Archive for the ‘Gene Therapy Research’ Category

Gene Therapy R&D and Revenue Forecasts 2020-2030

Retroviruses, Lentiviruses, Adenoviruses, Adeno Associated Virus, Herpes Simplex Virus, Poxvirus, Vaccinia Virus, Naked/Plasmid Vectors, Gene Gun, Electroporation, Lipofection, Cancer, Rare Diseases, Cardiovascular Disorders, Ophthalmologic Conditions, Infectious Disease, Neurological Disorders, Diabetes Mellitus

LONDON, April 17, 2020 /PRNewswire/ -- The gene therapy market is projected to grow at a CAGR of 32% in the first half of the forecast period. In 2019, the cancer treatment submarket accounted for 55.8% of the gene therapy drug market. Visiongain estimated that gene therapy for rare diseases will be the driver for market growth in the first half of the forecast period.

How this report will benefit youRead on to discover how you can exploit the future business opportunities emerging in this sector.

In this brand-new 215-page report you will receive 157 charts all unavailable elsewhere.

The 215-page Visiongain report provides clear detailed insight into the gene therapy market. Discover the key drivers and challenges affecting the market.

By ordering and reading our brand-new report today you stay better informed and ready to act.

To request sample pages from this report please contact Sara Peerun at sara.peerun@visiongain.com or refer to our website: https://www.visiongain.com/report/gene-therapy-rd-and-revenue-forecasts-2020-2030/#download_sampe_div

Report Scope

Gene Therapy market forecasts from2020-2030

This report assesses the approved gene therapy products in the market and gives revenue to 2030

Provides qualitative analysis and forecast of the submarket by indication for the period 2020-2030: Cancer Cardiovascular disorders Rare diseases Ophthalmological diseases Infectious Diseases Neurological Disorders Diabetes Mellitus Other therapeutic uses

Profiles leading companies that will be important in the development of the gene therapy market. For each company, developments and outlooks are discussed and companies covered in this chapter include: UniQure Biogen Bluebird Bio Spark Therapeutics Applied Genetics Technologies Corporation Oxford Biomedica GenSight Biologics & Other Companies

Assesses the outlook for the leading gene treatment R&D pipeline for 2019 and discusses technological progress and potential. Profiles appear for gene therapy drug candidates, with revenue forecasts for four leading agents: Collategene (AMG0001, AnGes MG/Vical) BC-819 (BioCancell) BC-821 BioCancell SPK-CHM Spark Therapeutics SPK-FIX Spark Therapeutics/Pfizer SPK-TPP1- Spark Therapeutics Lenti-D (Bluebird Bio) LentiGlobin (Bluebird Bio) VM202-DPN ViroMed

Provides qualitative analysis of trends that will affect the gene therapies market, from the perspective of pharmaceutical companies, during the period 2020 to 2030. SWOT analysis is provided and an overview of regulation of the gene therapy market by leading region given.

Our study discusses factors that influence the market including these: Translation of research into marketable products modifying human DNA gene transfer for therapeutic use, altering the nuclear genome Genomic editing technology and other supporting components Collaborations to develop and launch gene-based products acquisitions and licensing deals Supporting technologies for human genetic modification, gene replacement and targeted drug delivery Gene therapies for ophthalmologic diseases next-generation medicines Regulations in the United States, the European Union and Japan overcoming technological and medical challenges to pass clinical trials.

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Did you know that we also offer a report add-on service? Email sara.peerun@visiongain.comto discuss any customized research needs you may have.

Companies covered in the report include:

4DMT (4D Molecular Therapeutics)AbeonaAGTC (Applied Genetics Technologies Corporation)AMT (Amsterdam Molecular Therapeutics) AnGes MGAsklepios BioPharmaAstraZenecaAudentes TherapeuticsAvalanche BiotechBayer HealthcareBeijing Northland Biotech CoBenda PharmaceuticalBenitec BiopharmaBioCancellBiogenBiogen IdecBluebird BioBMS (Bristol-Myers Squibb)Broad Institute/Whitehead InstituteCelgeneCell Therapy CatapultCellectisChiesi Farmaceutici Clearside BiomedicalConvergence PharmaceuticalsDaiichi Sankyo Dimension TherapeuticsEditas MedicineFondazione TelethonFrancis Crick Institute Genable Technologies LtdGenethonGenSight BiologicsGenVecGoogleGSK (GlaxoSmithKline)Henry Ford Health SystemHSCI (Human Stem Cells Institute)HSR-TIGET (San Raffaele Telethon Institute for Gene Therapy), ImaginAbImmune Design Corp InoCardInovioIntellia TherapeuticsInvetechKite PharmaKolon GroupKolon Life ScienceLysogeneMitsubishi Tanabe Pharma Corporation NeuralgeneNightstaRxNorthwestern Memorial HospitalNovartisOXB (Oxford Biomedica)PfizerPNP TherapeuticsPrecision Genome Engineering Inc aka PregenenProNaiProtek GroupRaffaele HospitalREGENX BiosciencesRenova TherapeuticsRocheRoszdravnadzorSangamo BiosciencesSanofiSarepta TherapeuticsShanghai Sunway BiotechShenzhen SiBiono GeneTechSotex Pharm Firm Spark TherapeuticsSynerGene TherapeuticsTakara BioTAP BiosystemsThermo Fisher ScientificTissueGeneToolGenUC BerkeleyUC San Francisco uniQureUS Business Innovation Network Vertex PharmaceuticalsVical IncorporatedViroMedVM BiopharmaVoyage Therapeutics

List of Organisation Mentioned ASCO (American Society of Clinical Oncology)ASI (Agency for Strategic Initiatives) CAT (Committee for Advanced Therapies) CBER (Center for Biologics Evaluation and Research)CHMP (Committee for Medicinal Products for Human Use)CHOP (The Children's Hospital of Philadelphia)DCGI (Drugs Controller General of India)DHHS (Department of Health and Human Services)EMA (European Medicines Agency)FDA (US Food and Drug Administration)INSERM (Institut National de la Sant et de la Recherche Mdicale) IRB (Institutional Review Boards) MFDS (Korean Ministry of Food and Drug Safety) MHLW (Ministry of Health, Labour, and Welfare)MHRA (Medicines and Healthcare Products Regulatory Agency)Ministry of Health Commission NHS (National Health Service)NICE (the National Institute for Health and Care Excellence)NIH (National Institutes of Health) OHRP (Office for Human Research Protections)PMDA (Pharmaceuticals and Medical Devices Agency) RCGM (Review Committee of Genetic Manipulation) Russian Ministry of Healthcare and Social DevelopmentSFDA (State Food and Drug Administration of China) SMC (Scottish Medicines Consortium) The Fund for Promotion of Small Innovative Enterprises in Science and TechnologyThe IGI (Innovative Genomics Initiative)The Innovative Genomics Initiative The Walter and Eliza Hall Institute The Wellcome Trust Sanger Institute WFH (World Federation of Hemophilia)WHO (World Health Organization)

To see a report overview please e-mail Sara Peerun on sara.peerun@visiongain.com

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SOURCE Visiongain

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Visiongain Report: The Gene Therapy Market is Projected to Grow at a CAGR of 32% in the First Half of the Forecast Period - Yahoo Finance

How do we learn when to cooperate, and when to cheat? It likely depends in part on the social interactions we observe growing up. Turns out that the same is true for cleaner fish (Labroides dimidiatus), according to a recent study.

Cleaner fish, also known as bluestreak cleaner wrasse, feed on ectoparasites found on other fishes their clients. What cleaner fish really like to eat, however, is the protective mucus that covers their clients bodies. While eating ectoparasites is great for client fishes, cheating by eating mucus is bad. Client fishes will punish cheaters by fleeing, or by chasing the cheaters around.

Photo by David Clode on Unsplash

Whats the best option, to cheat and eat a brief but delicious snack (and risk being chased around), or to cooperate and eat a larger, peaceful, but not as tasty meal? A group of researchers led by Noa Truskanov, from the University of Neuchtel, set out to understand how juvenile cleaner fish learn to answer that question.

In a series of experiments recently published in Nature Communications, Truskanov and her collaborators allowed juvenile cleaner fish to observe adults interacting with model clients. They then placed these juveniles in the same situations they had just observed, and tested if they copied the adult fish, learned from the adult fish, or behaved independently of what they had just seen.

Their results show that juvenile fish are actively learning to cooperate by observing the interactions of adult fish with their clients. Juvenile fish are more cooperative when client fishes are intolerant of cheating. When given the choice, however, they prefer clients who allow them to cheat a little. Juvenile fish also didnt just imitate random adult preferences, showing that there are not copying, but rather learning.

Although both social learning and cooperation are widespread in nature, examples of animals that learn socially how to cooperate are extremely scarce. This leads us, humans, to erroneously assume that we are the only ones who learn to cooperate by observing others. Cleaner fish are putting us right back in our place: they can do it, too.

