential treatment of MS, through blocking the accumulation of monocytes/macrophages in the central nervous system, said Itzhak Mendel, Ph.D., Immunology Director of VBL Therapeutics. This mechanism is differentiated from the existing available treatments, which mostly target T and B cells and therefore, it is not surprising that our antibodies showed activity on top of any patient active therapy. Backed up by strong preclinical results, these first patient-driven proof-of-concept data reinforce the therapeutic potential of our MOSPD2 program.

VBL is currently advancing lead anti-MOSPD2 candidate VB-601 through IND-enabling studies, aiming to start a first-in-human study in 2H 2021.

For a link to VBL's presentation at the MS Virtual 2020 conference, see: LINK

About VBL's VB-600 PlatformVBL is conducting two parallel drug development programs that are exploring the potential of MOSPD2 (motile sperm domain-containing protein 2), a protein that VBL has identified as a key regulator of cell motility, as a therapeutic target for inflammatory diseases and cancer. Our VB-600 platform comprises classical anti-MOSPD2 monoclonal antibodies for inflammatory indications, as well as bi-specific antibody candidates for oncology.

About VBLVascular Biogenics Ltd., operating asVBL Therapeutics, is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class treatments for areas of unmet need in cancer and immune/inflammatory indications. VBL has developed three platform technologies: a gene-therapy based technology for targeting newly formed blood vessels with focus on cancer, an antibody-based technology targeting MOSPD2 for anti-inflammatory and immuno-oncology applications, and the Lecinoxoids, a family of small-molecules for immune-related indications. VBLs lead oncology product candidate, ofranergene obadenovec (VB-111), is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. It is conveniently administered as an IV infusion once every 6-8 weeks. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in a VBL-sponsored all comers Phase 1 trial as well as in three VBL-sponsored tumor-specific Phase 2 studies. Ofranergene obadenovec is currently being studied in a VBL-sponsored Phase 3 potential registration trial for platinum-resistant ovarian cancer.

Forward Looking StatementsThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. These forward-looking statements may include, but are not limited to, statements regarding our programs, including MOSPD2, including their clinical development, therapeutic potential and clinical results. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals, the risk that historical clinical trial results may not be predictive of future trial results, the impact of the COVID-19 pandemic on our business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines and clinical results, that our financial resources do not last for as long as anticipated, and that we may not realize the expected benefits of our intellectual property protection. A further list and description of these risks, uncertainties and other risks can be found in our regulatory filings with theU.S. Securities and Exchange Commission, including in our annual report on Form 20-F for the year endedDecember 31, 2019, and subsequent filings with theSEC. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.VBL Therapeuticsundertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

INVESTOR CONTACT:Michael RiceLifeSci Advisors, LLC(646) 597-6979

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VBL Presents Human Proof-of-Concept Data That Show the Potential of its Novel anti-MOSPD2 Monoclonal Antibodies for Multiple Sclerosis at the MS...

Precision Cancer Therapies Market, Share, Application Analysis, Regional Outlook, Competitive Strategies & Forecast up to 2025 |, Abbott…

LOS ANGELES, United States: QY Research as of late produced a research report titled, Global and United States Precision Cancer Therapies Market Size, Status and Forecast 2020-2026. The research report speak about the potential development openings that exist in the worldwide market. The report is broken down on the basis of research procedures procured from historical and forecast information. The global Precision Cancer Therapies market is relied upon to develop generously and flourish as far as volume and incentive during the gauge time frame. The report will give a knowledge about the development openings and controls that will build the market. Pursuers can increase important perception about the eventual fate of the market.

The global Precision Cancer Therapies market size is projected to reach US$ XX million by 2026, from US$ XX million in 2020, at a CAGR of XX% during 2021-2026.

Key Companies/Manufacturers operating in the global Precision Cancer Therapies market include: , Abbott Laboratories, Bayer HealthCare, GlaxoSmithKline, OncoGenex Pharmaceuticals, Hospira, Boehringer Ingelheim, AstraZeneca, Aveo Pharmaceuticals

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Segmental Analysis

The report incorporates significant sections, for example, type and end user and a variety of segments that decide the prospects of global Precision Cancer Therapies market. Each type provide data with respect to the business esteem during the conjecture time frame. The application area likewise gives information by volume and consumption during the estimate time frame. The comprehension of this segment direct the readers in perceiving the significance of variables that shape the market development.

Global Precision Cancer Therapies Market Segment By Type:

Hormone TherapyImmunotherapiesTargeted TherapyMonoclonal Antibody TherapyGene Therapy Precision Cancer Therapies

Global Precision Cancer Therapies Market Segment By Application:

HospitalsDiagnostic CentersOncology ClinicsResearch Institutes

Competitive Landscape

Competitor analysis is one of the best sections of the report that compares the progress of leading players based on crucial parameters, including market share, new developments, global reach, local competition, price, and production. From the nature of competition to future changes in the vendor landscape, the report provides in-depth analysis of the competition in the global Precision Cancer Therapies market.

Key questions answered in the report:

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TOC

1 Report Overview1.1 Study Scope1.2 Market Analysis by Type1.2.1 Global Precision Cancer Therapies Market Size Growth Rate by Type: 2020 VS 20261.2.2 Hormone Therapy1.2.3 Immunotherapies1.2.4 Targeted Therapy1.2.5 Monoclonal Antibody Therapy1.2.6 Gene Therapy1.3 Market by Application1.3.1 Global Precision Cancer Therapies Market Share by Application: 2020 VS 20261.3.2 Hospitals1.3.3 Diagnostic Centers1.3.4 Oncology Clinics1.3.5 Research Institutes1.4 Study Objectives1.5 Years Considered 2 Global Growth Trends2.1 Global Precision Cancer Therapies Market Perspective (2015-2026)2.2 Global Precision Cancer Therapies Growth Trends by Regions2.2.1 Precision Cancer Therapies Market Size by Regions: 2015 VS 2020 VS 20262.2.2 Precision Cancer Therapies Historic Market Share by Regions (2015-2020)2.2.3 Precision Cancer Therapies Forecasted Market Size by Regions (2021-2026)2.3 Industry Trends and Growth Strategy2.3.1 Market Trends2.3.2 Market Drivers2.3.3 Market Challenges2.3.4 Market Restraints 3 Competition Landscape by Key Players3.1 Global Top Precision Cancer Therapies Players by Market Size3.1.1 Global Top Precision Cancer Therapies Players by Revenue (2015-2020)3.1.2 Global Precision Cancer Therapies Revenue Market Share by Players (2015-2020)3.2 Global Precision Cancer Therapies Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.3 Players Covered: Ranking by Precision Cancer Therapies Revenue3.4 Global Precision Cancer Therapies Market Concentration Ratio3.4.1 Global Precision Cancer Therapies Market Concentration Ratio (CR5 and HHI)3.4.2 Global Top 10 and Top 5 Companies by Precision Cancer Therapies Revenue in 20193.5 Key Players Precision Cancer Therapies Area Served3.6 Key Players Precision Cancer Therapies Product Solution and Service3.7 Date of Enter into Precision Cancer Therapies Market3.8 Mergers & Acquisitions, Expansion Plans 4 Precision Cancer Therapies Breakdown Data by Type (2015-2026)4.1 Global Precision Cancer Therapies Historic Market Size by Type (2015-2020)4.2 Global Precision Cancer Therapies Forecasted Market Size by Type (2021-2026) 5 Precision Cancer Therapies Breakdown Data by Application (2015-2026)5.1 Global Precision Cancer Therapies Historic Market Size by Application (2015-2020)5.2 Global Precision Cancer Therapies Forecasted Market Size by Application (2021-2026) 6 North America6.1 North America Precision Cancer Therapies Market Size (2015-2026)6.2 North America Precision Cancer Therapies Market Size by Type (2015-2020)6.3 North America Precision Cancer Therapies Market Size by Application (2015-2020)6.4 North America Precision Cancer Therapies Market Size by Country (2015-2020)6.4.1 United States6.4.2 Canada 7 Europe7.1 Europe Precision Cancer Therapies Market Size (2015-2026)7.2 Europe Precision Cancer Therapies Market Size by Type (2015-2020)7.3 Europe Precision Cancer Therapies Market Size by Application (2015-2020)7.4 Europe Precision Cancer Therapies Market Size by Country (2015-2020)7.4.1 Germany7.4.2 France7.4.3 U.K.7.4.4 Italy7.4.5 Russia7.4.6 Nordic7.4.7 Rest of Europe 8 China8.1 China Precision Cancer Therapies Market Size (2015-2026)8.2 China Precision Cancer Therapies Market Size by Type (2015-2020)8.3 China Precision Cancer Therapies Market Size by Application (2015-2020)8.4 China Precision Cancer Therapies Market Size by Region (2015-2020)8.4.1 China8.4.2 Japan8.4.3 South Korea8.4.4 Southeast Asia8.4.5 India8.4.6 Australia8.4.7 Rest of Asia-Pacific 9 Japan9.1 Japan Precision Cancer Therapies Market Size (2015-2026)9.2 Japan Precision Cancer Therapies Market Size by Type (2015-2020)9.3 Japan Precision Cancer Therapies Market Size by Application (2015-2020)9.4 Japan Precision Cancer Therapies Market Size by Country (2015-2020)9.4.1 Mexico9.4.2 Brazil 10 Southeast Asia10.1 Southeast Asia Precision Cancer Therapies Market Size (2015-2026)10.2 Southeast Asia Precision Cancer Therapies Market Size by Type (2015-2020)10.3 Southeast Asia Precision Cancer Therapies Market Size by Application (2015-2020)10.4 Southeast Asia Precision Cancer Therapies Market Size by Country (2015-2020)10.4.1 Turkey10.4.2 Saudi Arabia10.4.3 UAE10.4.4 Rest of Middle East & Africa 11 Key Players Profiles11.1 Abbott Laboratories11.1.1 Abbott Laboratories Company Details11.1.2 Abbott Laboratories Business Overview11.1.3 Abbott Laboratories Precision Cancer Therapies Introduction11.1.4 Abbott Laboratories Revenue in Precision Cancer Therapies Business (2015-2020))11.1.5 Abbott Laboratories Recent Development11.2 Bayer HealthCare11.2.1 Bayer HealthCare Company Details11.2.2 Bayer HealthCare Business Overview11.2.3 Bayer HealthCare Precision Cancer Therapies Introduction11.2.4 Bayer HealthCare Revenue in Precision Cancer Therapies Business (2015-2020)11.2.5 Bayer HealthCare Recent Development11.3 GlaxoSmithKline11.3.1 GlaxoSmithKline Company Details11.3.2 GlaxoSmithKline Business Overview11.3.3 GlaxoSmithKline Precision Cancer Therapies Introduction11.3.4 GlaxoSmithKline Revenue in Precision Cancer Therapies Business (2015-2020)11.3.5 GlaxoSmithKline Recent Development11.4 OncoGenex Pharmaceuticals11.4.1 OncoGenex Pharmaceuticals Company Details11.4.2 OncoGenex Pharmaceuticals Business Overview11.4.3 OncoGenex Pharmaceuticals Precision Cancer Therapies Introduction11.4.4 OncoGenex Pharmaceuticals Revenue in Precision Cancer Therapies Business (2015-2020)11.4.5 OncoGenex Pharmaceuticals Recent Development11.5 Hospira11.5.1 Hospira Company Details11.5.2 Hospira Business Overview11.5.3 Hospira Precision Cancer Therapies Introduction11.5.4 Hospira Revenue in Precision Cancer Therapies Business (2015-2020)11.5.5 Hospira Recent Development11.6 Boehringer Ingelheim11.6.1 Boehringer Ingelheim Company Details11.6.2 Boehringer Ingelheim Business Overview11.6.3 Boehringer Ingelheim Precision Cancer Therapies Introduction11.6.4 Boehringer Ingelheim Revenue in Precision Cancer Therapies Business (2015-2020)11.6.5 Boehringer Ingelheim Recent Development11.7 AstraZeneca11.7.1 AstraZeneca Company Details11.7.2 AstraZeneca Business Overview11.7.3 AstraZeneca Precision Cancer Therapies Introduction11.7.4 AstraZeneca Revenue in Precision Cancer Therapies Business (2015-2020)11.7.5 AstraZeneca Recent Development11.8 Aveo Pharmaceuticals11.8.1 Aveo Pharmaceuticals Company Details11.8.2 Aveo Pharmaceuticals Business Overview11.8.3 Aveo Pharmaceuticals Precision Cancer Therapies Introduction11.8.4 Aveo Pharmaceuticals Revenue in Precision Cancer Therapies Business (2015-2020)11.8.5 Aveo Pharmaceuticals Recent Development 12 Analysts Viewpoints/Conclusions 13 Appendix13.1 Research Methodology13.1.1 Methodology/Research Approach13.1.2 Data Source13.2 Disclaimer13.3 Author Details

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News briefing: Exelixis rolls the dice with 2 ADC deals tied to $35M cash upfronts; A rare antibiotic win for prominently-backed Spero – Endpoints…

Catalents Redwood Bioscience subsidiary is taking the lead role in developing ADCs for Exelixis $EXEL. In one of 2 deals announced this morning, Exelixis is turning to Catalent to do the discovery work on the ADCs, which will employ their SMARTag site-specific bioconjugation platform technology using antibodies out of the biotechs pipeline.

Catalent gets $10 million upfront to trigger the deal, with Exelixis holding worldwide development rights on anything it chooses.

In a separate ADC development deal, Exelixis is turning to NBE-Therapeutics for another pact that could steer more product candidates its way. In this deal Exelixis is paying $25 million upfront to get a 2-year alliance underway.

Exelixis is pursuing both internal drug discovery and external business development approaches to build a pipeline with the potential to make a difference for patients with cancer, saidPeter Lamb, the CSO at Exelixis. John Carroll

Back in the days when investors thought there was still good business to be made in antibiotics, Spero raised a fair bit of capital: a $30 million Series A and B led by Atlas Ventures, a $51.7 million Series C led by GV, and a $77 million IPO. In 2018, they received an up to $54 million contract with BARDA too.

The market for antibiotics has since soured, but that cash has allowed Spero to get through Phase III with its lead drug. And on Tuesday they announced positive results, showing in a 1,372-person study that their oral antibiotic tebipenem was non-inferior to the approved IV antibiotic ertapenem in treating patients with complicated urinary tract infections and acute pyelonephritis.

The drug, said trial investigator Keith Kaye, will give cUTI patients a new oral option after evolving microbiotic resistance had left them with only the IV drug.

Due to the increasing prevalence of antibiotic-resistant bacteria, many patients with cUTI now receive intravenous antibiotics as their only available treatment option, Kaye, who is also director of research in the division of infectious diseases at the University of Michigan Medical School, said in a statement. The much-anticipated data from this head-to-head comparison against an IV standard-of-care carbapenem antibiotic suggest that in many instances oral, outpatient treatment of these complicated bacterial infections is a viable option.

Spero CEO Ankit Mahadevia said it would be the first oral cUTI drug approved in 26 years. The company said it plans to start a rolling NDA and complete it by the second quarter of 2021.

An approval would be a major boon to any biotech, but when it comes to antibiotics, regulatory success doesnt necessarily translate into commercial success. Melinta and Achaoegen are well proof of that. Jason Mast

A UK-based biotech is buying outright a software developer in Brooklyn, NY, in order to bring artificial intelligence to its diagnostics tools.

APIS Assay Technologies announced the acquisition of Beogenomics on Friday, hoping to use data-mining processes to identify biomarker targets in oncology, as well as inflammatory, autoimmune and infectious diseases. The technology from Beogenomics, which has been developing both on-prem and secure cloud-based data analysis solutions, will help support the launch of a new proprietary service line. APIS mainly works in R&D and diagnostics, developing new tests for the prediction, prevention, and diagnosis of disease from discovery to regulatory approval.

The companys business model focuses on three aspects: biomarker diagnostics development, molecular diagnostic contract development, and applied bioinformatics. Max Gelman

Intravacc, a Dutch vaccines company, landed an up to $9.4 million contract from NIAID to develop a vaccine for enterovirus D68, a respiratory virus that can cause paralysis and has become increasingly common in America, Europe and Asia over the last few years.

The small biotech, which also has programs for RSV, gonorrhea and of course Covid-19, will develop an inactivated virus vaccine in Vero cells. The contract is for early product selection through Phase I. Jason Mast

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News briefing: Exelixis rolls the dice with 2 ADC deals tied to $35M cash upfronts; A rare antibiotic win for prominently-backed Spero - Endpoints...

COVID-19 Impact on Cell and Gene Therapy Market Research, Size, Growth And Trends 2020 to 2025: Keyplayer-Amgen Inc., bluebird bio, Inc. – Scientect

Chicago, United States:The global Cell and Gene Therapy Market report offers a complete research study that includes accurate estimations of market growth rate and size for the forecast period 2020-2025. It offers a broad analysis of market competition, regional expansion, and market segmentation by type, application, and geography supported by exact market figures. The all-inclusive market research report also offers Porters Five Forces Analysis and profiles some of the leading players of the global Cell and Gene Therapy Market. It sheds light on changing market dynamics and discusses about different growth drivers, market challenges and restraints, and trends and opportunities in detail. Interested parties are provided with market recommendations and business advice to ensure success in the global Cell and Gene Therapy Market.

Top Key players cited in the report:Amgen Inc., bluebird bio, Inc., Dendreon Pharmaceuticals LLC., Fibrocell Science, Inc., Human Stem Cells Institute, Kite Pharma, Inc., Kolon TissueGene, Inc., Novartis AG, Orchard Therapeutics plc., Organogenesis Holdings Inc., Pfizer, Inc., RENOVA THERAPEUTICS, .

Get Free PDF Sample Copy of this Report to understand the structure of the complete report: (Including Full TOC, List of Tables & Figures, Chart): https://www.reporthive.com/request_sample/2369809

The final report will add the analysis of the Impact of Covid-19 in this report Cell and Gene Therapy Market

Cell and Gene Therapy Marketreports offers important insights which help the industry experts, product managers, CEOs, and business executives to draft their policies on various parameters including expansion, acquisition, and new product launch as well as analyzing and understanding the market trends.

Each segment of the global Cell and Gene Therapy market is extensively evaluated in the research study. The segmental analysis offered in the report pinpoints key opportunities available in the global Cell and Gene Therapy market through leading segments. The regional study of the global Cell and Gene Therapy market included in the report helps readers to gain a sound understanding of the development of different geographical markets in recent years and also going forth. We have provided a detailed study on the critical dynamics of the global Cell and Gene Therapy market, which include the market influence and market effect factors, drivers, challenges, restraints, trends, and prospects. The research study also includes other types of analysis such as qualitative and quantitative.

Global Cell and Gene Therapy Market: Competitive Rivalry

The chapter on company profiles studies the various companies operating in the global Cell and Gene Therapy market. It evaluates the financial outlooks of these companies, their research and development statuses, and their expansion strategies for the coming years. Analysts have also provided a detailed list of the strategic initiatives taken by the Cell and Gene Therapy market participants in the past few years to remain ahead of the competition.

Global Cell and Gene Therapy Market: Regional Segments

The chapter on regional segmentation details the regional aspects of the global Cell and Gene Therapy market. This chapter explains the regulatory framework that is likely to impact the overall market. It highlights the political scenario in the market and the anticipates its influence on the global Cell and Gene Therapy market.

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Report Highlights

Table of Contents

Report Overview:It includes six chapters, viz. research scope, major manufacturers covered, market segments by type, Cell and Gene Therapy market segments by application, study objectives, and years considered.

Global Growth Trends:There are three chapters included in this section, i.e. industry trends, the growth rate of key producers, and production analysis.

Cell and Gene Therapy Market Share by Manufacturer:Here, production, revenue, and price analysis by the manufacturer are included along with other chapters such as expansion plans and merger and acquisition, products offered by key manufacturers, and areas served and headquarters distribution.

Market Size by Type:It includes analysis of price, production value market share, and production market share by type.

Market Size by Application:This section includes Cell and Gene Therapy market consumption analysis by application.

Profiles of Manufacturers:Here, leading players of the global Cell and Gene Therapy market are studied based on sales area, key products, gross margin, revenue, price, and production.

Cell and Gene Therapy Market Value Chain and Sales Channel Analysis:It includes customer, distributor, Cell and Gene Therapy market value chain, and sales channel analysis.

Market Forecast Production Side: In this part of the report, the authors have focused on production and production value forecast, key producers forecast, and production and production value forecast by type.

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COVID-19 Impact on Cell and Gene Therapy Market Research, Size, Growth And Trends 2020 to 2025: Keyplayer-Amgen Inc., bluebird bio, Inc. - Scientect

Grifols makes a $146M bet on a Stanford play in a controversial anti-aging field – Endpoints News

For the last couple of years, when it came to the vaguely vampiric field of young blood plasma transfusions, there was Alkahest and there was everyone else.

Since the field was briefly mocked onSilicon Valley in 2017, one startup charged $8,000 to $12,000 to pump the elderly with the plasma of young donors and one doctor pitched his clinical trial in a glitzy West Palm Beach gala where he reminded retirees they were likely to die soon, prompting FDAs chief Scott Gottlieb to warn such treatments were unproven and that some patients are being preyed upon by unscrupulous actors. But Alkahest, founded by a Genentech alumn and a Stanford neuroscientist, has promised to take a more measured, scientific approach to the still-fringe science, running phased clinical trials for their plasma-derived products and couching their press releases accordingly.

Now, the plasma giant Grifols is going all-in on their work. On Labor Day, the Spanish company announced they would buy out the remaining shares of the startup they didnt already own, spending $146 million for just over 50% of Alkahests stock.

Grifols is a roughly $15 billion company, so the buyout is not exactly a huge outlay for them. Nor is the nearly $300 million market value this deal places on Alkahest nearly enough to rank them among the industrys most valuable players. Still, the acquisition represents a major validation for a company and a young-blood field that has grown up largely on the fringes of the biomedical mainstream. And it amounts to a step forward for the anti-aging field more broadly, which has struggled to make gains despite significant big-name investment over the last few years.

The new deal is about four times what Grifols paid for the first 45% of Alkahests stock in 2015. The startup has relied on Grifols to collect plasma for its products.

We saw the promise of Alkahests understanding of aging when we made our first investment and entered into a collaboration agreement with them five years ago, Grifols CEO Vctor Grfols said in a statement. Now we see a wealth of plasma-derived and non-plasma therapeutic candidates identified by Alkahest that can significantly support the unmet needs of many diseases associated with aging.

Unlike, say, Ambrosia (the aforementioned $8,000 infusion company), Alkahest doesnt give plasma directly from young donors to the elderly. Instead, it has worked since 2014 on mapping the proteins in plasma and distilling a cocktail of roughly 400 different types of proteins it believes can make a difference in treating Alzheimers and other CNS disorders. Thats a ton of proteins compared to most biologics, which are made of 1, but it amounts to just 3% of whats in plasma. The Long Island Ice Tea of a lead drug is known as GRF-6019. The science is based on work from Stanford neuroscientist Tony Wyss-Coray, which was spun out by former Genentech scientist Karoly Nikolich.

So far, evidence for effectiveness remains scant. Last year, the company said that, in a 47-person Phase II study, patients with mild to moderate Alzheimers maintained their level of cognition for 6 months. But they still have yet to release data from that study and while this is certainly a new approach to the evasive disease, many other therapies have failed after showing promise in early trials. Their Phase I study, published inJAMA, showed little change in cognitive performance tests among 18 Alzheimers patients, although it was only powered for safety.

Alkahest is also testing GRF-6019 in severe Alzheimers patients, an oft-overlooked population, and said recently that it proved safe in a 26-person pilot study. They have studies ongoing in Parkinsons, age-related macular degeneration, and patients with end-stage renal disease and cognitive impairment.

Outside of plasma, the company is also developing an oral drug aimed at another anti-aging target: Eotaxin. Its an immunomodulatory protein, they say, that increases with age. Alkahest is in Phase II studies to see if blocking it can curb Parkinsons and AMD.

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Grifols makes a $146M bet on a Stanford play in a controversial anti-aging field - Endpoints News

New California law gives consumers more agency over data they share with genetic testing firms – Business Insider

California lawmakers passed a law that allows consumers to revoke consent for genetic testing companies like 23andMe and Ancestry to use their data, mandating the companies to destroy the DNA samples within 30 days, STAT reported in its weeklynewsletter.

California law boosts genetic data privacy. Business Insider Intelligence

For context, direct-to-consumer (D2C) DNA testing firms often give customers the opportunity to opt into research by consenting to pass along their samples: For instance, 8 million of 23andMe's network of 10 million users haveopted in to participate in research. States are taking the helm at passing D2C genetic testing regulation, while federal lawmakers remain mumcreating a patchworked legal landscape for genetic testing companies to operate in.