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Your socially isolated brain craves interaction with other people just as much as it craves a meal when you are hungry - Massive Science

Analysis Report on Gene Therapy Market

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Some key points of Gene Therapy Market research report:

Strategic Developments: The custom analysis gives the key strategic developments of the market, comprising R&D, new product launch, growth rate, collaborations, partnerships, joint ventures, and regional growth of the leading competitors operating in the market on a global and regional scale.

Market Features: The report comprises market features, capacity, capacity utilization rate, revenue, price, gross, production, production rate, consumption, import, export, supply, demand, cost, market share, CAGR, and gross margin. In addition, the report offers a comprehensive study of the market dynamics and their latest trends, along with market segments and sub-segments.

Analytical Tools: The Global Gene Therapy Market report includes the accurately studied and assessed data of the key industry players and their scope in the market by means of a number of analytical tools. The analytical tools such as Porters five forces analysis, feasibility study, and many other market research tools have been used to analyze the growth of the key players operating in the market.

COVID-19 Impact on Gene Therapy Market

Adapting to the recent novel COVID-19 pandemic, the impact of the COVID-19 pandemic on the global Gene Therapy market is included in the present report. The influence of the novel coronavirus pandemic on the growth of the Gene Therapy market is analyzed and depicted in the report.

The global Gene Therapy market segment by manufacturers include

segmented as follows:

Global Gene Therapy Market, by Product

Global Gene Therapy Market, by Application

Global Gene Therapy Market, by Region

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Important queries addressed in the report:

Moreover, the report highlighted revenue, sales, manufacturing cost, and product and the States that are most competitive in the lucrative market share idea. There is a discussion on the background and financial trouble in the global Gene Therapy economic market. This included the CAGR value during the outlook period leading to 2025.

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The Economic Impact of Coronavirus on Gene Therapy Market Analysis and Value Forecast Snapshot by End-use Industry 2019-2022 - Jewish Life News

U.S. Sectors: Equities NDR Ned Davis Research April 17: Retailing is early-cycle because it is most often the consumer who leads us into a recession and the consumer who leads us out. We think that will be the case this recession, as well. As soon as shelter-in-place restrictions end, we expect consumer spending to pick up or at least become less bad. Improved spending should be the start of the emergence from recession....

If you went overweight S&P Retailing at the start of every recession and closed out the trade at the end of every recession, you would have an average annualized relative return of 12.3%. Since 1954, Retailing has only underperformed during the 1960-1961 recession. Retailing had some of its best relative performance during the two recessions with the biggest declines in payrolls (1957, 2007)...

Even though Amazon.com [ticker: AMZN] is the primary reason S&P 500 Retailing has outperformed by 1,800 basis points, year-to-date, we are encouraged by Retailing sub-industry performance. Only two of nine sub-industries, Department Stores and Computer & Electronics Retail, have made lower relative-strength lows since March 23. Since April 2, equal-weighted S&P 500 Retailing has outperformed the equal-weighted S&P 500 by over 800 basis points.

--Pat Tschosik

Equity Research RBC Capital Markets April 17: Funding continues to pour into biotech, with the first quarter of 2020 just setting a fresh record for biopharma venture funding in the U.S., highlighting an unprecedented level of innovation and expectations for sustained value creation. Though we could see some reprioritization among early-stage investments with the crisis, with modalities such as cell and gene therapy, antisense oligonucleotides, and RNA inter-ference technologies breaking full steam into the commercial spotlight, we do not foresee any major changes to the innovative environment.

We are witnessing an unprecedented level of cooperation and speed in this pandemic from basic science to even manufacturing as 70+ biotechs rapidly shift focus toward the common goal of developing a treatment and vaccine. Again and again, we have heard from management and doctors the unprecedented level of communication and access available at all levels. Partnerships are being created at record speeds to immediately join the huntsometimes even with deal-term negotiations ongoing. It took our community roughly three months from viral genetic-sequence selection to the first human study for vaccine developmenta record milestone nearly 17 months ahead of the vaccine-development timeline for SARS (2003) and, most recently, several weeks ahead of Zika virus (2016) development.

--Brian Abrahams, Gregory Renza, Gilbert Kinsey, Yinglu Zhang

BTIG Quick View BTIG April 16: The economic aspect of the virus is centered on Main Streetsmall businesses and their employees devastated by the sudden shutdown, in contrast to 2007-09s Great Financial Crisis, driven by imbalances from Wall Street and the broader financial industry. Nowhere is this more apparent than in measuring the relationship of the small-cap Russell 2000 volatility index, RVX, to the S&P 500s VIX; the spread closed today at 13.82, RVX over VIX, an all-time high in small-cap relative riskiness. Yet when this spread reaches the top decile (>8.25), stocks tend already to be in a distressed state, and while the near-term forward return profile is mixed, stocks are higher 12 months forward 91% of the time, with Russell 2000 outperformance the rule. These findings are consistent with our work, which shows that the bear-market laggards become the new bull-market leaders.

--Julian Emanuel, Michael Chu

Cyclical Outlook Pimco April 15: The economic loss [in China] is unprecedented: In nine weeks of lockdown, industrial production and services contracted about 10 to 15 percentage points from their pre-pandemic levels, and the unemployment rate could rise to 8%-9% from 5%. The first quarter of 2020 will mark the recession trough in China, in our view, with gross domestic product estimated to fall 10 percentage points below its pre-Covid level. [China reported Friday that first-quarter GDP contracted by 6.8%.] For comparison, even during the global financial crisis, Chinas GDP didnt contract.

Fiscal stimulus to date has increased to 16% of GDP, consisting of a 5% budget deficit, 6% in special government-bond financing investments, and the rest in liquidity/credit supports. Although the fiscal impulse could add four to five percentage points to GDP, the multiplier effects are weak for both corporate and household spending. We expect the Peoples Bank of China to cut policy rates by 30 to 50 basis points and keep liquidity ample, while efforts to maintain stability in the yuan will be continued.

Economic activity will likely follow a U shape over the course of 2020. The global recession will hit exports primarily in the second and third quarters of 2020, domestic demand is still hampered by quarantine curbs, and stimulus transmission is weak amid spreading bankruptcies and job losses. We forecast GDP growth in 2020 will be in a broad -4% to +2% range, the first severe economic recession in China since 1976. The economy likely will not recover to the fourth-quarter 2019 level until the first quarter of 2021.

--Isaac Meng, Stephen Chang

Rates Strategy MUFG Securities April 10: We remain deeply concerned over risks our models glean for a double-dip economic recession, beginning over the time period from 2H-2022 through year-end 2023. Our models forecast for a double-dip U.S. recession remains a complete outlier in comparison to the market consensus estimates, Blue Chip surveys, and official central banks and governmental projections.

Notwithstanding such a controversial intermediate-term outlook, should our models macro-economic forecasts for the years 2020 through 2023 prove accurate, then our interest-rate models spy risk that official monetary policy in the U.S. (and likely elsewhere) remains remarkably accommodative:

The federal funds policy target rate likely may remain at ZIRP [zero interest-rate policy] from 2020 through 2023.

The Federal Reserves Q.E. Treasury purchase program likely may be expanded by an additional $1.50 trillion over the near-term, followed by another expansion of near $2.00 trillion over the intermediate-term.

The Federal Reserves other non-conventional policies likely may be extended and expanded, as well, over the near and intermediate terms.

In turn, it would not surprise us that the shape, slope and level of the U.S. Treasury yield curve might resemble that of Japanese government bonds from a few years ago. For example: a 2-year Treasury Note yield of near 0.075%; a 5-year Treasury Note yield of near 0.25%, a 10-year Treasury Note yield of near 0.54%, and a 30-year Treasury Bond yield of near 1.77%.

--John D. Herrmann

To be considered for this section, material, with the author's name and address, should be sent to MarketWatch@barrons.com.

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Retail Stocks Are Outperforming. Amazon.com Isnt the Only Reason Why. - Barron's

Dublin, April 17, 2020 (GLOBE NEWSWIRE) -- The "Nucleic Acid Based Gene Therapy Market Global Report 2020-30" report has been added to ResearchAndMarkets.com's offering.

The global nucleic acid based gene therapy market was worth $557.47 million in 2019. It is expected to grow at a compound annual growth rate (CAGR) of 10.00% and reach $816.18 million by 2023.

The nucleic acid-based gene therapy market covered in this report is segmented by technology into anti-sence and anti-gene, short inhibitory sequences, gene transfer therapy, nucleoside analogs, ribozymes, aptamers, others. It is also segmented by application into oncology, muscular dystrophy/ muscular disorders, rare diseases.

Nucleic Acid Based Gene Therapy Market Market Global Report 2020-30 from the author provides the strategists, marketers and senior management with the critical information they need to assess the global nucleic acid based gene therapy market market.

Reasons to Purchase

Where is the largest and fastest growing market for the nucleic acid based gene therapy market? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? The Nucleic Acid Based Gene Therapy Market market global report from the author answers all these questions and many more.

The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market's historic and forecast market growth by geography. It places the market within the context of the wider nucleic acid based gene therapy market market, and compares it with other markets.