Privacy laws have yet to be enacted on a federal level, so state lawmakers are stepping in: Florida recently passedlegislation prohibiting insurance companies from accessing members' genetic insights, which could impact the type and cost of coverage. But as states take charge putting forth their own laws, genetic testing companies will be faced with new obstacles, and it's unclear how they'll navigate adhering to the changing legal ecosystem.

In reference to the new law passed in California, Justin Yedor, a Los Angeles-based data privacy attorney, wascitedin Bloomberg asking, "are [D2C companies] going to provide these rights strictly for Californians or are they going to extend them to all consumers regardless of jurisdiction?" Contending with new rules passed on a state-by-state basis could cause hangups in operations, exacerbating the alreadysofteningD2C genetic testing market.

While a hodgepodge of legislation across the US will be a hurdle, increased privacy laws could assuage consumers' fears and translate into more sales.In a recent YourDNA survey,40%of consumers who had never taken a DNA test cited privacy concerns as the driving reason for why they've shied away. But if companies are transparent about granting consumers more autonomy over their data and how it's handled, they may be more likely to take the plunge.

Still, we think high-flying genetic testing firms will lean more heavily on their healthcare-focused initiatives as they navigate the shifting D2C realm: Some DNA testing firms likeColorandYouScriptthe latter of which is now owned by Invitaeare powering hospitals' precision medicine initiatives, for example.

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New California law gives consumers more agency over data they share with genetic testing firms - Business Insider

Sarepta Therapeutics Provides Program Update for SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy -…

CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C written response only meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA), to obtain OTATs concurrence on the commencement of its next clinical trial for SRP-9001 using commercial process material. SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) is Sareptas investigational gene transfer therapy for the treatment of Duchenne muscular dystrophy.

Among other items, OTAT has requested that Sarepta utilize an additional potency assay for release of SRP-9001 commercial process material prior to dosing in a clinical study. Sarepta has several existing assays and data that it believes could be employed in response to OTATs request. However, additional dialogue with the Agency is required to determine the acceptability of the potency assay approach.

We look forward to working with OTAT to potentially satisfy their requests and to obtain clarity on the timing of the commencement of our commercial supply study. We will provide further updates as we are able, said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. Every day, thousands of children degenerate from the irreversible damage caused by Duchenne muscular dystrophy. It is for that reason that we will work relentlessly with the Division to satisfy any requests of OTAT and continue the advancement of a potentially transformative therapy for these patients.

About SRP-9001 (AAVrh74.MHCK7.micro-dystrophin)SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States. In December 2019, the Company announced a licensing agreement granting Roche the exclusive right to launch and commercialize SRP-9001 outside the United States. Sarepta has exclusive rights to the micro-dystrophin gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

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.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Sarepta Forward-Looking Statements

This 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 Sareptas belief that its existing assays and data could be employed in response to OTATs request; the acceptability of Sareptas potency assay approach by the FDA; our plan to work with OTAT to potentially satisfy their requests and to obtain clarity on the timing of the commencement of our commercial supply study; and the potential of SRP-9001 to be a transformative therapy for DMD patients.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: delays in the commencement of Sareptas next clinical study for SRP-9001 could delay, prevent or limit our ability to gain regulatory approval for SRP-9001; any inability to complete successfully clinical development could result in additional costs to Sarepta or impair Sareptas ability to generate revenues from product sales, regulatory and commercialization milestones and royalties; SRP-9001 may not result in a viable treatment suitable for commercialization due to a variety of reasons, including the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas 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 Sareptas product candidates and the COVID-19 pandemic; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas 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 the SEC filings 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.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to 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 Provides Program Update for SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy -...

$14.6M Grant to Explore a Therapy to Control HIV Without Meds – Cancer Health Treatment News

In nearly 40 years of the HIV epidemic, only two people have likely been cured of the virus. Both scenarios resulted from stem cell transplants needed to fight blood cancers such as leukemia. Inspired by these two cases, a team of scientists is studying a multipronged way to potentially control HIV without medication. It involves two different genetic alterations of immune cells and with a safer method of stem cell transplants, also referred to as bone marrow transplants, a procedure that is generally toxic and dangerous.

The research is being funded by a five-year $14.6 million grant from the National Institutes of Health. The scientists coleading the preclinical studies are Paula Cannon, PhD, a distinguished professor of molecular microbiology and immunology at the Keck School of Medicine of the University of Southern California, and Hans-Peter Kiem, MD, PhD, who directs the stem cell and gene therapy program at the Fred Hutchinson Cancer Research Center, also known as Fred Hutch. According to a Keck School of Medicine press release, the two other main partners are David Scadden, MD, a bone marrow transplant specialist and professor at Harvard University and the Harvard Stem Cell Institute, and the biotechnology company Magenta Therapeutics.

In the HIV cure scenariosinvolving the so-called Berlin and London patientsboth men received stem cell transplants from donors with a natural genetic mutation that made them resistant to HIV. Specifically, their genes resulted in immune cells that lack CCR5 receptors on their surface (HIV latches onto these receptors to infect cells). Unfortunately, this method isnt viable for the nearly 38 million people worldwide living with HIV. Not only is it expensive, toxic and riskyit involves wiping out the patients immune system and replacing it with the new immune cellsbut it also requires matched donors who are CCR5 negative. According to the press release, about 1% of the population have this mutation.

With funding from this new grant, researchers hope to overcome these challenges in several ways. First, Cannon has already developed a gene-editing method to remove the CCR5 receptors from a patients own stem cells. She now hopes to further genetically engineer stem cells so they release antibodies that block HIV.

Our engineered cells will be good neighbors, Cannon said in the press release. They secrete these protective molecules so that other cells, even if they arent engineered to be CCR5 negative, have some chance of being protected.

Fred Hutchs Kiem will use CAR-T therapya new method of genetically modifying immune cells that is emerging out of cancer researchwith the goal of creating T cells that attack HIV-infected cells.

In addition, other scientists involved in the federal grant aim to develop less toxic methods of bone marrow transplantationfor example, by reducing the amount of chemotherapy required and speeding up the process of creating the new immune system.

The research finding could translate to other illnesses, such as cancer, sickle cell anemia and autoimmune disorders.

A home run would be that we completely cure people of HIV, Cannon said. What Id be fine with is the idea that somebody no longer needs to take anti-HIV drugs every day because their immune system is keeping the virus under control so that it no longer causes health problems and, importantly, they cant transmit it to anybody else.

For the latest on the cure cases, see Famed London Man Probably Cured of HIV from earlier this year. And in related news, see $14M Federal Grant to Research CAR-T Gene Therapy to Cure HIV.

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$14.6M Grant to Explore a Therapy to Control HIV Without Meds - Cancer Health Treatment News

Gyroscope Therapeutics Announces Appointment of Sean Bohen to the Board of Directors – Business Wire

LONDON--(BUSINESS WIRE)--Gyroscope Therapeutics Limited, a clinical-stage retinal gene therapy company, today announced that biotech industry veteran Sean Bohen, M.D., Ph.D., has been appointed to the Gyroscope Board of Directors, effective immediately. Dr. Bohen will also serve as Chair of the Boards Research and Development Committee. Dr. Bohen brings more than 30 years of experience in the discovery and development of new medicines. Over the course of his career, he has made significant contributions to the early- and late-stage development of numerous FDA-approved drugs.

Sean brings a wealth of experience to our board in researching potential new medicines and successfully bringing them to patients in need. He also brings expertise and knowledge in helping companies build impressive R&D and commercial units side by side. We are excited to work with him as we advance Gyroscopes programmes, said Khurem Farooq, Chief Executive Officer.

Dr. Bohen currently serves on the board of directors of Altrubio, Inc. Previously, Dr. Bohen was the Chief Medical Officer and Executive Vice President, Global Medicines Development, AstraZeneca LP, where he was responsible for AstraZenecas worldwide product development and clinical programmes. Prior to joining AstraZeneca, he was Senior Vice President of Genentech Early Development (gRED), where he led preclinical and clinical development programmes to deliver pivotal trial-ready drug candidates to Genentechs late-stage development pipeline. During this time, he also served as a clinical instructor at the Stanford University School of Medicine. He received both his Ph.D. in Biochemistry and M.D. from the University of California, San Francisco, and has been board certified in Internal Medicine and Medical Oncology.

About Gyroscope: Vision for Life

Gyroscope Therapeutics is a clinical-stage retinal gene therapy company developing and delivering gene therapy beyond rare disease to treat a leading cause of blindness, dry age-related macular degeneration. Our lead investigational gene therapy, GT005, is a one-time therapy delivered under the retina. GT005 is designed to restore balance to an overactive complement system by increasing production of the Complement Factor I protein. GT005 is currently being evaluated in a Phase I/II clinical trial called FOCUS and a Phase II clinical trial called EXPLORE.

Syncona Ltd, our lead investor, helped us create the only retinal gene therapy company to combine discovery, research, drug development, a manufacturing platform and surgical delivery capabilities. Headquartered in London with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness. For more information visit: http://www.gyroscopetx.com and follow us on Twitter (@GyroscopeTx) and on LinkedIn.

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Gyroscope Therapeutics Announces Appointment of Sean Bohen to the Board of Directors - Business Wire

Gene Therapy Market is Thriving Worldwide 2020 | Trends, Growth and Profit Analysis, Forecast by 2027 – The Daily Chronicle

New Jersey, United States, The Gene Therapy Market report 2020 provides a detailed impression, describe the product industry scope and the market expanded insights and forecasts up to 2027. It shows market data according to industry drivers, restraints and opportunities, analyzes the market status, the industry share, size, future Trends and growth rate of the market. The Gene Therapy Market report is categorized by application, end user, technology, product / service types, and other, as well as by region. In addition, the report includes the calculated expected CAGR of chitosan acetate-market derivative from the earlier records of the Gene Therapy Market, and current market trends, which are organized with future developments.

Gene Therapy Market was valued at USD 3.69 Billion in 2019 and is projected to reach USD 24.78 Billion by 2027, growing at a CAGR of 26.9% from 2020 to 2027.

Gene Therapy Market, By Product

Viral Vectorso Adeno-associated virus vectorso Retroviral vectors Gammaretroviral vectors Lentiviral vectorso Other viral vectors (herpes simplex and adenoviral vectors) Non-viral Vectorso Oligonucleotideso Other non-viral vectors (plasmids and RNAi)

Gene Therapy Market, By Indication

Neurological Diseases Cancer Hepatological Diseases Duchenne Muscular Dystrophy Other Indications

Gene Therapy Market, By Delivery Method

Ex vivo In vivo

The report provides detailed coverage of the Gene Therapy Market, including structure, definitions, applications, and Industry Chain classifications. The Gene Therapy Market analysis is provided for the international markets including development trends, competitive landscape analysis, investment plan, business strategy, opportunities and development status of key regions. Development policies and plans are discussed and manufacturing processes and cost structures analyzed. This report also includes information on import / export consumption, supply and demand, costs, industry share, policy, Price, Sales and gross margins.

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Gene Therapy Market forecast up to 2027, with information such as company profiles, product picture and specification, capacity production, price, cost, revenue, and contact information. Upstream raw materials and equipment as well as downstream demand analyses are also carried out. The Gene Therapy Market size, development trends and marketing channels are analyzed. Finally, the feasibility of new investment projects is assessed and general research results are offered.

The Gene Therapy Market was created on the basis of an in-depth market analysis with contributions from industry experts. The report covers the growth prospects in the coming years and the discussion of the main providers.

To understand how the effects of COVID-19 are addressed in this report. A sample copy of the report is available at https://www.verifiedmarketresearch.com/product/gene-therapy-market/?utm_source=TDC&utm_medium=001

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Gene Therapy Market is Thriving Worldwide 2020 | Trends, Growth and Profit Analysis, Forecast by 2027 - The Daily Chronicle

Prevail Therapeutics Announces Appointment of William H. Carson, MD, to Board of Directors – BioSpace

NEW YORK, Sept. 10, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc.(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced the appointment ofWilliam H. Carson, M.D., to its Board of Directors.

Bill knows how to drive the development of innovative treatments including drugs for a wide range of diseases that affect the brain from concept to commercialization, saidFrancois Nader, M.D., Non-Executive Chairman of Prevails Board of Directors. We are thrilled that he will bring that expertise to bear at Prevail as we continue to develop gene therapies for patients suffering from devastating neurodegenerative diseases, including Parkinsons disease, frontotemporal dementia and Gaucher disease.

Dr. Carson was most recently the President and CEO of Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC), leading the development and regulatory approvals of Otsukas global compounds including Abilify Maintena, Rexulti, Samsca and Jynarque. He also oversaw the approval of Abilify MyCite, the first FDA-approved digital medicine. Dr. Carson joined Otsuka as Vice President of the Princeton Aripiprazole Unit in 2002, establishing the companys presence there. Before joining Otsuka, he rose through the ranks in the CNS Research and Development department at Bristol-Myers Squibb. Prior to joining the pharmaceutical industry, Dr. Carson, a board-certified psychiatrist, was an Associate Professor in the Department of Psychiatry and Behavioral Sciences at the Medical University of South Carolina.

Dr. Carson currently serves as Chairman of the Board of Directors of OPDC, and is also the Chairman of the Board of the Sozosei Foundation, a newly established Otsuka charitable organization with a main focus on decriminalization of mental illness. He is a Board Member of Excision Biotherapeutics and Trustee of the non-profit Internet2. He is Board Chair Emeritus of the Sphinx Organization, which advocates for parity and inclusion in the arts. Dr. Carson holds an A.B. Degree from Harvard College and a M.D. degree from Case Western Reserve University. He is a Distinguished Fellow of the American Psychiatric Association, the National Medical Association and the Executive Leadership Council. Dr. Carson was named to Savoy Magazines 2018 list of the Top 100 Most Influential Blacks in Corporate America.

Dr. Carson is an outstanding and important addition to our Board of Directors, said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. I know that he will bring both deep medical expertise and compassion for patients to the role, as well as his stellar track record of advancing therapies through clinical development, FDA approval and commercialization.

I am honored to join Prevail at this exciting time for the company, said Dr. Carson. Prevails programs are moving forward quickly, and promising data are beginning to emerge. I am looking forward to helping the company continue to advance its mission of developing potentially disease-modifying treatments for patients with neurodegenerative disorders.

AboutPrevail Therapeutics

Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinsons disease withGBA1mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia withGRNmutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr.Asa Abeliovichin 2017, through a collaborative effort withThe Silverstein Foundationfor Parkinsons with GBA and OrbiMed, and is headquartered inNewYork, NY.

Forward-Looking Statements Related to Prevail

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, as amended. Examples of these forward-looking statements include statements concerning Prevails ability to develop potentially disease-modifying treatments for patients with neurodegenerative disorders. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevails novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; initial data or other preliminary analyses or results of early clinical trials may not be predictive of final trial results or of the results of later clinical trials; Prevails gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on Prevails business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevails filings with theSecurities and Exchange Commission(SEC), including the Risk Factors section of the Companys Quarterly Report on Form 10-Q for the period endedJune 30, 2020, filed with theSEConAugust 11, 2020, and its other documents subsequently filed with or furnished to theSEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Media Contact:Mary CarmichaelTenBridge Communicationsmary@tenbridgecommunications.com617-413-3543

Investor Contact:investors@prevailtherapeutics.com

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Prevail Therapeutics Announces Appointment of William H. Carson, MD, to Board of Directors - BioSpace

Reasons Industries to Thrive Post-Pandemic! Gene Therapy for Age related Macular Degeneration Market Report xyz Answers it Analysis by Key Companies…

Global Coronavirus pandemic has impacted all industries across the globe, Gene Therapy for Age related Macular Degeneration market being no exception. As Global economy heads towards major recession post 2009 crisis, Cognitive Market Research has published a recent study which meticulously studies impact of this crisis on Global Gene Therapy for Age related Macular Degeneration market and suggests possible measures to curtail them. This press release is a snapshot of research study and further information can be gathered by accessing complete report. To Contact Research Advisor Mail us @ [emailprotected] or call us on +1-312-376-8303.

The research report on global Gene Therapy for Age related Macular Degeneration market as well as industry is a detailed study that provides detailed information of major key players, product types & applications/end-users; historical figures, region analysis, market drivers/opportunities & restraints forecast scenarios, strategic planning, and a precise section for the effect of Covid-19 on the market. Our research analysts intensively determine the significant outlook of the global Gene Therapy for Age related Macular Degeneration market study with regard to primary & secondary data and they have represented it in the form of graphs, pie charts, tables & other pictorial representations for better understanding.

Subretinal, Intravitreal, Unspecified are some of the key types of market. All the type segments have been analyzed based on present and future trends and the market is estimated from 2020 to 2027. Based on the application segment, the global market can be classified into Monotherapy, Combination Therapy . The analysis of application segment will help to analyze the demand for market across different end-use industries.

Request Free Sample Copy of Gene Therapy for Age related Macular Degeneration Market Research [emailprotected] https://cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-age-related-macular-degeneration-market-report#download_report

Amid the COVID-19 pandemic, the industry is witnessing a major change in operations.Some of the key players include RetroSense Therapeutics, REGENXBIO, AGTC . key players are changing their recruitment practices to comply with the social distancing norms enforced across several regions to mitigate the risk of infection. Additionally, companies are emphasizing on using advanced recruiting solutions and digital assets to avoid in-person meetings. Advanced technologies and manufacturing process are expected to play a decisive role in influencing the competitiveness of the market players.

Regional Analysis for Gene Therapy for Age related Macular Degeneration Market:North America (United States, Canada)Europe (Germany, Spain, France, UK, Russia, and Italy)Asia-Pacific (China, Japan, India, Australia, and South Korea)Latin America (Brazil, Mexico, etc.)The Middle East and Africa (GCC and South Africa)

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NOTE: Whole world is experiencing the impact of Covid-19 pandemic due to its increasing spread hence, the report comprises of an up to date scenario of the Gene Therapy for Age related Macular Degeneration market report. Research analyst team of our company is understanding & reviewing the Covid19 Impact on Market and all the necessary areas of the market that have been altered due to the change caused by Covid19 impact. Get in touch with us for more precise/in-depth information of the Gene Therapy for Age related Macular Degeneration market.

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At the end of May, many states began lifting lockdown restrictions and reopening in order to revive their economies, despite warnings that it was still too early. As a result, by mid-July, around 33 states were reporting higher rates of new cases compared to the previous week with only three states reporting declining rates. Due to this Covid-19 pandemic, there has been disruptions in the supply chain which have made end-use businesses realize destructive in the manufacturing and business process. During this lockdown period, the plastic packaging helps the products to have longer shelf life as the public would not be able to buy new replacements for the expired products because most of the production units are closed.

About Us:Cognitive Market Research is one of the finest and most efficient Market Research and Consulting firm. The company strives to provide research studies which include syndicate research, customized research, round the clock assistance service, monthly subscription services, and consulting services to our clients. We focus on making sure that based on our reports, our clients are enabled to make most vital business decisions in easiest and yet effective way. Hence, we are committed to delivering them outcomes from market intelligence studies which are based on relevant and fact-based research across the global market.Contact Us: +1-312-376-8303Email: [emailprotected]Web: https://www.cognitivemarketresearch.com/

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Reasons Industries to Thrive Post-Pandemic! Gene Therapy for Age related Macular Degeneration Market Report xyz Answers it Analysis by Key Companies...

Md. Labs Developing Vaccines and Therapeutics, Attracting Federal Dollars – Josh Kurtz

A 2016 decision to link the research labs at the University of Maryland College Park with those at the University of Maryland at Baltimore is paying dividends in the competition for federal COVID-19 research dollars, a top education official told a science advisory panel on Wednesday.

The decision was a spinoff of the University of Maryland Strategic Partnership Act, said Laurie Ellen Locasio, vice president for research at the two institutions.

Now well be ranked together by the [National Science Foundation] in the U.S. rankings of research universities and together this makes us [an] over $1 billion public research enterprise, she told the Maryland Life Sciences Advisory Board.

With a multi-billion dollar research powerhouse, the privately-funded Johns Hopkins University, also operating here, Maryland is unusually well-positioned, Locasio said.

Very few states in the country have the distinction of having two research universities with over a billion dollars in research spending per year. Weve really shown up as central to this pandemic.

Martin Rosendale, the CEO of the Maryland Tech Council, said there are so many therapeutics and vaccines in development that keeping up with them all is a daunting task.

Many firms adopted a drop everything approach when the scope of the coronavirus challenge started to become apparent, he said.

We just saw a lot of amazing thing happening here in Maryland so many companies were pivoting immediately to support the response to the pandemic, he said. They were basically dropping their other work and on their own dime spending their own money evaluating their technologies, their platforms, how they could apply them to COVID-19.

Many of the big players have grabbed global headlines for their work on a possible vaccine. But smaller companies are making potentially valuable strides as well, Commerce Secretary Kelly M. Schulz told the panel.

Other firms are working to develop COVID tests that are faster and more reliable.

The Tech Council formed the Maryland COVID-19 Coalition to bring companies together, to make sure they knew who each other were and begin a conversation, Rosendale said. Twenty-five firms were part of the coalitions first call; 40 were on the second.

Chris P. Austin, director of the National Center for Advancing Translation Sciences at NIH, told the panel that the federal government has pumped more than $20 billion into COVID research.

Of that, we know that over $3 billion is coming here to Maryland, Rosendale said. Thats a testament to the amazing vaccine and cell-and-gene therapy industries that have grown up here.

If foundation and other non-government funds are added to the public dollars, the number is well over $4 billion, he added.

To play off the capital regions strengths in biotech, a group of industry leaders in Maryland, Washington, D.C., and Virginia is organizing a pandemic and bio-defense center to help countries around the globe guard against future pandemics.

Because of the prominence of whats going on with COVID-19 development in our region especially within Maryland with vaccine, diagnostics, and therapeutic research we are sort of the epicenter in the world right now, said Richard A. Bendis, president and CEO of BioHealth Innovation, Inc. in Rockville.

People are starting to take notice.

Schulz said the Department of Commerce paused its planned marketing efforts in the spring to focus on the pandemic, but now is ready to gear back up again with an Innovation Uncovered campaign.

Were going back to where we wanted to be at the beginning of the year, which was to push the bio- and the life sciences worlds out, because we have heard from [company executives] that Maryland needs to be seen as a strong, tight ecosystem for this industry, she said.

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Md. Labs Developing Vaccines and Therapeutics, Attracting Federal Dollars - Josh Kurtz

Gene Therapy in Oncology Market Research Report By Application, Type, Key Players, Region and Forecast to 2029 – News Monitoring

Good Growth Opportunities In Global Gene Therapy in Oncology Market: Distinguished Technology Development with Major Production Goals Analysis by 2029

According to the latest market analysis report Gene Therapy in Oncology Market in-depth study and complete information about the market size, market offers and market dynamics. Global Gene Therapy in Oncology Market provides different sections and sub-sections based on separation by type, application, key players, and end-user, segments, developments, topographical areas of this market. This extensive report also highlights key insights on the factors that drive the growth of the market as well as key challenges that are required to Gene Therapy in Oncology growth in the projection period. The report presents a full analysis of the key factors such as developments, trends, predictions, drivers, and business growth, developing trends, competitive landscape analysis. Development strategies and policies are presented as well as manufacturing processes and value structures are also examined presented in the analytical surveying report, besides the cost structures and production methods.

For Better Understanding, Download Sample PDF of Gene Therapy in Oncology Market Research Report @https://market.us/report/gene-therapy-in-oncology-market/request-sample

Global Gene Therapy in Oncology Market gives a comprehensive overview, the key features that are required to improve the growth of the market in the coming future. Gene Therapy in Oncology Market presenting information such as supply and demand situation, the competitive scenario, and the objections for market growth, market opportunities, and the threats faced by Top key players Bristol-Myers Squibb, Cold Genesys, Advantagene, Amgen, AstraZeneca, Bio-Path Holdings, CRISPR Therapeutics, Editas Medicine, Geron Corp, Idera Pharmaceuticals, Intellia Therapeutics, Johnson & Johnson, Marsala Biotech, Merck, . Many of the market players are interested in Gene Therapy in Oncology Market companies, raw material suppliers, machine suppliers, end users, traders, distributors. Building opportunities in this Gene Therapy in Oncology market and the growing progressions in the Gene Therapy in Oncology Market.It has been giving a clear understanding of primary and secondary research techniques and they are now intended towards collaborating accurate and exact data. The data gathered to structure this report is based on the data collection modules with large sample quantities.