The nucleic acid-based gene therapy market consists of sales of nucleic acid-based gene therapy products and services. Gene therapy is used for correcting defective genes that are responsible for disease development. Moreover, nucleic acid-based therapeutics are used to treat genetic disorders and diseases for which there exists no permanent cure such as anaemia, sickle cell, cystic fibrosis, diabetes, and thalassemia.

Rising financial support by the government and the companies is projected to drive the demand for nucleic acid based gene therapy. Nucleic acid therapeutics are analogs of naturally occurring acids or proteins responsible for genetic expression. The traditional therapies do not have any cure for the treatment of diseases such as cystic fibrosis, hemophilia, sickle cell anaemia, thalassemia, and diabetes. Genetic profiling and molecular target identification form the backbone of these classes of drugs. Nucleic acid medication has greater potential for the treatment of these diseases, as they target the genetic basis of diseases and have a permanent cure. Rising financial support by the government and the companies dealing in the market for gene therapy is expected to contribute to increasing the demand for nucleic acid-based gene therapies. For instance in October 2019, the USA National Institutes of Health (NIH) announced plans to invest $100 million for the next four years to cure HIV and sickle cell disease with gene therapies. The government support for gene therapies will contribute to the growth of the market.

Stringent regulations imposed on gene therapies raises the price of gene therapies, which in turn hinders the demand for nucleic acid-based gene therapies. The excessive regulatory oversights create an expensive and elongated route for approval increasing the expenses. According to Foundation for Economic Education (FEE), unlike other drugs approved or regulated by the Food and Drug Administration (FDA), gene therapies are not only subject to the regulatory structure of FDA, but also the Recombinant DNA Advisory Committee and Office of Biotechnology Activities. Also, as estimated by FEE, an approved gene therapy drug cost nearly $5.0 billion, which is five times higher than that of the average cost of FDA approval. The high cost of gene therapeutics drugs places them beyond the financial reach of the populace. This scenario is anticipated to restrain the market growth of the nucleic acid-based gene therapy market.

In May 2018, Vectalys, a leading biotechnology company specializing in the manufacturing of high-quality solutions for gene delivery and FlashCell, a company engaged in developing non-integrated lentiviral delivered RNA Therapeutics announced the merger to create Flash Therapeutics, a privately held gene therapy company developing cell and gene therapeutics. The new company Flash Therapeutics is expected to focus on the development of RNA therapeutics based on LentiFlash, a non-integrative lentiviral delivery technology for incurable diseases.

Major players in the market are Wave Life Sciences, Imugene, Caperna, Phylogica, Protagonist Therapeutics, Benitec Biopharma, EGEN, BioMedica, Transgene and Copernicus Therapeutics.

Key Topics Covered:

1. Executive Summary

2. Nucleic Acid Based Gene Therapy Market Market Characteristics

3. Nucleic Acid Based Gene Therapy Market Market Size And Growth 3.1. Global Nucleic Acid Based Gene Therapy Market Historic Market, 2015 - 2019, $ Billion 3.1.1. Drivers Of The Market 3.1.2. Restraints On The Market 3.2. Global Nucleic Acid Based Gene Therapy Market Forecast Market, 2019 - 2023F, 2025F, 2030F, $ Billion 3.2.1. Drivers Of The Market 3.2.2. Restraints On the Market

4. Nucleic Acid Based Gene Therapy Market Market Segmentation 4.1. Global Nucleic Acid Based Gene Therapy Market Market, Segmentation By Technology, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion4.2. Global Nucleic Acid Based Gene Therapy Market Market, Segmentation By Application, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

5. Nucleic Acid Based Gene Therapy Market Market Regional And Country Analysis 5.1. Global Nucleic Acid Based Gene Therapy Market Market, Split By Region, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion 5.2. Global Nucleic Acid Based Gene Therapy Market Market, Split By Country, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

6. Asia-Pacific Nucleic Acid Based Gene Therapy Market Market

7. China Nucleic Acid Based Gene Therapy Market Market

8. India Nucleic Acid Based Gene Therapy Market Market

9. Japan Nucleic Acid Based Gene Therapy Market Market

10. Australia Nucleic Acid Based Gene Therapy Market Market

11. Indonesia Nucleic Acid Based Gene Therapy Market Market

12. South Korea Nucleic Acid Based Gene Therapy Market Market

13. Western Europe Nucleic Acid Based Gene Therapy Market Market

14. UK Nucleic Acid Based Gene Therapy Market Market

15. Germany Nucleic Acid Based Gene Therapy Market Market

16. France Nucleic Acid Based Gene Therapy Market Market

17. Eastern Europe Nucleic Acid Based Gene Therapy Market Market

18. Russia Nucleic Acid Based Gene Therapy Market Market

19. North America Nucleic Acid Based Gene Therapy Market Market

20. USA Nucleic Acid Based Gene Therapy Market Market

21. South America Nucleic Acid Based Gene Therapy Market Market

22. Brazil Nucleic Acid Based Gene Therapy Market Market

23. Middle East Nucleic Acid Based Gene Therapy Market Market

24. Africa Nucleic Acid Based Gene Therapy Market Market25. Nucleic Acid Based Gene Therapy Market Market Competitive Landscape And Company Profiles 25.1. Nucleic Acid Based Gene Therapy Market Market Competitive Landscape 25.2. Nucleic Acid Based Gene Therapy Market Market Company Profiles

26. Key Mergers And Acquisitions In The Nucleic Acid Based Gene Therapy Market Market

27. Nucleic Acid Based Gene Therapy Market Market Trends And Strategies

28. Nucleic Acid Based Gene Therapy Market Market Future Outlook and Potential Analysis

29. Appendix

Companies Mentioned

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Global Nucleic Acid Based Gene Therapy Industry to 2030 - Featuring Celsion Corporation, Wave Life Sciences & Imugene Among Others - Yahoo Finance...

NEW YORK and BASEL, Switzerland, April 15, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd., a clinical-stage company developing innovative gene therapies for neurological diseases, today announced its collaboration with Invitae, a leading medical genetics company, in the Detect Lysosomal Storage Diseases (Detect) program to facilitate faster diagnoses for children with lysosomal storage disorders (LSDs), including GM1 gangliosidosis and GM2 gangliosidosis, also known as Tay-Sachs/Sandhoff disease. Invitae offers genetic testing and counseling at no charge to patients suspected of having an LSD.

Axovant is committed to developing novel gene therapies for those living with rapidly progressive neurodegenerative diseases. We are hopeful that our collaboration with Invitae will provide families with easier access to genetic testing and bring us one step closer to identifying patients who may benefit from potential therapies, said Parag Meswani, PharmD., Axovants SVP of Commercial Strategy & Operations. Our AXO-AAV-GM1 clinical program targeting GM1 gangliosidosis is currently enrolling at the National Institutes of Health, and we are seeking IND clearance for the AXO-AAV-GM2 clinical trial targeting Tay-Sachs and Sandhoff diseases. Early intervention is ideal with potentially disease-modifying genetic therapies, and our diagnostics partnership with Invitae should allow us to identify and enroll children at even earlier stages of disease progression.

LSDs are progressive, multi-system, inherited metabolic diseases associated with premature death, and genetic testing is a crucial first step to arriving at a diagnosis. LSDs are misdiagnosed or undiagnosed in the majority of patients. The Detect program includes a specific LSD testing panel of 53 genes designed to provide patients and families accurate information quickly to preserve valuable treatment time.

Genetic testing can expedite an accurate diagnosis, facilitate earlier interventions, allow genetic counseling of family members, and support clinical research for LSDs such as GM1 and GM2 gangliosidosis, said Robert Nussbaum, M.D., chief medical officer of Invitae. Were pleased Axovant has joined the Detect program to help offer no-charge, sponsored genetic testing for those patients suspected of having the disease.

Research has shown no-charge testing programs with large well-designed panels help increase utilization of genetic testing, which can shorten the time to diagnosis by as much as 2 years in some conditions. Accurate diagnoses enable clinicians to focus on providing disease-specific care sooner, helping reduce costs and improve outcomes.

Additional details, as well as terms and conditions of the program, can be found at https://www.invitae.com/en/detectLSDs/.

About Axovant Gene Therapies

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

About Invitae

Invitae Corporation (NYSE: NVTA) is a leading medical genetics company, whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website atinvitae.com.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "might," "will," "would," "should," "expect," "believe," "estimate," and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding costs associated with its operating activities are forward-looking. All forward-looking statements are based on estimates and assumptions by Axovants management that, although Axovant believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Axovant expected. Such risks and uncertainties include, among others, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the expectations for regulatory submissions and approvals; the continued development of its gene therapy product candidates and platforms; Axovants scientific approach and general development progress; and the availability or commercial potential of Axovants product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Axovants most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on February 10, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Axovant undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Media Contact:

Parag MeswaniAxovant Gene Therapies(212) 547-2523investors@axovant.commedia@axovant.com

Excerpt from:
Axovant Announces Partnership with Invitae to Increase Access to Genetic Testing and Accelerate Diagnoses of GM1 and GM2 Gangliosidosis -...