The detailed segments and sub-segment of the market are explained below:

Top growing factors by Key Companies like:

Bristol-Myers Squibb, Advantagene, Amgen, Marsala Biotech, Idera Pharmaceuticals, Merck, Geron Corp, Intellia Therapeutics, , Editas Medicine, Johnson & Johnson, CRISPR Therapeutics, AstraZeneca, Cold Genesys and Bio-Path Holdings

Current Market Status, Trends, Types:

Ex Vivo, In Vivo

Review of Market Growth, Future Prospects, and Applications:

Hospitals, Diagnostics Centers, Research Institutes

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Global Gene Therapy in Oncology Market: Regional Segmentation

1.North America (United States, Mexico, and Canada)

2.Europe (France, UK, Germany, Russia, and Italy)

3.Asia-Pacific (Korea, India, China, Japan, and Southeast Asia)

4.South America (Brazil, Argentina, Colombia, etc.)

5.Middle East and Africa (UAE, Egypt, Saudi Arabia, Nigeria, and South Africa)

Research Objectives Of Gene Therapy in Oncology Market Report:

A comprehensive overview of the market share and growth rate by type, application.

A complete analysis of the market opportunities, product scope, market risk, market driving force.

Analyze the top companies of Gene Therapy in Oncology Market Industry, with sales, revenue, and price

The report presents a forward-looking prospect on the global gene therapy in oncology market past data, status, and expected forecast, product, revenue, consumption.

It helps to recognize rising trends, drivers, growth influencing factors in global gene therapy in oncology market and regions.

It helps to prepare Marketing Policies by understanding the rising trends developing and improving gene therapy in oncology market development.

It supports to understand competitive developments such as developments, acquisitions, agreements, and new product launches in the gene therapy in oncology market.

*Please contact us for any specific customized requirements or changes you want and we will offer you the report as your requirement.

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Gene Therapy in Oncology Market Research Report By Application, Type, Key Players, Region and Forecast to 2029 - News Monitoring

VBL Therapeutics Announces Enrollment of the First Patients in the Phase 2 Clinical Trial of VB-111 in Metastatic Colorectal Cancer – GlobeNewswire

TEL AVIV, Israel, Sept. 10, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT) announced today that the first two patients have been enrolled in the Phase 2 clinical trial of VB-111 in combination with nivolumab (Opdivo), an immune checkpoint inhibitor, for the treatment of metastatic colorectal cancer. The study is being conducted under a Cooperative Research and Development Agreement (CRADA) between the National Cancer Institute (NCI) and VBL.

Colon cancer is one of the most common cancers worldwide, but immune-based approaches in gastrointestinal cancers have unfortunately been largely unsuccessful, said Tim F. Greten, M.D., Deputy Branch Chief & Senior Investigator of the Thoracic and GI Malignancies Branch (TGMB), Co-Director of the NCI Center for Cancer Research (CCR) Liver Cancer Program, and the principal investigator of the study. The reasons for this are unclear, but no doubt relate to the fact that, in advanced disease, GI cancer appears to be less immunogenic. The goal of this Phase 2 study is to investigate whether priming with VB-111 followed by the addition of nivolumab may induce anti-tumor immune response in metastatic colorectal cancer, for which there remains a major unmet need.

We are pleased to see beginning of enrollment in this study, despite the challenges of COVID-19, said Dror Harats, M.D., Chief Executive Officer of VBL Therapeutics. We look forward to collaborating with the NCI on investigating VB-111 for the potential benefit of patients with colorectal cancer.

For additional information on the study refer to https://clinicaltrials.gov/show/NCT04166383.

For patients interested in enrolling in this clinical study, please contact NCIs toll-free number 1-800-4-Cancer (1-800-422-6237) (TTY: 1-800-332-8615) and/or the Web site: https://trials.cancer.gov

About VBLVascular Biogenics Ltd., operating asVBL Therapeutics, is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class treatments for areas of unmet need in cancer and immune/inflammatory indications. VBL has developed three platform technologies: a gene-therapy based technology for targeting newly formed blood vessels with focus on cancer, an antibody-based technology targeting MOSPD2 for anti-inflammatory and immuno-oncology applications, and the Lecinoxoids, a family of small-molecules for immune-related indications. VBLs lead oncology product candidate, ofranergene obadenovec (VB-111), is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. It is conveniently administered as an IV infusion once every 6-8 weeks. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in a VBL-sponsored all comers Phase 1 trial as well as in three VBL-sponsored tumor-specific Phase 2 studies. Ofranergene obadenovec is currently being studied in a VBL-sponsored Phase 3 potential registration trial for platinum-resistant ovarian cancer.

Forward Looking StatementsThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. These forward-looking statements include, but are not limited to, statements regarding our programs, including VB-111, including their clinical development, such as the timing of clinical trials and expected announcement of data, therapeutic potential and clinical results, and our financial position and cash runway. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals, the risk that historical clinical trial results may not be predictive of future trial results, the impact of the COVID-19 pandemic on our business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines and clinical results, that our financial resources do not last for as long as anticipated, and that we may not realize the expected benefits of our intellectual property protection. A further list and description of these risks, uncertainties and other risks can be found in our regulatory filings with the U.S. Securities and Exchange Commission, including in our annual report on Form 20-F for the year ended December 31, 2019, and subsequent filings with the SEC. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. VBL Therapeutics undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

INVESTOR CONTACT:

Michael RiceLifeSci Advisorsmrice@lifesciadvisors.com(646) 597-6979

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VBL Therapeutics Announces Enrollment of the First Patients in the Phase 2 Clinical Trial of VB-111 in Metastatic Colorectal Cancer - GlobeNewswire

Government investment to help build robots for nuclear plants and batteries for electric aeroplanes – GOV.UK

Future technologies that could transform peoples lives such as high-performance batteries for electric vehicles, advanced medical treatments and robotics will receive a 65 million government cash boost today (Thursday 10 September).

The funding will be available through the governments Industrial Strategy Challenge Fund, which has been extended today to help develop solutions to some of the biggest global challenges, including climate change and tackling diseases such as cancer and Alzheimers.

Of the investment announced today:

nearly 44 million to develop the next generation of high-performance batteries for electric vehicles and wind turbines, which could also be used for new technologies such as electric aeroplanes. The funding will also be used to complete a first-of-its-kind UK Battery Industrialisation Centre, in Coventry, West Midlands, creating 100 high-skilled jobs. Organisations across the automotive, rail and aerospace sectors will have access a unique battery production facility combining manufacturing, experimentation and innovation

15 million to enable universities, research organisations and businesses to build robots to inspect, maintain and repair nuclear power stations, satellites and wind turbines. The robotics will also be used to address new problems resulting from the pandemic, including ones that can operated remotely and make contact-free deliveries or move hospital beds.

6.5 million will be allocated to the Advanced Therapy Treatment Centre network to accelerate patient access to advanced therapies through the development of specialised infrastructure for the delivery of these products in the NHS. These cell and gene based therapies are aimed at the treatment of life-limiting and inherited diseases such as cancer, Duchenne muscular dystrophy or cystic fibrosis.

Science Minister Amanda Solloway said:

We want to build back better by putting the UK at the forefront of new technologies to create high-skilled jobs, increase productivity and grow the economy as we recover from coronavirus.

This new funding will strengthen the UKs global status in a range of areas, including battery technologies for electric vehicles and robotics, helping us develop innovative solutions to some of our biggest global challenges and creating jobs in rewarding careers right across the country.

Challenge Director for The Faraday Battery Challenge Tony Harper said:

In order for batteries to play their full environmental and economic role in achieving Net Zero we need to deploy at scale and build supply chains for todays technology, shift from strong potential to commercial dominance in a new generation of batteries and continue to build world-class scientific capability to sustain us into the future. The announcement today confirms our commitment and determination to build on the hard-won progress the UK has made in the last 3 years on all these fronts and to accelerate progress post COVID-19.

CEO of the Cell and Gene Therapy Catapult Matthew Durdy said:

The ATTC network is a fantastic example of effective government intervention and the international community recognises this as part of the UKs leadership in the field. Bringing together companies, the NHS and regulatory bodies to make the use of cell and gene therapies easier, more cost effective, and more widespread both boosts the industry and brings these life changing medicines to patients who need them.

The fact that 12% of global clinical trials in cell and gene therapy take place in the UK and half of those involve ATTCs is a testament to the success of this highly respected programme.

Andrew Tyrer, Challenge Director for Robotics for a Safer World said:

I am delighted that the government has provided an extra 15 million funding to help academics and businesses bridge the gap to: complete on-going deliverables set against the Robots for a Safer World Challenge, and also; utilise knowledge gained to the benefit of new sectors, ahead of this Autumns spending review.

Established in 2017, the Industrial Strategy Challenge Fund is delivering 2.6 billion of government investment with the aim of funding world-leading research and highly innovative businesses to address the biggest industrial and societal challenges.

Todays announcement furthers the governments commitment through its R&D Roadmap to put the UK at the forefront of transformational technologies and is part of the governments wider commitment to increase R&D investment to 2.4% of GDP by 2027.

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Government investment to help build robots for nuclear plants and batteries for electric aeroplanes - GOV.UK

Was COVID-19 Manmade? Meet the Scientist Behind the Theory – Boston magazine

Research

The worlds preeminent scientists say a theory from the Broad Institutes Alina Chan is too wild to be believed. But when the theory is about the possibility of COVID being man-made, is this science or censorship?

Illustration by Benjamen Purvis

In January, as she watched the news about a novel virus spreading out of control in China, Alina Chan braced for a shutdown. The molecular biologist at the Broad Institute of Harvard and MIT started stockpiling medicine and supplies. By the time March rolled around and a quarantine seemed imminent, shed bought hundreds of dollars worth of fillets from her favorite fishmonger in Cambridge and packed them into her freezer. Then she began to ramp down her projects in the lab, isolating her experimental cells from their cultures and freezing them in small tubes.

As prepared as she was for the shutdown, though, she found herself unprepared for the frustration of being frozen out of work. She paced the walls of her tiny apartment feeling bored and useless. Chan has been a puzzle demon since childhood, which was precisely what she loved about her workthe chance to solve fiendishly difficult problems about how viruses operate and how, through gene therapy, they could be repurposed to help cure devastating genetic diseases. Staring out her window at the eerily quiet streets of her Inman Square neighborhood, she groaned at the thought that it could be months before she was at it again. Her mind wandered back to 2003, when she was a teenager growing up in Singapore and the first SARS virus, a close relative of this coronavirus, appeared in Asia. It hadnt been anything like this. That one had been relatively easy to corral. How had this virus come out of nowhere and shut down the planet? Why was it so different? she asked herself.

Then it hit her: The worlds greatest puzzle was staring her in the face. Stuck at home, all she had to work with was her brain and her laptop. Maybe they were enough. Chan fired up the kettle for the first of what would become hundreds of cups of tea, stacked four boxes on her kitchen counter to raise her laptop to the proper height, pulled back her long dark hair, and began reading all of the scientific literature she could find on the coronavirus.

It wasnt long before she came across an article about the remarkable stability of the virus, whose genome had barely changed from the earliest human cases, despite trillions of replications. This perplexed Chan. Like many emerging infectious diseases, COVID-19 was thought to be zoonoticit originated in animals, then somehow found its way into people. At the time, the Chinese government and most scientists insisted the jump had happened at Wuhans seafood market, but that didnt make sense to Chan. If the virus had leapt from animals to humans in the market, it should have immediately started evolving to life inside its new human hosts. But it hadnt.

On a hunch, she decided to look at the literature on the 2003 SARS virus, which had jumped from civets to people. Bingo. A few papers mentioned its rapid evolution in its first months of existence. Chan felt the familiar surge of puzzle endorphins. The new virus really wasnt behaving like it should. Chan knew that delving further into this puzzle would require some deep genetic analysis, and she knew just the person for the task. She opened Google Chat and fired off a message to Shing Hei Zhan. He was an old friend from her days at the University of British Columbia and, more important, he was a computational god.

Do you want to partner on a very unusual paper? she wrote.

Sure, he replied.

One thing Chan noticed about the original SARS was that the virus in the first human cases was subtly differenta few dozen letters of genetic codefrom the one in the civets. That meant it had immediately morphed. She asked Zhan to pull up the genomes for the coronaviruses that had been found on surfaces in the Wuhan seafood market. Were they at all different from the earliest documented cases in humans?