IRVINE, Calif., April 17, 2020 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. (Nasdaq: CLPT) is pleased to announce that Matthew B. Klein, MD, MS, FACS, has been appointed to ClearPoint Neuros Board of Directors effective immediately. Dr. Klein, Chief Development Officer of PTC Therapeutics, Inc. (PTC), is a veteran biotechnology company executive with extensive experience in drug discovery and development, and a board-certified surgeon. He succeeds Marcio Souza, who joined ClearPoint Neuros Board as PTCs representative in connection with PTCs May 2019 equity investment in ClearPoint Neuro. Mr. Souza, who resigned from his position as PTCs Chief Operating Officer, as was announced by PTC on March 16, 2020, will remain on ClearPoint Neuros Board as an independent member.

Prior to joining PTC, Dr. Klein served in several executive positions with BioElectron Technology Corporation (BioElectron), most recently as BioElectrons Chief Executive Officer and a Director prior to its acquisition by PTC in 2019. Dr. Klein has a BA from the University of Pennsylvania, an MD from Yale University School of Medicine and an MS in epidemiology from the University of Washington School of Public Health.

We are thrilled by the addition of Matt to our board and the contributions he will make to our company and culture, commented Joe Burnett, President and CEO. As a patient-centric company, adding an established physician, scientist and leader to our board will improve our ability to evaluate new technologies and partnerships.

I am very excited to be joining ClearPoint Neuros Board and look forward to bringing my experience as a drug developer and a surgeon to help the company continue its growth and to fulfill its incredibly important mission, said Dr. Klein.

About ClearPoint Neuro

ClearPoint Neuros mission is to improve and restore quality of life to patients and their families by enabling therapies for the most complex neurological disorders with pinpoint accuracy. Applications of the Companys current product portfolio include deep-brain stimulation, laser ablation, biopsy, neuro-aspiration, and delivery of drugs, biologics and gene therapy to the brain. The ClearPoint Neuro Navigation System has FDA clearance, is CE-marked, and is installed in 60 active clinical sites in the United States. The Companys SmartFlow cannula is being used in partnership or evaluation with more than 20 individual biologics and drug delivery companies in various stages from preclinical research to late stage regulatory trials. To date, more than 3,500 cases have been performed and supported by the Companys field-based clinical specialist team which offers support and services for our partners. For more information, please visit http://www.clearpointneuro.com.

Forward-Looking Statements

Statements herein concerning the Companys plans, growth and strategies may include forward-looking statements within the context of the federal securities laws. Statements regarding the Company's future events, developments and future performance, as well as management's expectations, beliefs, plans, estimates or projections relating to the future, are forward-looking statements within the meaning of these laws. Uncertainties and risks may cause the Company's actual results to differ materially from those expressed in or implied by forward-looking statements. Particular uncertainties and risks include those relating to: future revenues from sales of the Companys ClearPoint Neuro Navigation System products; the Companys ability to market, commercialize and achieve broader market acceptance for the Companys ClearPoint Neuro Navigation System products; and estimates regarding the sufficiency of the Companys cash resources. More detailed information on these and additional factors that could affect the Companys actual results are described in the Risk Factors section of the Companys Annual Report on Form 10-K for the year ended December 31, 2019, which has been filed with the Securities and Exchange Commission.

Contact:Harold A. Hurwitz, Chief Financial Officer(949) 900-6833hhurwitz@clearpointneuro.com

Jacqueline KellerVice President, Marketing(949) 900-6833jkeller@clearpointneuro.com

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Biotechnology Leader and Surgeon Dr. Matthew Klein Joins ClearPoint Neuro Board of Directors - BioSpace

The Gene Therapy Products Market report offers a complete and intelligent analysis of the competition, segmentation, dynamics, and geographical advancement of the global Gene Therapy Products market. The market report is sure to lend a hand in enhancing sales and improving return on investment (ROI). This market research report provides clients with the supreme level of market data and information which is specific to their niche and their business requirements. This global Gene Therapy Products market research report is a proven source to gain valuable market insights and take better decisions about the important business strategies.

Get ExclusiveSample Copy of This Report Herehttps://www.databridgemarketresearch.com/request-a-sample?dbmr=global-gene-therapy-products-market

Global gene therapy products market is set to witness a substantial CAGR in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. Rising cancer cases and unused potential for emerging markets are the major factors for the growth of this market.

Few of the major competitors currently working in the globalgene therapy products marketareAdaptimmune., Anchiano Therapeutics, bluebird bio, Inc., CELGENE CORPORATION, GlaxoSmithKline plc., Merck KGaA, Novartis AG, Achieve Life Sciences, Inc., Spark Therapeutics, Inc., Abeona Therapeutics, Inc, Adverum, agtc, Arbutus Biopharma, Audentes Therapeutics, AveXis, Inc., CRISPR Therapeutics, Intellia Therapeutics, Inc and Gilead Sciences,Inc. among others.

Market Definition:Global Gene Therapy Products Market

Gene therapy or human gene therapy is a process which is used to modify gene for the treatment of any disease. Plasmid DNA, bacterial vector, human gene editing technology and viral vectors are some of the most common type of gene therapy products. The main aim of the gene therapy is to replace the dysfunctional genes. Somatic and germline are some of the most common type of the gene therapy.

Complete report on Global Gene Therapy Product Market Research Report 2019-2026 spread across 350 Pages, profiling Top companies and supports with tables and figures

Segmentation: Global Gene Therapy Products Market

Gene Therapy Products Market : By Product

Gene Therapy Products Market : By Application

Gene Therapy Products Market : ByGeography

Read Complete Details with TOC Herehttps://www.databridgemarketresearch.com/toc?dbmr=global-gene-therapy-products-market

Key Developments in the Gene Therapy Products Market:

Gene Therapy Products Market Drivers

Gene Therapy Products Market Restraints

Competitive Analysis: Gene Therapy Products Market

Global gene therapy products market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of gene therapy products market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

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Gene Therapy Products Market (COVID -19 Impact Analysis) : Emerging Trends, Business Opportunities and Segmentation. Major Players are Adverum, agtc,...

The times are strange. We began the year raging into this new decade with an untamable pace, adding quite a bit of stress, we might add. Three months into 2020 and we find ourselves cooped up inside our home, again, with elevated levels of stress. The fact remains, ones mental wellness directly impacts their physical being. As a result, this mental unrest will take a toll on your body, inside and out. While pale skin, outbreaks, and hair fall are some of the first and most visible problems you may face with, the problem goes much deeper.

That said, let this pause act as that much-needed stress buster. Taking care of yourself at this time and investing in your physical well-being will significantly shape how you feel. Dr Simal Soin, founder of AAYNA clinic, says, Under these exceptional circumstances, it is easy to slip into despair and depression because of a lack of routine. Remaining healthy is of topmost priority, and this is the time to develop a self-care regime.

Soin adds, Having a good skin and hair care routine can boost your self-confidence, apart from helping you to productively utilise your time. In fact, this is the ideal time to try all those DIY home remedies that could otherwise never be a part of your routine because of your busy schedule. As these efforts give you refreshing, supple skin, and your precious mane truly becomes healthy and gorgeous, you will naturally feel positive and happy about taking the time out for yourself.

Also Read: 5 Self-Care Practices You Need To Incorporate In 2020

Also Read: Meditation techniques to help reduce stress

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Self-Care In Testing Times: An Expert Guide - Femina

Takeda has a number of early-stage gene therapies in its pipeline, including several for rare diseases. However, the pharma giant is looking to bolster its early-stage assets further with an agreement with Evotec GT.

Evotec GT is housed in Orth an der Donau, Austria, and specializes in the development of gene therapy for hemophilia, hematology, metabolic and muscle diseases.

After its acquisition of Shire, hematology now constitutes a core part of Takedas portfolio, meaning that this collaboration could offer the potential to strengthen its treatment base in the years to come.

Few further details were offered, and no financials were disclosed. Evotec only acknowledged that there would be an upfront payment and additional various payments over time.

What is known is that the long-term partnership will support multiple targets within Takedas four core therapeutic areas, oncology, rare diseases, neuroscience and gastroenterology. With Steven Hitchcock, global head of research for Takeda, suggesting that the rare disease area would likely be the principal focus.

With the Shire takeover complete, Takeda has turned its attention to signing small deals to fill out its early-stage pipeline in complementary areas to the formers portfolio, which saw it sign a deal for a microbiome-based therapeutic for Crohns disease and saw it acquire PvP Biologics to take on the companys therapeutic enzyme for celiac disease.

Read more:
Takeda and Evotec partner on gene therapy - BioPharma-Reporter.com

The COVID-19, pandemic has brought the world to its knees in a way that has not been seen in most peoples lifetimes.

In addition to an enormous impact on human health in many countries, restrictions imposed in an attempt to control the virus and protect vulnerable populations are having far reaching impacts across the global economy.

Biotech and pharma companies in Europe and elsewhere have rushed to support work on developing new diagnostic tests, treatments and vaccines for the virus. But with most non-COVID-19 clinical research at a standstill, laboratories shut and staff working from home, many companies are losing out.

Recruiting patients into new or ongoing clinical trials seems to be an issue not for a few companies, but at least half of the companies I follow, said Marcus Wieprecht, healthcare specialist and managing director of equity research at Mainfirst bank in Frankfurt.

Frontrunners in the race to develop a vaccine include Moderna in the US and BioNTech and CureVac in Germany. Moderna has begun phase I tests in human volunteers, BioNTech plans to start testing this month and CureVac hopes to begin tests in June.

The move into infectious disease vaccines marks a change in direction for Moderna and BioNTech, which initially were applying their messenger RNA technologies in the development of cancer vaccines. CureVac too, has taken an mRNA-based cancer vaccine into clinical development, but it also has positive safety and tolerability data for a rabies vaccine, and an approved manufacturing facility in Tbingen.

If the data from our COVID-19 project is as positive as our rabies data, then certainly there will be an accelerated kind of approval process, said Franz-Werner Haas, acting CEO of CureVac. This is what we have to discuss with the regulatory authorities, but there is a potential that this could be the first one.

You have to follow the same manufacturing steps whether its a rabies vaccine, a COVID vaccine, or an oncologic vaccine, Haas said. The mechanism is always the same.

Who stands to lose?

In the rush to test drugs against COVID-19, clinical trials in other indications are being interrupted by the strains on healthcare systems and the fact that many potential patients are those most likely to suffer severe effects from coronavirus infection.

This will have an impact on the financial health of small biotechs with limited cash runways, which are now facing longer waits for clinical trials to read out.

For example, in March, Swiss biotech Addex Therapeutics announced it is postponing the registration study of its lead programme dipraglurant in the treatment of Parkinsons disease. Similarly, fellow Swiss company Geneuro said the start of a phase II trial of its lead programme, temelimab in multiple sclerosis, due to be held at the Karolinska Instituet, was being deferred.

Delays in starting trials will lead to delays in getting results and this will hit the finances of small companies that rely on delivering data to raise the next tranche of private capital or trigger a milestone payment from a commercialisation partner. Companies have a base level of cash burn, even if they arent running an active clinical trial.

Addex and Geneuro are fortunate not to have started their trials. Many companies with ongoing clinical studies that have already sucked up a large amount of time and money, are now unclear when they will end. Regulators are taking a flexible approach and allowing deviations from trial protocols, for example, allowing patients to have assessments by telephone and for supplies of trial drugs to be delivered to patients homes. They also promise flexibility when the time comes that trials can restart.

One company that is benefitting from this flexibility is French biotech Sensorion, which is currently running a phase II study testing a gene therapy in hearing loss. While it has had to delay the recruitment of new patients, it continues to monitor those already enrolled.

Since some patients enrolled in the study may be unable to complete the follow-up visits as planned in the protocol, follow-up visits are being planned via teleconference or videoconference with the clinical sites, said Nawal Ouzren, CEO of Sensorion.

It is important the regulators work with companies to ensure trials that have been put on hold are not invalidated, said Eric Le Berrigaud, managing partner of equity at the French investment bank Bryan Garnier & Co, which has a focus on healthcare. We really have to make sure that everything can remain statistically meaningful, he said. When theres a few weeks delay, protocols are [drawn up] in such a way that you can adjust for that, but if it's a matter of months, then it could put a lot of trials in jeopardy.

Investor sentiment

Biotech and pharma shares have suffered far less than other sectors from the havoc COVID-19 has wreaked in stock markets around the world, but there have been falls nonetheless. On January 23 as the COVID-19 crisis began to unfold, the share price of the Nasdaq-quoted Austrian biotech Hookipa Pharma was US$13.80. It hit a low of $5.90 on March 16, recovering to $7.98 at the close last Thursday, 9 April.

Theyre doing well, but in the aftermath of COVID-19, in-sync with the overall market, suddenly their share price goes down, with no good reason, said Markus Hosang, General Partner at BioMed Partners venture capital firm in Basel, an early backer of Hookipa.

Hosang said BioMed Partners is continuing to invest in startups, but is taking a more cautious approach. Already at the outset we will have to make sure that enough capital is made available, particularly to young earlier-stage companies.

Andreas Schmidt, CEO of Proteona, a specialist in single cell proteomic analysis, believes this is a time for dynamic rethinking. The Singapore-based company was due to run tests of its technology in Germany, but with many big research centres in the country closed, it needs to do these elsewhere.

We are very international. We have operations in California and Germany and Singapore ... right now, we are hardly affected in terms of operations in Singapore. So we just say, okay, anything we would have done in Germany, we will now do in Singapore.

Government assistance

The sheer scale of the problem has resulted in governments across Europe introducing grants, loans and other assistance for companies in difficulty due to COVID-19. The French government for example, introduced measures that have been taken up by local biotechs including Pixium Vision, which is developing a retinal implant for correcting sight loss.

The European Commission is also providing support for smaller companies and will make 1 billion available from the EU budget over the next few weeks, to activate 8 billion of financing for 100,000 European small and medium enterprises.

The commission has also announced dedicated research and development funding. Over 1,000 start-ups and small and medium-sized companies with ideas for tackling the COVID-19 pandemic have applied for funding in the European Innovation Councils special 164 million call.

Some small life sciences companies have benefitted from 47.5 million in dedicated funding from Horizon 2020, to support development of diagnostic tests, treatments, vaccines and monitoring and other medical systems.

One beneficiary is the Irish company HiberGene, which was awarded 1 million to fast track development of a COVID-19 diagnostic.

Another source of finance is crowdfunding, with equity crowdfunder Aescuvest for example collaborating with other crowdfunding platforms to raise money for biotechs working on combating COVID-19.

Schmidt is on the board of Aescuvest. All the crowd investment platforms are aligning forces to look at raising money very quickly beyond the normal business model, he said. It's a bit of an experiment. I think nobody has tried that before.

Vaccines, and antiviral and antibiotic drugs are not seen as an attractive area for investment, due to long and expensive development timelines and low returns on investment. The current pandemic is so devastating and widespread it could leave more of a long-term legacy than other recent outbreaks, such as Ebola and Zika, which have had less impact in terms of their geographical spread.

Its not clear as yet what this legacy will be, but when the dust settles and the reports are written, one likely outcome will be a greater prioritisation of pandemic preparedness in western countries.

Its like the immune system. If you want to prime with active immunisation, you want to prime your body to be protected if the situation occurs, said Haas. If it doesnt occur, fine, but if it occurs, the body is already alerted and knows what to fight for.

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Balancing the scales in healthcare: winners and losers from COVID-19 - Science Business

Lev Gerlovin, vice president in the Life Sciences Practice at Charles River Associates (CRA), and Pascale Diesel former vice president in the Life Sciences Practice at CRA, discuss how pharma companies are changing their strategies when it comes to cell and gene therapies.

In recent years, drug development has been transitioning from a one pill fits all approach to a focus on more targeted and personalised therapies, including many cell and gene therapies. These innovative treatments often bring the promise of high efficacy and even curative benefit and, in many cases, are options for patients with diseases that previously had limited or no treatments available. With several cell and gene therapies now approved and many more advancing toward late-stage clinical development, patient communities and healthcare systems are rapidly recognising their potential benefits, whilst pharmaceutical companies are considering opportunities including mergers, acquisitions, licensing agreements, and partnerships. The deal-making landscape for cell and gene therapies is already very active and positioned to grow exponentially in the years ahead.

While the pace of deal-making is accelerating, the unique qualities of cell and gene therapies are also requiring dealmakers on both sides to consider some innovative and previously untried strategies designed to optimise returns and reduce risk. They are structuring deals to address many factors, including the lack of commercial benchmarks for these products and limited long-term safety and efficacy data often associated with cell and gene therapies. We conducted an analysis of more than 30 deals executed in the cell and gene therapy sector recently and compared them to deals executed for monoclonal antibodies (mAbs) between 1999-2013. Emerging cell and gene therapies are often considered magic bullets in the treatment of many serious diseases a claim that was applied to many mAbs when they were first introduced to the market about 20 to 25 years ago and the level of interest in deal-making for these therapies is a reflection of the level of optimism companies have regarding their clinical and commercial potential. Findings from our analysis show that the pace of deal-making in cell and gene therapy is faster and occurring much earlier in the drug development process compared to deals seen in the past for breakthrough therapies including mAbs.

When the first mAb was approved in the mid-1980s, deal-making interactions were mostly limited to collaborations and licensing agreements between smaller biotechnology firms. It took more than 20 years for big pharma to become significantly interested in mAbs and start proactively pursuing deals for these drugs. Conversely, deals within the cell and gene therapy sector have shown a much faster uptake, quickly growing in momentum since approval of the first cell and gene therapies in the early 2010s. More than 50 cell and gene therapy-related partnerships and investments were established between 2010-2016, with big pharma involved in many deals from the beginning. In one example, Novartis initiated a collaboration with GenVec in 2010 for clinical development of adeno-based gene therapies a deal worth potentially $213 million (excluding royalties). That same year Novartis also formed a strategic alliance with GlaxoSmithKline and the Telethon Institute of Gene Therapy to pursue additional gene therapy research and development.