Zhan ran the analysis. Nope, they were 100 percent the same. Definitely from humans, not animals. The seafood-market theory, which Chinese health officials and the World Health Organization espoused in the early days of the pandemic, was wrong. Chans puzzle detectors pulsed again. Shing, she messaged Zhan, this paper is going to be insane.

In the coming weeks, as the spring sun chased shadows across her kitchen floor, Chan stood at her counter and pounded out her paper, barely pausing to eat or sleep. It was clear that the first SARS evolved rapidly during its first three months of existence, constantly fine-tuning its ability to infect humans, and settling down only during the later stages of the epidemic. In contrast, the new virus looked a lot more like late-stage SARS. Its almost as if were missing the early phase, Chan marveled to Zhan. Or, as she put it in their paper, as if it was already well adapted for human transmission.

That was a profoundly provocative line. Chan was implying that the virus was already familiar with human physiology when it had its coming-out party in Wuhan in late 2019. If so, there were three possible explanations.

Perhaps it was just staggeringly bad luck: The mutations had all occurred in an earlier host species, and just happened to be the perfect genetic arrangement for an invasion of humanity. But that made no sense. Those mutations would have been disadvantageous in the old host.

Maybe the virus had been circulating undetected in humans for months, working out the kinks, and nobody had noticed. Also unlikely. Chinas health officials would not have missed it, and even if they had, theyd be able to go back now through stored samples to find the trail of earlier versions. And they werent coming up with anything.

That left a third possibility: The missing phase had happened in a lab, where the virus had been trained on human cells. Chan knew this was the third rail of potential explanations. At the time, conspiracy theorists were spinning bioweapon fantasies, and Chan was loath to give them any ammunition. But she also didnt want to play politics by withholding her findings. Chan is in her early thirties, still at the start of her career, and an absolute idealist about the purity of the scientific process. Facts were facts.

Or at least they used to be. Since the start of the pandemic, the Trump administration has been criticized for playing fast and loose with factsdenying, exaggerating, or spinning them to suit the presidents political needs. As a result, many scientists have learned to censor themselves for fear that their words will be misrepresented. Still, Chan thought, if she were to sit on scientific research just to avoid providing ammunition to conspiracy theorists or Trump, would she be any better than them?

Chan knew she had to move forward and make her findings public. In the final draft of her paper, she torpedoed the seafood-market theory, then laid out a case that the virus seemed curiously well adapted to humans. She mentioned all three possible explanations, carefully wording the third to emphasize that if the novel coronavirus did come from a lab, it would have been the result of an accident in the course of legitimate research.

On May 2, Chan uploaded the paper to a site where as-yet-unpublished biology papers known as preprints are shared for open peer review. She tweeted out the news and waited. On May 16, the Daily Mail, a British tabloid, picked up her research. The very next day, Newsweek ran a story with the headline Scientists Shouldnt Rule Out Lab as Source of Coronavirus, New Study Says.

And that, Chan says, is when shit exploded everywhere.

Alina Chan, a molecular biologist at the Broad Institute, says we cant rule out the possibility that the novel coronavirus originated in a labeven though she knows its a politically radioactive thing to say. / Photo by Mona Miri

Chan had come to my attention a week before the Newsweek story was published through her smart and straightforward tweets, which I found refreshing at a time when most scientists were avoiding any serious discussion about the possibility that COVID-19 had escaped from a biolab. Id written a lot about genetic engineering and so-called gain-of-function researchthe fascinating, if scary, line of science in which scientists alter viruses to make them more transmissible or lethal as a way of assessing how close those viruses are to causing pandemics. I also knew that deadly pathogens escape from biolabs with surprising frequency. Most of these accidents end up being harmless, but many researchers have been infected, and people have died as a result.

For years, concerned scientists have warned that this type of pathogen research was going to trigger a pandemic. Foremost among them was Harvard epidemiologist Marc Lipsitch, who founded the Cambridge Working Group in 2014 to lobby against these experiments. In a series of policy papers, op-eds, and scientific forums, he pointed out that accidents involving deadly pathogens occurred more than twice a week in U.S. labs, and estimated that just 10 labs performing gain-of-function research over a 10-year period would run a nearly 20 percent risk of an accidental release. In 2018, he argued that such a release could lead to global spread of a virulent virus, a biosafety incident on a scale never before seen.

Thanks in part to the Cambridge Working Group, the federal government briefly instituted a moratorium on such research. By 2017, however, the ban was lifted and U.S. labs were at it again. Today, in the United States and across the globe, there are dozens of labs conducting experiments on a daily basis with the deadliest known pathogens. One of them is the Wuhan Institute of Virology. For more than a decade, its scientists have been discovering coronaviruses in bats in southern China and bringing them back to their lab in Wuhan. There, they mix genes from different strains of these novel viruses to test their infectivity in human cells and lab animals.

When word spread in January that a novel coronavirus had caused an outbreak in Wuhanwhich is a thousand miles from where the bats that carry this lineage of viruses are naturally foundmany experts were quietly alarmed. There was no proof that the lab was the source of the virus, but the pieces fit.

Despite the evidence, the scientific community quickly dismissed the idea. Peter Daszak, president of EcoHealth Alliance, which has funded the work of the Wuhan Institute of Virology and other labs searching for new viruses, called the notion preposterous, and many other experts echoed that sentiment.

That wasnt necessarily what every scientist thought in private, though. They cant speak directly, one scientist told me confidentially, referring to the virology communitys fear of having their comments sensationalized in todays politically charged environment. Many virologists dont want to be hated by everyone in the field.

There are other potential reasons for the pushback. Theres long been a sense that if the public and politicians really knew about the dangerous pathogen research being conducted in many laboratories, theyd be outraged. Denying the possibility of a catastrophic incident like this, then, could be seen as a form of career preservation. For the substantial subset of virologists who perform gain-of-function research, Richard Ebright, a Rutgers microbiologist and another founding member of the Cambridge Working Group, told me, avoiding restrictions on research funding, avoiding implementation of appropriate biosafety standards, and avoiding implementation of appropriate research oversight are powerful motivators. Antonio Regalado, biomedicine editor of MIT Technology Review, put it more bluntly. If it turned out COVID-19 came from a lab, he tweeted, it would shatter the scientific edifice top to bottom.

Thats a pretty good incentive to simply dismiss the whole hypothesis, but it quickly amounted to a global gaslighting of the mediaand, by proxy, the public. An unhealthy absolutism set in: Either you insisted that any questions about lab involvement were absurd, or you were a tool of the Trump administration and its desperation to blame China for the virus. I was used to social media pundits ignoring inconvenient or politically toxic facts, but Id never expected to see that from some of our best scientists.

Which is why Chan stood out on Twitter, daring to speak truth to power. It is very difficult to do research when one hypothesis has been negatively cast as a conspiracy theory, she wrote. Then she offered some earnest advice to researchers, suggesting that most viral research should be done with neutered viruses that have had their replicating machinery removed in advance, so that even if they escaped confinement, they would be incapable of making copies of themselves. When these precautions are not followed, risk of lab escape is exponentially higher, she explained, adding, I hope the pandemic motivates local ethics and biosafety committees to think carefully about how they can reduce risk. She elaborated on this in another tweet several days later: Id alsopersonallyprefer if high biosafety level labs were not located in the most populous cities on earth.

How Safe Are Bostons Biolabs?

As one of the world centers of biotech, the Hub is peppered with academic and corporate labs doing research on pathogens. Foremost among them is Boston Universitys National Emerging Infectious Diseases Laboratories (NEIDL), the only lab in the city designated as BSL-4 (the highest level of biosafety and the same level as the Wuhan Institute of Virology). It is one of just a dozen or so in the United States equipped to work with live versions of the worlds most dangerous viruses, including Ebola and Marburg. Researchers there began doing so in 2018 after a decade of controversy: Many locals objected to the risks of siting such a facility in the center of a major metropolitan area.

The good news? Before opening, NEIDL undertook one of the most thorough risk assessments in history, learning from the mistakes of other facilities. Even Lynn Klotz, a senior science fellow at the Washington, DCbased Center for Arms Control and Non-Proliferation, who advised local groups that opposed NEIDL, told the medical website Contagion that the lab likely has the best possible security protocols and measures in place.

But the reality, Klotz added, is that most lab accidents are caused by human error, and there is only so much that can be done through good design and protocols to proactively prevent such mistakes. (Or to guard against an intentional release by a disgruntled researcher, as allegedly happened in the anthrax attacks of 2001.) Rutgers molecular biologist Richard Ebright, a longtime critic of potentially dangerous pathogen research, says the risks introduced by NEIDL are not low enough and definitely not worth the negligible benefits.

Still, risk is relative. Klotz has estimated the chance of a pathogen escape from a BSL-4 lab at 0.3 percent per year, and NEIDL is probably significantly safer than the typical BSL-4 lab. And if catching a deadly pathogen is your fear, well, currently you run a good risk of finding one in your own neighborhood. Until that gets cleared up, the citys biolabs are probably among the safer spaces in town.

Chan had started using her Twitter account this intensely only a few days earlier, as a form of outreach for her paper. The social platform has become the way many scientists find out about one anothers work, and studies have shown that attention on Twitter translates to increased citations for a paper in scientific literature. But its a famously raw forum. Many scientists are not prepared for the digital storms that roil the Twitterverse, and they dont handle it well. Chan dreaded it at first, but quickly took to Twitter like a digital native. Having Twitter elevates your work, she says. And I think its really fun to talk to nonscientists about that work.

After reading her tweets, I reviewed her preprint, which I found mind-blowing, and wrote her to say so. She thanked me and joked that she worried it might be career suicide.

It wasnt long before it began to look like she might be right.

Speaking her mind, it turns outeven in the face of censurewas nothing new for Chan, who is Canadian but was raised in Singapore, one of the more repressive regimes on earth. Her parents, both computer science professionals, encouraged free thinking and earnest inquiry in their daughter, but the local school system did not. Instead, it was a pressure-cooker of a system that rewarded students for falling in line, and moved quickly to silence rebels.

That was a bad fit for Chan. You have to bow to teachers, she says. Sometimes teachers from other classes would show up and ask me to bow to them. And I would say, No, youre not my teacher. Back then they believed in corporal punishment. A teacher could just take a big stick and beat you in front of the class. I got whacked so many times.

Still, Chan rebelled in small ways, skipping school and hanging out at the arcade. She also lost interest in her studies. I just really didnt like school. And I didnt like all the extracurriculars they pack you with in Singapore, she says. That changed when a teacher recruited her for math Olympiads, in which teams of students compete to solve devilishly hard arithmetic puzzles. I really loved it, she says. You just sit in a room and think about problems.

Chan might well have pursued a career in math, but then she came up against teams from China in Olympiad competitions. They would just wipe everyone else off the board, she says. They were machines. Theyd been trained in math since they could walk. Theyd hit the buzzer before you could even comprehend the question. I thought, Im not going to survive in this field.

Chan decided to pursue biology instead, studying at the University of British Columbia. I liked viruses from the time I was a teen, she says. I remember the first time I learned about HIV. I thought it was a puzzle and a challenge. That instinct took her to Harvard Medical School as a postdoc, where the puzzle became how to build virus-like biomolecules to accomplish tasks inside cells, and then to Ben Devermans lab at the Broad Institute. When I see an interesting question, I want to spend 100 percent of my time working on it, she says. I get really fixated on answering scientific questions.

Deverman, for his part, says he wasnt actively looking to expand his team when Chan came along, but when opportunities to hire extraordinary people fall in my lap, he takes them. Alina brings a ton of value to the lab, he explains, adding that she has an ability to pivot between different topics and cut to the chase. Nowhere was that more on display than with her coronavirus work, which Deverman was able to closely observe. In fact, Chan ran so many ideas past him that he eventually became a coauthor. She is insightful, determined, and has the rare ability to explain complex scientific findings to other scientists and to the public, he says.

Those skills would prove highly useful when word got out about her coronavirus paper.

If Chan had spent a lifetime learning how to pursue scientific questions, she spent most of the shutdown learning what happens when the answers you come up with are politically radioactive. After the Newsweek story ran, conservative-leaning publications seized on her paper as conclusive evidence that the virus had come from a lab. Everyone focused on the one line, Chan laments. The tabloids just zoomed in on it. Meanwhile, conspiracists took it as hard evidence of their wild theories that there had been an intentional leak.

Chan spent several exhausting days putting out online fires with the many people who had misconstrued her findings. I was so naive, she tells me with a quick, self-deprecating laugh. I just thought, Shouldnt the world be thinking about this fairly? I really have to kick myself now.