Larger pharmaceutical companies such as Novartis seem more eager to embrace cell and gene therapies compared to prior innovative therapies including mAbs, but the types and values of related deals are evolving compared to what has historically been seen. While the largest mAb deals were often characterised by the acquisitions of late-stage and marketed products, which present more robust data and potentially lower risk to acquisition or licensing partners, most major cell and gene therapy acquisitions involve product pipelines, platform technologies, and manufacturing capabilities rather than one single product. Larger pharmaceutical companies are also targeting earlier stage opportunities, with a higher proportion of partnerships involving Phase 1 or even preclinical stage assets. For example, Pfizer established a collaboration deal with Spark Therapeutics for the development and potential commercialisation of a Phase 1/2 gene therapy for the treatment of haemophilia B and also acquired Bamboo Therapeutics based on a promising gene therapy portfolio including one Phase 1 and several preclinical assets for the treatment of rare diseases impacting the nervous system.

As more companies work to establish a presence in this sector, both larger and smaller companies now often prefer innovative licensing and collaborative agreements, whereas the majority of deals executed for mAbs were straightforward mergers or acquisitions. Given that cell and gene therapy development is still in its nascent stages and many stakeholders, including payers, have questions related to their long-term clinical benefit and budget impact, the fact that mergers or acquisitions are deemed less attractive is unsurprising. Innovative partnerships can be a lower risk option for licensors or investors who want to expand their cell and gene therapy portfolios without assuming full financial responsibility. Partnership and licensing deals can also help reduce the risk of possible disruptions in business operations and productivity, which often occur following M&A deals and the subsequent restructuring of company resources and teams.

The opportunity to access complementary business capabilities is another factor that is driving interest in innovative licensing and collaborative agreements. With licensing arrangements, a licensor might be seeking a partner who can offer specialised expertise in a specific indication whereas a licensee might see the benefit of collaborating with a company with technical abilities or assets that align with their own product pipeline. The partnership between Neurocrine Bioscience and Voyager Therapeutics reflects this type of synergistic collaboration, where Neurocrine provides expertise in central nervous system (CNS) drug development as well as financial support for the clinical development of gene therapies for Parkinson's disease and Friedreichs ataxia. In return, Neurocrine gains the opportunity to co-commercialise and potentially obtain global rights to portfolio-compatible therapies.

As new cell and gene therapies emerge and others progress to late-stage clinical development and regulatory approval, most industry stakeholders anticipate that both the structure and value of deals in the sector will mature. Companies will exercise the option to follow up on previous collaborative deals, punctuating the space with potentially massive exclusive licensing agreements and acquisitions.

*The views expressed herein are the authors and not those of Charles River Associates (CRA) or any of the organisations with which the authors are affiliated.The authors wish to acknowledge the contributions ofSil Collins and Alex Davidto this article.

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How pharma companies are shifting deal strategies for cell and gene therapies - EPM Magazine

Acute Lymphoblastic Leukemia (ALL) is the most common childhood cancer. While most children respond well to chemotherapy, some experience resistant or relapsed disease. Building on an immunotherapy treatment called CAR T cell therapy, Dr. Nabil Ahmed and his colleagues from multiple institutions were able to engineer a new fighter T cell that more effectively targeted leukemia cells in preclinical studies.

Chimeric antigen receptor (CAR) T cell therapy, a form of immunotherapy, was developed to help children for whom chemotherapy was not enough, said Ahmed, associate professor of pediatrics, section of hematology-oncology at Baylor College of Medicine and Texas Childrens Hospital. He is the senior author of the findings published by Dr. Kristen Fousek and colleagues in the journal Leukemia.

CAR T cell therapy uses a patients T cells, a type of immune cell that is usually involved in fighting cancer. Several research groups have genetically engineered patients T cells to express a CAR molecule to recognize CD19, a protein (antigen) found on leukemia cells. When these CAR T cells are introduced back into the patient, they can attack the cancer more effectively. Although initial treatment using CAR T cells offered impressive initial results, nearly half of the patients who received the therapy later relapsed because the cancer adapted to the attack by not producing the target protein CD19 anymore. As a result of this adaptation, the cancer became invisible to the engineered T cells.

Looking to improve this promising immunotherapy, Ahmed collaborated with Dr. Hisham Abdel-Azim, an investigator in the Saban Research Institute of Childrens Hospital Los Angeles and co-corresponding author, to study the pattern of target change before and after treatment with CD19 CAR T cells. Accordingly, Fousek, then a graduate student in Ahmeds laboratory at the Center for Cell and Gene Therapy, engineered a T cell that targets not only CD19, but also two other proteins found on leukemia cells, CD20 and CD22.

Its like using a trident to attack the cancer instead of a spear, said Ahmed, who also is a member of Baylors Dan L Duncan Comprehensive Cancer Center.

The team used this three-pronged weapon against leukemia cells in preclinical studies and developed new methods to monitor how well it worked.

The new CAR T cells, named TriCAR T cells, targeting CD19/20/22 were significantly more effective than T cells that target CD19 alone. When the leukemia cells stopped producing CD19 and were no longer visible to CD19 CAR T cells, TriCAR T cells were still effective.

TriCAR T cells bind to more cancer cells and these connections are much stronger, said Abdel-Azim.

We studied the behavior of individual TriCAR T cells in the laboratory of Dr. Varadarajan at the University of Houston; the new TriCAR T cells are more effective serial killers of leukemia, Ahmed said.

Although a clinical trial will be needed before the new TriCAR T cell therapy could be used on patients, the early lab results suggest a path that could be significantly more effective in battling resistant leukemia.

See the publication to find the complete list of authors, their affiliations and sources of support for this project.

By Ana Mara Rodrguez, Ph.D.

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Trading a spear for a trident is promising in the fight against leukemia - Baylor College of Medicine News

Latest market study on Gene Therapy Market to 2027 Global Analysis and Forecasts By Cell Type (Somatic Gene Therapy, Germline Gene Therapy); By Application (Genetic Disorder, Cancer, Neurological Disorder, and Others). Global Gene Therapy Market Status and Future Forecast 2020-2027. The Insight Partners evaluating the market, highlighting opportunities, risk side analysis, and leveraged with strategic and tactical decision-making support. The study provides information on market trends and development, drivers, capacities, technologies, and on the changing investment structure of the Global Gene Therapy Market. Some of the key players profiled in the study are Sangamo Therapeutics, Inc., bluebird bio, Inc., uniQure N.V., AveXis, Inc., Vineti, Solid Biosciences., Spark Therapeutics, Inc., CHIMERON BIO, RENOVA THERAPEUTICS, HORAMA S.A., etc.

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MARKET INTRODUCTION

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

MARKET DYNAMICS

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

Answers that the report acknowledges:

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MARKET SCOPE

The Global Gene Therapy Market Analysis to 2027 is a specialized and in-depth study of the biotechnology industry with a special focus on the global market trend analysis. The report aims to provide an overview of gene therapy market with detailed market segmentation by cell type, application and geography. The global gene therapy market is expected to witness high growth during the forecast period. The report provides key statistics on the market status of the leading gene therapy market players and offers key trends and opportunities in the market.

MARKET SEGMENTATION

The gene therapy market is segmented based on cell type as, somatic gene therapy and germline gene therapy. The application segment is classified as genetic disorder, cancer, neurological disorder and others. The cancer segment is expected to grow in coming years as the need of gene correction is must as the gene corrector is introduced to produce a another gene or to inhibit the expression of another gene or to disturb the activity of another gene.

The report analyzes factors affecting gene therapy market from both demand and supply side and further evaluates market dynamics effecting the market during the forecast period i.e., drivers, restraints, opportunities, and future trend. The report also provides exhaustive PEST analysis for all five regions namely; North America, Europe, APAC, MEA and South & Central America after evaluating political, economic, social and technological factors effecting the gene therapy market in these regions.

Strategic Points Covered in Table of Content of Global Gene Therapy Market:

Chapter 1: Introduction, market driving force product Objective of Study and Research Scope the Gene Therapy market

Chapter 2: Exclusive Summary the basic information of the Gene Therapy Market.

Chapter 3: Displaying the Market Dynamics- Drivers, Trends and Challenges of the Gene Therapy

Chapter 4: Presenting the Gene Therapy Market Factor Analysis Porters Five Forces, Supply/Value Chain, PESTEL analysis, Market Entropy, Patent/Trademark Analysis.

Chapter 5: Evaluating the leading manufacturers of the Gene Therapy market which consists of its Competitive Landscape, Peer Group Analysis, BCG Matrix & Company Profile

Chapter 6: To evaluate the market by segments, by countries and by manufacturers with revenue share and sales by key countries in these various regions.

Reasons To buy

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Gene Therapy Market Segmented by Region/Country: North America, Europe, Global, Middle East & Africa, and Central & South America

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The Insight Partners is a one stop industry research provider of actionable intelligence. We help our clients in getting solutions to their research requirements through our syndicated and consulting research services. We are committed to provide highest quality research and consulting services to our customers. We help our clients understand the key market trends, identify opportunities, and make informed decisions with our market research offerings at an affordable cost.

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Gene Therapy Market: Study Navigating the Future Growth Outlook | Sangamo Therapeutics, Inc., bluebird bio, Inc., uniQure NV - Science In Me

The Gene Therapy Products market report is an important element which gains demand from all corners of the globe. It provides product overview and highlights of product and application segments of the global market including price, revenue, sales, sales growth rate, and market share by product. Primary and secondary research methodologies have been employed for a detailed study to generate this report. This report performs SWOT analysis of major key players of Healthcare industry based on strengths, weaknesses, opportunities and threats as well as companys internal & external environments. To generate this world-class Gene Therapy Products Market report, the combination of best industry insight, practical solutions, talent solutions and latest technology have been used.

With this Gene Therapy Products market research report, it becomes easy to save and reduce time, that would be otherwise required for entry-level research, by identifying the growth, size, leading players and segments in the global Gene Therapy Products market. The report suggests that the market has shown rapid development in the current and past years and is going to progress with continuing growth in the upcoming years. For this report, market analysts have studied the various products in the market and offered an even-handed opinion about the factors that are likely to drive the market or restrain it.

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Global gene therapy products market is set to witness a substantial CAGR in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. Rising cancer cases and unused potential for emerging markets are the major factors for the growth of this market.

Few of the major competitors currently working in the globalgene therapy products marketareAdaptimmune., Anchiano Therapeutics, bluebird bio, Inc., CELGENE CORPORATION, GlaxoSmithKline plc., Merck KGaA, Novartis AG, Achieve Life Sciences, Inc., Spark Therapeutics, Inc., Abeona Therapeutics, Inc, Adverum, agtc, Arbutus Biopharma, Audentes Therapeutics, AveXis, Inc., CRISPR Therapeutics, Intellia Therapeutics, Inc and Gilead Sciences,Inc. among others.

Market Definition:Global Gene Therapy Products Market

Gene therapy or human gene therapy is a process which is used to modify gene for the treatment of any disease. Plasmid DNA, bacterial vector, human gene editing technology and viral vectors are some of the most common type of gene therapy products. The main aim of the gene therapy is to replace the dysfunctional genes. Somatic and germline are some of the most common type of the gene therapy.

Complete report on Global Gene Therapy Product Market Research Report 2019-2026 spread across 350 Pages, profiling Top companies and supports with tables and figures

Segmentation: Global Gene Therapy Products Market

Gene Therapy Products Market : By Product

Gene Therapy Products Market : By Application

Gene Therapy Products Market : ByGeography

Read Complete Details with TOC Herehttps://www.databridgemarketresearch.com/toc?dbmr=global-gene-therapy-products-market

Key Developments in the Gene Therapy Products Market:

Gene Therapy Products Market Drivers

Gene Therapy Products Market Restraints

Competitive Analysis: Gene Therapy Products Market

Global gene therapy products market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of gene therapy products market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Key questions answered in the report :-

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Gene Therapy Products Market Sees Astonishing Growth in 2020. Major Players are Adverum, agtc, Arbutus Biopharma, Audentes Therapeutics, AveXis Inc. -...

HER2-positive breast cancer may be a misnomer, according to a growing body of evidence that one of the most widely recognized oncogenes, HER2, may not be the primary driver of the disease. The research, which comes from several groups including Genentech, makers of the prevailing anti-HER2 treatment, has researchers questioning whether current clinical guidelines for classifying and treating breast cancer may be off the mark. New classification schemes may better identify those patients more likely to benefit from anti-HER2 treatment, or point to therapies that might be more effective.

HER2 is short for human epidermal growth factor receptor 2. Researchers discovered HER2 breast cancer in the 1980s. Too many copies of the HER2 gene or its overexpression appeared to cause an especially aggressive form of the disease. By the late 90s, a drug called Herceptin that targeted the HER2 receptor, sped into the clinic on the US Food and Drug Administrations new fast-track status. As Dana-Farber Cancer Institute oncologist Harold Burstein put it a couple of years ago, HER2 breast cancer, once the most feared type of the disease, had gone from worst to first, largely because of Herceptin.

After analyzing multiple, published genomic data sets, researchers at Genentech, which makes Herceptin, reported in 2018 that there was no evidence that HER2 amplification represents a breast cancer subtype.

About 14 percent of breast cancers are HER2-positive, a diagnosis largely based on immunohistochemical staining to reveal an overabundance of HER2 receptors spanning the cancer cells membranes, with ambiguous cases decided by testing for an abnormally high HER2 gene copy number. The American Society of Clinical Oncology recommends Herceptin as the first-line treatment for HER2-positive breast cancer. In the US, tens of thousands of patients receive Herceptin each year. Recently, however, researchersincluding those from Herceptin maker Genentechhave begun to reconsider the HER2-positive subtype.

In the HER2-centric model of breast cancer, highly dysregulated HER2 signaling drives uncontrolled cell growth. HER2-positive breast cancers carry as many as 50 copies of the HER2 gene, compared with just two in noncancerous cells, and the gene can be highly expressed, leading to HER2 protein levels as much as 40- to 100-fold higher than normal.

But after analyzing multiple, published genomic datasets, researchers at Genentech reported in 2018 that there was no evidence that HER2 amplification (and, by extension, HER2 overabundance) represents a breast cancer subtype at all. HER2 amplification by itself seemed to have a negligible influence on the expression of genes thought to be mediated by the HER2 signaling. Rather, HER2 amplification was associated with upregulation of genes governed by androgen receptor (AR) signaling, which orchestrates the actions of testosterone and related hormones and is often boosted in breast cancer. This link between HER2 amplification and AR signaling suggests the androgen receptor may play a bigger role in the disease than previously recognized.

Other researchers have similarly reevaluated breast cancers using transcriptional profiles that look at gene expression across the genome, rather than focusing on individual genes thought to have large effect. The resulting subtypes require a more nuanced view of HER2 or dont include HER2 at all.

[T]he whole state of subdividing and classifying breast cancers is in a state of flux, says Larry Norton, a medical oncologist at Memorial Sloan Kettering Cancer Center who played a key role in the clinical trials establishing HER2-positive breast cancer as a subtype and Herceptin as the first drug for treating it. Were in a molecular era now, so we think we know more.

The conventional view of HER2 breast cancer that guides current clinical practice has been under pressure for some time. While most researchers remain convinced that HER2 is a clear driver of some breast cancers, Herceptin by itself struggled to knock down HER2-positive tumors in early clinical trials. It was the synergy with [the chemotherapy drug] paclitaxel that really put the drug on the map, says Norton, who was involved in the early trials of the combination therapy. Had we focused in on trastuzumab [Herceptin] as a single agent . . . the drug would have been abandoned.

Norton was the senior author of the published trial that led to FDA approval of Herceptin for advanced breast cancer in 1998. However, that trial did not actually demonstrate that the combination of Herceptin and paclitaxel, a type of chemotherapy drug known as a taxane, was superior to paclitaxel alone in increasing survival. Only when the data were combined with separate trial arms testing non-taxane chemotherapy with and without Herceptin did the researchers see a statistically significant difference in survival.

In addition, the two trials that led to Herceptins 2006 approval for early breast cancer later cast doubt on HER2 as a biomarker for response to the drug. A 2007 reanalysis of one of the trials found no statistical interaction between patients HER2 status and whether they benefitted from Herceptin. A similar reanalysis of the second trial corroborated this result: [Herceptin] benefit seemed independent of HER2 amplification, the researchers wrote in their report.

In 2000, a group at the Stanford University School of Medicine used state-of-the-art microarray technology to propose molecular subtypes of breast cancer based on the expression of more than 8,000 genes in biopsies from 42 patients. While the researchers identified a molecular signature for a HER2-based subtype, it wasnt strictly based on overabundance of the HER2 receptor or high HER2 copy numbers in the genome. Rather, they used the expression patterns from nearly 500 genes as indicators of the activation of the HER2 signaling pathway.

Breast cancer tissue stained for HER2 using antibody clone IHC002

The new subtype, which the team later dubbed HER2-enriched (HER2E), fractured the HER2 classification: a tumor might be HER2-positive based on the classical methods but not show evidence of HER2-mediated signaling activity at the transcriptional level. Only 47 percent of HER2-amplified tumors are HER2E, a concordance the 2018 paper from Genentech calls remarkably weak. Margaret Gatti-Mays of the National Cancer Institute estimates that 30 percent to 40 percent of tumors classified as HER2E tumors are not clinically HER2-positive.

A number of research groups have found no HER2-based subtype at all. In a 2005 paper, Richard Iggo, then at the Swiss Institute for Experimental Cancer Research, and colleagues performed experiments similar to those that had identified the molecular subtypes put forward by the Stanford researchers in 2000. Iggos team found that the previously discovered HER2 group did not appear to be defined by HER2, but by genes that looked like they might be related to androgen signaling, says Iggo, now at INSERM and the University of Bordeaux in France.