Even more troubling, though, were the reactions from other scientists. As soon as her paper got picked up by the media, luminaries in the field sought to censure her. Jonathan Eisen, a well-known professor at UC Davis, criticized the study in Newsweek and on his influential Twitter account, writing, Personally, I do not find the analysis in this new paper remotely convincing. In a long thread, he argued that comparing the new virus to SARS was not enough to show that it was preadapted to humans. He wanted to see comparisons to the initial leap of other viruses from animals to humans.

Moments later, Daszak piled on. The NIH had recently cut its grant to his organization, EcoHealth Alliance, after the Trump administration learned that some of it had gone to fund the Wuhan Institute of Virologys work. Daszak was working hard to get it restored and trying to stamp out any suggestion of a lab connection. He didnt hold back on Chan. This is sloppy research, he tweeted, calling it a poorly designed phylogenetic study with too many inferences and not enough data, riding on a wave of conspiracy to drive a higher impact. Peppering his tweets with exclamation points, he attacked the wording of the paper, arguing that one experiment it cited was impossible, and told Chan she didnt understand her own data. Afterward, a Daszak supporter followed up his thread with a GIF of a mike drop.

It was an old and familiar dynamic: threatened silverback male attempts to bully a junior female member of the tribe. As a postdoc, Chan was in a vulnerable position. The world of science is still a bit medieval in its power structure, with a handful of institutions and individuals deciding who gets published, who gets positions, who gets grants. Theres little room for rebels.

What happened next was neither old nor familiar: Chan didnt back down. Sorry to disrupt mike drop, she tweeted, providing a link to a paper in the prestigious journal Nature that does that exact experiment you thought was impossible. Politely but firmly, she justified each point Daszak had attacked, showing him his mistakes. In the end, Daszak was reduced to arguing that she had used the word isolate incorrectly. In a coup de grce, Chan pointed out that actually the word had come from online data provided by GenBank, the NIHs genetic sequence database. She offered to change it to whatever made sense. At that point, Daszak stopped replying. He insists, however, that Chan is overinterpreting her findings.

With Eisen, Chan readily agreed to test her hypothesis by finding other examples of viruses infecting new hosts. Within days, a perfect opportunity came along when news broke that the coronavirus had jumped from humans to minks at European fur farms. Sure enough, the mink version began to rapidly mutate. You actually see the rapid evolution happening, Chan said. Just in the first few weeks, the changes are quite drastic.

Chan also pointed out to Eisen that the whole goal of a website such as bioRxiv (pronounced bioarchive)where she posted the paperis to elicit feedback that will make papers better before publication. Good point, he replied. Eventually he conceded that there was a lot of interesting analysis in the paper and agreed to work with Chan on the next draft.

The Twitter duels with her powerful colleagues didnt rattle Chan. I thought Jonathan was very reasonable, she says. I really appreciated his expertise, even if he disagreed with me. I like that kind of feedback. It helped to make our paper better.

With Daszak, Chan is more circumspect. Some people have trouble keeping their emotions in check, she says. Whenever I saw his comments, Id just think, Is there something I can learn here? Is there something hes right about that I should be fixing? Ultimately, she decided, there was not.

By late May, both journalists and armchair detectives interested in the mystery of the coronavirus were discovering Chan as a kind of Holmes to our Watson. She crunched information at twice our speed, zeroing in on small details wed overlooked, and became a go-to for anyone looking for spin-free explications of the latest science on COVID-19. It was thrilling to see her reasoning in real time, a reminder of why Ive always loved science, with its pursuit of patterns that sometimes leads to exciting revelations. The website CNET featured her in a story about a league of scientists-turned-detectives who were using genetic sequencing technologies to uncover COVID-19s origins. After it came out, Chan added scientist-turned-detective to her Twitter bio.

Shes lived up to her new nom de tweet. As the search for the source of the virus continued, several scientific teams published papers identifying a closely related coronavirus in pangolinsanteater-like animals that are heavily trafficked in Asia for their meat and scales. The number of different studies made it seem as though this virus was ubiquitous in pangolins. Many scientists eagerly embraced the notion that the animals might have been the intermediate hosts that had passed the novel coronavirus to humans. It fit their preexisting theories about wet markets, and it would have meant no lab had been involved.

As Chan read the pangolin papers, she grew suspicious. The first one was by a team that had analyzed a group of the animals intercepted by anti-smuggling authorities in southern China. They found the closely related virus in a few of them, and published the genomes for that virus. Some of the other papers, though, were strangely ambiguous about where their data was coming from, or how their genomes had been constructed. Had they really taken samples from actual pangolins?

Once again, Chan messaged Shing Hei Zhan. Shing, somethings weird here, she wrote. Zhan pulled up the raw data from the papers and compared the genomes they had published. Individual copies of a virus coming from different animals should have small differences, just as individuals of a species have genetic differences. Yet the genomes in all of the pangolin papers were perfect matchesthe authors were all simply using the first groups data set. Far from being ubiquitous, the virus had been found only in a few pangolins who were held together, and it was unclear where they had caught it. The animals might have even caught it from their own smuggler.

Remarkably, one group of authors in Nature even appeared to use the same genetic sequences from the other paper as if it were confirmation of their own discovery. These sequences appear to be from the same virus (Pangolin-CoV) that we identified in the present study.

Chan called them out on Twitter: Of course its the same Pangolin-CoV, you used the same dataset! For context, she later added, Imagine if clinical trials were playing fast and loose with their patient data; renaming patients, throwing them into different datasets without clarification, possibly even describing the same patient multiple times across different studies unintentionally.

She and Zhan posted a new preprint on bioRxiv dismantling the pangolin papers. Confirmation came in June when the results of a study of hundreds of pangolins in the wildlife trade were announced: Not a single pangolin had any sign of a coronavirus. Chan took a victory lap on Twitter: Supports our hypothesis all this time. The pangolin theory collapsed.

Chan then turned her Holmesian powers on bigger game: Daszak and the Wuhan Institute of Virology. Daszak had been pleading his case everywhere from 60 Minutes to the New York Times and has been successful in rallying sympathy to his cause, even getting 77 Nobel laureates to sign a letter calling for the NIH to restore EcoHealth Alliances funding.

In several long and detailed tweetorials, Chan began to cast a cloud of suspicion on the WIVs work. She pointed out that scientists there had discovered a virus that is more than 96 percent identical to the COVID-19 coronavirus in 2013 in a mineshaft soon after three miners working there had died from a COVID-like illness. The WIV didnt share these findings until 2020, even though the goal of such work, Chan pointed out, was supposedly to identify viruses with the potential to cause human illnesses and warn the world about them.

Even though that virus had killed three miners, Daszak said it wasnt considered a priority to study at the time. We were looking for SARS-related virus, and this one was 20 percent different. We thought it was interesting, but not high risk. So we didnt do anything about it and put it in the freezer, he told a reporter from Wired. It was only in 2020, he maintained, that they started looking into it once they realized its similarity to COVID-19. But Chan pointed to an online database showing that the WIV had been genetically sequencing the mine virus in 2017 and 2018, analyzing it in a way they had done in the past with other viruses in preparation for running experiments with them. Diplomatic yet deadpan, she wrote, I think Daszak was misinformed.

For good measure, almost in passing, Chan pointed out a detail no one else had noticed: COVID-19 contains an uncommon genetic sequence that has been used by genetic engineers in the past to insert genes into coronaviruses without leaving a trace, and it falls at the exact point that would allow experimenters to swap out different genetic parts to change the infectivity. That same sequence can occur naturally in a coronavirus, so this was not irrefutable proof of an unnatural origin, Chan explained, only an observation. Still, it was enough for one Twitter user to muse, If capital punishment were as painful as what Alina Chan is doing to Daszak/WIV regarding their story, it would be illegal.

Daszak says that indeed he had been misinformed and was unaware that that virus found in the mine shaft had been sequenced before 2020. He also says that a great lab, with great scientists, is now being picked apart to search for suspicious behavior to support a preconceived theory. If you believe, deep down, something fishy went on, then what you do is you go through all the evidence and you try to look for things that support that belief, he says, adding, That is not how you find the truth.

Many of the points in Chans tweetorials had also been made by others, but she was the first reputable scientist to put it all together. That same week, Londons Sunday Times and the BBC ran stories following the same trail of breadcrumbs that Chan had laid out to suggest that there had been a coverup at the WIV. The story soon circulated around the world. In the meantime, the WIV has steadfastly denied any viral leak. Lab director Yanyi Wang went on Chinese television and described such charges as pure fabrication, and went on to explain that the bat coronavirus from 2013 was so different than COVID that it could not have evolved into it this quickly and that the lab only sequenced it and didnt obtain a live virus from it.

To this day, there is no definitive evidence as to whether the virus occurred naturally or had its origins in a lab, but the hypothesis that the Wuhan facility was the source is increasingly mainstream and the science behind it can no longer be ignored. And Chan is largely to thank for that.

In late spring, Chan walked through the tall glass doors of the Broad Institute for the first time in months. As she made her way across the gleaming marble foyer, her sneaker squeaks echoed in the silence. It was like the zombie apocalypse version of the Broad; all the bright lights but none of the people. It felt all the weirder that she was wearing her gym clothes to work.

A few days earlier, the Broad had begun letting researchers back into their labs to restart their projects. All computer work still needed to be done remotely, but bench scientists such as Chan could pop in just long enough to move along their cell cultures, provided they got tested for the virus every four days.

In her lab, Chan donned her white lab coat and took inventory, throwing out months of expired reagents and ordering new materials. Then she rescued a few samples from the freezer, took her seat at one of the tissue-culture hoodsstainless steel, air-controlled cabinets in which cell engineers do their workand began reviving some of her old experiments.

She had mixed emotions about being back. It felt good to free her gene-therapy projects from their stasis, and she was even more excited about the new project she and Deverman were working on: an online tool that allows vaccine developers to track changes in the viruss genome by time, location, and other characteristics. It came out of my personal frustration at not being able to get answers fast, she says.

On the other hand, she missed being all-consumed by her detective work. I wanted to stop after the pangolin preprint, she says, but this mystery keeps drawing me back in. So while she waits for her cell cultures to grow, shes been sleuthing on the sideonly this time she has more company: Increasingly, scientists have been quietly contacting her to share their own theories and papers about COVID-19s origins, forming something of a growing underground resistance. Theres a lot of curiosity, she says. People are starting to think more deeply about it. And they have to, she says, if we are going to prevent future outbreaks: Its really important to find out where this came from so it doesnt happen again.

That is what keeps Chan up at nightthe possibility of new outbreaks in humans from the same source. If the virus emerged naturally from a bat cave, there could well be other strains in existence ready to spill over. If they are closely related, whatever vaccines we develop might work on them, too. But that might not be the case with manipulated viruses from a laboratory. Someone could have been sampling viruses from different caves for a decade and just playing mix-and-match in the lab, and those viruses could be so different from one another that none of our vaccines will work on them, she says. Either way, We need to find where this came from, and close it down.

Whatever important information she finds, we can be sure Chan will share it with the world. Far from being shaken by the controversy her paper stirred, she is more committed than ever to holding a line that could all too easily be overrun. Scientists shouldnt be censoring themselves, she says. Were obliged to put all the data out there. We shouldnt be deciding that its better if the public doesnt know about this or that. If we start doing that, we lose credibility, and eventually we lose the publics trust. And thats not good for science. In fact, it would cause an epidemic of doubt, and that wouldnt be good for any of us.

Link:
Was COVID-19 Manmade? Meet the Scientist Behind the Theory - Boston magazine

Catalyst Biosciences to Participate in Two Upcoming Investor Conferences – GlobeNewswire

SOUTH SAN FRANCISCO, Calif., Sept. 10, 2020 (GLOBE NEWSWIRE) -- Catalyst Biosciences, Inc. (NASDAQ: CBIO) today announced thatNassim Usman, Ph.D., president and chief executive officer and Clinton Musil, chief financial officer ofCatalyst Biosciences, will participate in fireside chats at two investor conferences in September: The Morgan Stanley Global Healthcare Conference and the Cantor Virtual Global Healthcare Conference. Details for each can be found below.

An archived webcast of the presentations will be available for 90 days on theEvents and Presentationspage under the Investors section of the Companys website.

About Catalyst BiosciencesCatalyst is a research and clinical development biopharmaceutical company focused on addressing unmet needs in rare hematologic and complement-mediated disorders. Our protease engineering platform includes two late-stage clinical programs in hemophilia; a research program on engineering of subcutaneous (SQ) complement inhibitors; and a partnered preclinical development program with Biogen for dry age-related macular degeneration (AMD). The product candidates generated by our protease engineering platform have improved functionality and potency that allow for: SQ administration of recombinant coagulation factors and complement inhibitors; low-dose, high activity gene therapy constructs; and less frequently dosed intravitreal therapeutics. Our most advanced product candidate is marzeptacog alfa (activated) (MarzAA), a next-generation SQ FVIIa entering a Phase 3 registration study in late 2020. Our next late-stage product candidate is dalcinonacog alfa (DalcA), a next-generation SQ FIX, which has demonstrated efficacy and safety in a Phase 2b clinical trial in individuals with Hemophilia B. 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.

Forward-Looking StatementsThis press release contains forward-looking statements that involve substantial risks and uncertainties. Forward-looking statements include statements about the potential benefits of products based on Catalysts engineered protease platform, plans to enroll the first patients in a Phase 3 registration study of MarzAA in late 2020, the potential for MarzAA and DalcA to effectively and therapeutically treat hemophilia subcutaneously, the superiority of CB 2679d-GT over other gene therapy, and the development of anti-complement Factor 3 (C3) pegylated CB 2782 by 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 pandemic and other factors, that trials may not have satisfactory outcomes, that additional human trials will not replicate the results from earlier trials, that potential adverse effects may arise from the testing or use of DalcA or MarzAA, including the generation of neutralizing antibodies, which has been observed in patients treated with DalcA, the risk that costs required to develop or manufacture the Companys products will be higher than anticipated, including as a result of delays in development and manufacturing resulting from COVID-19 and other factors, the risk that Biogen will terminate Catalysts agreement, competition and other risks described in the Risk Factors section of the Companys quarterly report filed with the Securities and Exchange Commission on August 6, 2020, and in other filings with the Securities and Exchange Commission. The Company does not assume any obligation to update any forward-looking statements, except as required by law.

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

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Catalyst Biosciences to Participate in Two Upcoming Investor Conferences - GlobeNewswire

The Global Biologic Therapeutics Market is expected to grow by $ 190.94 bn during 2020-2024 progressing at a CAGR of 12% during the forecast period -…

New York, Sept. 10, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Biologic Therapeutics Market 2020-2024" - https://www.reportlinker.com/p02448159/?utm_source=GNW Our reports on biologic therapeutics market provides a holistic analysis, market size and forecast, trends, growth drivers, and challenges, as well as vendor analysis covering around 25 vendors. The report offers an up-to-date analysis regarding the current global market scenario, latest trends and drivers, and the overall market environment. The market is driven by the strong R&D pipeline, targeted therapeutic mechanism of biologics, and increasing investment in development of biologics. In addition, strong R&D pipeline is anticipated to boost the growth of the market as well. The biologic therapeutics market analysis includes product segment and geographical landscapes

The biologic therapeutics market is segmented as below: By Product Antibody therapeutics Vaccines Cell therapy Gene therapy Other therapies

By Geographic Landscapes North America Europe Asia ROW

This study identifies the introduction of biosimilars as one of the prime reasons driving the biologic therapeutics market growth during the next few years. Also, increasing incidence of chronic diseases and immunological disorders and patent expiry of major biologics will lead to sizable demand in the market.

The analyst presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources by an analysis of key parameters. Our biologic therapeutics market covers the following areas: Biologic therapeutics market sizing Biologic therapeutics market forecast Biologic therapeutics market industry analysis

Read the full report: https://www.reportlinker.com/p02448159/?utm_source=GNW

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The Global Biologic Therapeutics Market is expected to grow by $ 190.94 bn during 2020-2024 progressing at a CAGR of 12% during the forecast period -...

Inflammation in the Liver Reversed by Metformin – Genetic Engineering & Biotechnology News

The diabetes drug metforminderived from a lilac plant thats been used medicinally for more than a thousand yearshas been prescribed to hundreds of millions of people worldwide as the frontline treatment for type 2 diabetes. Yet scientists dont fully understand how the drug is so effective at controlling blood glucose.

Now, researchers at the Salk Institute say they have shown the importance of specific enzymes in the body for metformins function. In addition, the new work showed that the same proteins, regulated by metformin, controlled aspects of inflammation in mice, something the drug has not typically been prescribed for.

Apart from clarifying how metformin works, the research AMPK regulation of Raptor and TSC2 mediate Metformin effects on transcriptional control of anabolism and inflammation, which appears in Genes & Developmentreportedly has relevance for many other inflammatory diseases.

Despite being the frontline therapy for type 2 diabetes, the mechanisms of action of the biguanide drug metformin are still being discovered. In particular, the detailed molecular interplays between the AMPK and the mTORC1 pathway in the hepatic benefits of metformin are still ill-defined. Metformin-dependent activation of AMPK classically inhibits mTORC1 via TSC/RHEB. But several lines of evidence suggest additional mechanisms at play in metformin inhibition of mTORC1, write the investigators.

Here we investigated the role of direct AMPK-mediated serinephosphorylation of RAPTOR in a new RaptorAA mouse model, in which AMPK phospho-serine sites Ser722 and Ser792 of RAPTOR were mutated to alanine. Metformin treatment of primary hepatocytes and intact murine liver requires AMPK regulation of both RAPTOR and TSC2 to fully inhibit mTORC1, and this regulation is critical for both the translational and transcriptional response to metformin.

Transcriptionally, AMPK and mTORC1 were both important for regulation of anabolic metabolism and inflammatory programs triggered by metformin treatment. The hepatic transcriptional response in mice on high fat diet treated with metformin was largely ablated by AMPK-deficiency under the conditions examined, indicating the essential role of this kinase and its targets in metformin action in vivo.

These findings let us dig into precisely what metformin is doing at a molecular level, says Reuben Shaw, PhD, a professor in Salks Molecular and Cell Biology Laboratory and the senior author of the new paper. This more granular understanding of the drug is important because there is increasing interest in targeting these pathways for not only diabetes but immune diseases and cancer.

Researchers have known for 20 years that metformin activates a metabolic master switch, a protein called AMPK, which conserves a cells energy under low nutrient conditions, and which is activated naturally in the body following exercise. Twelve years ago, Shaw discovered that in healthy cells, AMPK starts a cascade effect, regulating two proteins called Raptor and TSC2, which results in a block of the central pro-growth protein complex called mTORC1 (mammalian target of rapamycin complex 1).

These findings helped explain the ability of metformin to inhibit the growth of tumor cells, an area of research that began to generate excitement after Shaw and others connected AMPK to a bona fide cancer gene in the early 2000s.

But in the intervening years, many additional proteins and pathways that metformin regulates have been discovered, drawing into question which of the targets of metformin are most important for different beneficial consequences of metformin treatment. Indeed, metformin is currently entering clinical trials in the United States as a general anti-aging treatment because it is effects are so well established from millions of patients and its side effects are minimal.

But whether AMPK or its targets Raptor or TSC2 are important for different effects of metformin remains poorly understood.

In the new work, in mice, Shaw and his colleagues genetically disconnected the master protein, AMPK, from the other proteins, so they could not receive signals from AMPK, but were able to otherwise function normally and receive input from other proteins.

When these mice were put on a high-fat diet triggering diabetes and then treated with metformin, the drug no longer had the same effects on liver cells as it did in normally diabetic animals, suggesting that communication between AMPK and mTORC1 is crucial for metformin to work.

By looking at genes regulated in the liver, the researchers found that when AMPK couldnt communicate with Raptor or TSC2, metformins effect on hundreds of genes was blocked. Some of these genes were related to lipid metabolism, helping explain some of metformins beneficial effects. But surprisingly, many others were linked to inflammation.

Metformin, the genetic data showed, normally turned on anti-inflammatory pathways and these effects required AMPK, TSC2 and Raptor.

We didnt go looking for a role in inflammation, so for it to come up so strongly was surprising, says Salk postdoctoral fellow and first author Jeanine Van Nostrand, PhD.

People suffering from obesity and diabetes often exhibit chronic inflammation, which further leads to additional weight gain and other maladies including heart disease and stroke. Therefore, identifying an important role for metformin and the interrelationship between AMPK and mTORC1 in control of both blood glucose and inflammation reveals how metformin can treat metabolic diseases by multiple means.

Metformin and exercise elicit similar beneficial outcomes, and research has previously shown that AMPK helps mediate some of the positive effects of exercise on the body, so among other questions, Shaw and Van Nostrand are interested in exploring whether Raptor and TSC2 are involved in the many beneficial effects of exercise, as well.

If turning on AMPK and shutting off mTORC1 are responsible for some of the systemic benefits of exercise, that means we might be able to better mimic this with new therapeutics designed to mimic some of those effects, says Shaw, who holds the William R. Brody Chair.

In the meantime, the new data suggest that researchers should study the potential use of metformin in inflammatory diseases, particular those involving liver inflammation. The findings also point toward AMPK, Raptor and TSC2 more broadly as potential targets in inflammatory conditions, suggesting the need for a deeper investigation of metformin, as well as newer AMPK agonists and mTOR inhibitors, the researchers say.

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Inflammation in the Liver Reversed by Metformin - Genetic Engineering & Biotechnology News

Global Biologic Therapeutics Market 2020-2024: COVID-19 Business Continuity Plan | Evolving Opportunities With AbbVie Inc. and AstraZeneca Plc |…

LONDON--(BUSINESS WIRE)--The global biologic therapeutics market size is expected to grow by USD 190.94 billion as per Technavio. This marks a significant market growth compared to the 2019 growth estimates due to the impact of the COVID-19 pandemic in the first half of 2020. Moreover, steady growth is expected to continue throughout the forecast period, and the market is expected to grow at a CAGR of 12%. Request Free Sample Report on COVID-19 Impacts

Read the 120-page report with TOC on "Biologic Therapeutics Market Analysis Report by Product (Antibody therapeutics, Vaccines, Cell therapy, Gene therapy, and Other therapies) and Geography (North America, Europe, Asia, and ROW), and the Segment Forecasts, 2020-2024".

https://www.technavio.com/report/biologic-therapeutics-market-industry-analysis

The market is driven by the strong R&D pipeline. In addition, the introduction of biosimilars is anticipated to boost the growth of the Biologic Therapeutics Market.

The efficiency of biologics in the treatment of severe infections, malignancies, and immunological and hormonal disorders is encouraging manufacturers to invest in R&D for the development of biologics. Monoclonal antibodies constitute one of the fastest growing segments amongst biological therapies. To date, 88 monoclonal antibodies have been approved for different indications. The pipeline of vendors such as AbbVie, Amgen, Johnson & Johnson, and F. Hoffmann-La Roche clearly indicates huge investments in R&D. Apart from manufacturers that are actively involved in research on biologics, many research institutes are also engaged in developing novel biologics through industrial collaborations. Thus, the strong R&D pipeline is expected to drive the biologic therapeutics market growth during the forecast period.

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Major Five Biologic Therapeutics Companies:

AbbVie Inc.

AbbVie Inc. operates in only one business segment that focuses on the discovery, development, manufacture, and commercialization of drugs for the treatment of various indications. The company's key offerings in the market include HUMIRA, CREON, and SURVANTA.

AstraZeneca Plc

AstraZeneca Plc generates revenue by developing and manufacturing pharmaceutical products. The company offers IMFINZI, which is a human immunoglobulin G1 kappa monoclonal antibody that blocks the interaction of PD-L 1 with the PD-1 and CD80 molecules.

Bristol-Myers Squibb Co.

Bristol-Myers Squibb Co. operates in only one segment. This segment is responsible for the discovery, licensing, manufacture, marketing, distribution, and sales of medicines required to cure serious diseases. The company's key offerings include OPDIVO and YERVOY.

F. Hoffmann-La Roche Ltd.

F. Hoffmann-La Roche Ltd. has business operations under two segments: pharmaceuticals and diagnostics. The company offers atezolizumab, a monoclonal antibody, sold under the brand name TECENTRIQ for treating various cancers.

GlaxoSmithKline Plc

GlaxoSmithKline Plc operates its business through three segments: pharmaceuticals, consumer healthcare, and vaccines. The company's key offerings include NUCALA, BENLYSTA, and BEXXAR.

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Global Biologic Therapeutics Market 2020-2024: COVID-19 Business Continuity Plan | Evolving Opportunities With AbbVie Inc. and AstraZeneca Plc |...

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