Similarly, in their 2018 study from Genentech, Anneleen Daemen and Gerard Manning found that AR activity is a key part of the HER2E subtype. HER2-enriched is not HER2-driven, per se. Its rather the androgen signaling, says Daemen, now head of translational medicine at ORIC Pharmaceuticals. You have androgen signaling that is massively on . . . and that is regardless of whether the HER2-enriched tumor is also HER2-positive or not.

Gerard Tarulli, an endocrinology researcher at the University of Melbourne who was not involved in the study, explains that this may be because the androgen receptor can play a role similar to the estrogen receptor (ER), which, in the most common form of breast cancer, drives tumor growth. In HER2E tumors, AR signaling is thought to replace ER as the driver, with the androgen receptor almost physically substituting for the presence of the estrogen receptor, says Tarulli.

Iggo and his colleagues called for the HER2E subtype to be renamed molecular apocrine to reflect a pathology very different from a HER2-centric model of disease, one that appears to involve signaling by AR and perhaps other pathways as well. The thing thats slightly disappointing for me, says Iggo, [is that] rather than changing the name . . . [the Stanford team] just said HER2-enriched, which perpetuates the idea that [HER2 amplification] defines a biological subtype.

Daemen agrees. When they originally defined these subtypes, they should not have used the word HER2-enriched because thats misleading, she says. [The nomenclature is] so ingrained in the research thats happening for breast cancer that people really hold on to it. . . . The field at large has not questioned those subtypes or deviated from them as much as is happening in other fields or other cancer types. The Stanford team did not respond to The Scientists request for comment.

Which subtypes are real is a very difficult question, says Tarulli. One recently published breast cancer scheme includes seven types, three of which could be considered subsets of the traditional HER2-positive categorization, while yet another research group has proposed 10 molecular subtypes, including one that pertains to HER2-type disease.

According to Daemen, her work was closely followed by the clinicians and the biomarker scientists who were running trials in breast and gastric cancers. I worked closely with the clinical teams at Genentech at that time to see if we could explain why some patients in our trials were not responding as well to anti-HER2 agents because of AR expression, for example. However, Daemen left Genentech last fall and says, to my knowledge, there is no additional research that is being undertaken [at the company] as a result of this. Genentech spokesperson Kayla Bruneau says that the company did not have any researchers available to discuss the [Daemen and Manning] paper at this time.

For now, clinical practice continues in strict adherence to the HER2-positive subtype as classically defined. HER2 receptor overabundance or HER2amplification has been validated as a biomarker of clinical effect for HER2-targeting therapies, Gatti-Mays says in a statement emailed to The Scientist. But the diversity of subtypes churned out by recent molecular analyses has thrown the field for a loop.

We thought that there should be some underlying molecular characteristic that is shared by all the HER2-positive breast tumors, and that turned out not to be the case, says Daemen. This absence of HER2-related biology in HER2-positive tumors seems to clash with how targeted therapies, including anti-HER2 treatments, are thought to work, namely, by interrupting a well-understood, cancer-driving pathway.

If you can demonstrate that adding a HER2 enrichment analysis in terms of clinical decision-making improves patient outcomes, then that classification needs to go in.

Gerard Tarulli, University of Melbourne

HER2E in particular is gaining traction as a possible subtype to consider for eventual clinical use. According to a meta-analysis published earlier this year, HER2E patients show a stronger clinical benefit from anti-HER2 therapies than other molecular subtypes. Tarulli says the inclusion of HER2E in future clinical trials of anti-HER2 therapies makes a lot of sense, because now were understanding more about the mechanisms behind what [HER2] is doing.

Once clinical trial data start rolling in, researchers can make a more informed decision, he says. If you can demonstrate that adding a HER2 enrichment analysis in terms of clinical decision-making improves patient outcomes, then that classification needs to go in. Tarulli also says that for HER2E patients, inhibiting both the androgen receptor and HER2 makes a lot of sense and will probably increase the effect of both. The authors of the meta-analysis did not respond to a request for comment.

Daemen disagrees with using HER2E to decide who gets anti-HER2 therapy, which can have heart-related side effects, in a clinical trial. If you were to base treatment decisions on the HER2-enriched label of a tumor, you would do harm, rather, in patients that lack HER2 amplification.

HER2E is an interesting subgroup, agrees Gatti-Mays, but [m]ore information is needed about the long-term clinical outcomes of these HER2E patients.

Still, earlier conceptions might be falling away, Norton says. The simplistic notionHER2 is a signaling molecule, you blockade, stop the signaling, so you get apoptosis rather than going into cell division[that] all makes sense, but it could be wrong.

Robert Fortner is a freelance journalist based in Portland, Oregon. Email him atrobertwfortner@gmail.comor follow him on Twitter@robertfortner1.

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Who Benefits from Herceptin and Other Anti-HER2 Cancer Therapies? - The Scientist

RANCHO CORDOVA, Calif., April 13, 2020 /PRNewswire/ --ThermoGenesis Holdings, Inc.(Nasdaq: THMO), a market leader in automated cell processing tools and services in the cell and gene therapy field, announced today that its joint venture, ImmuneCyte Life Sciences, Inc., has completed the acquisition of worldwide intellectual property for developing fully human antibody therapeutics for COVID-19. The acquired intellectual property includes four high-affinity monoclonal antibody drug candidates against SARS-CoV-2 (the virus causing COVID-19) and tools for screening and quantifying efficacy of such neutralizing antibodies. These high-affinity neutralizing monoclonal antibodies were obtained from the screening of enriched B cell libraries from individuals who have recently recovered from COVID-19 (i.e. from convalescent whole blood). The technologies were originally developed by a research team affiliated withEast China Normal University, one of the most prestigious universities in China. The acquisition includes an undisclosed upfront cash payment and low double-digit future revenue sharing for the monoclonal antibody drug candidates under development. ThermoGenesis currently holds approximately 19% of the equity of ImmuneCyte.

There are currently no regulatory approved vaccine or drug therapies for COVID-19. Current estimates are that a vaccine for emergency use may be available by early 2021. Convalescent plasma therapy is an exploratory approach that involves giving patients an infusion of plasma from people who have recovered from COVID-19. ThermoGenesis is developing a convalescent plasma strategy for COVID-19 with its SARS-CoV-2 (COVID-19) IgM/IgG Antibody Fast Detection Kit, which, together with ImmuneCyte's newly acquired technologies, can quickly identify individuals who have developed protective immunity against the virus, and then, using ThermoGenesis' proprietary cell processing platform, allows for the simultaneous isolation of convalescent plasma and immune cells for potential anti-COVID-19 antibody development.

The key effectors in convalescent plasma are neutralizing antibodies, which can block the entrance of the virus into the cells, thus achieving its anti-viral effects. Purified neutralizing polyclonal and monoclonal antibodies could be safer and more potent and are expected to have much higher efficacy than convalescent plasma. With the acquisition of these key intellectual property technologies, ImmuneCyte will be further engaged in the development of anti-COVID-19 polyclonal and monoclonal neutralizing antibodies for COVID-19 treatment.

"The technologies acquired from a team associated with the world's leading institute in biological research will allow us to spearhead our antibody drug development against COVID-19," Chris Xu, Ph.D, Chairman and Chief Executive Officer of ThermoGenesis, commented, "Given the gravity of the situation, both ThermoGenesis and ImmuneCyte are rapidly mobilizing our respective expertise and resources to fight against the pandemic. The acquisition of the new technologies by ImmuneCyte, along with ThermoGenesis' established proprietary technologies in the automated cellular processing field, will allow us to develop highly efficient processing methods for making convalescent plasma and pursue the development of fully human polyclonal and monoclonal antibody therapies against COVID-19."

About ThermoGenesis Holdings, Inc.

ThermoGenesis Holdings, Inc. develops, commercializes and markets a range of automated technologies for CAR-T and other cell-based therapies. The Company currently markets a full suite of solutions for automated clinical biobanking, point-of-care applications, and automation for immuno-oncology, including its semi-automated, functionally-closed CAR-TXpressplatform, which streamlines the manufacturing process for the emerging CAR-T immunotherapy market. For more information about ThermoGenesis, please visit:www.thermogenesis.com.

About ImmuneCyte Life Sciences, Inc.

ImmuneCyte, a joint venture which is 19% owned by ThermoGenesis, is one of the first U.S. based immune cell banks to develop and commercialize immunological products and services. The Company provides clients the opportunity to bank their own immune cells when the cells are "healthy and unaffected" as a future resource for cellular immunotherapies, such as CAR-T. ImmuneCyte utilizes a proprietary CAR-TXpress platform, a GMP compliant close-system capable of automated separating and cryopreserving different components from blood, simultaneously. For more information about ImmuneCyte Life Sciences Inc., please visit: http://www.immunecyte.com.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements are not statements of historical facts, but rather reflect the current expectations of ThermoGenesis concerning future events and results. Forward-looking statements based on ThermoGenesis' current assumptions, expectations and beliefs are generally identifiable by use of words "may," "might," "will," "should," "expects," "plans," "anticipates," "believes," "estimates," "predicts," "potential" or "continue," or similar expressions and involve significant risks and uncertainties that could cause actual results, developments and business decisions to differ materially from those contemplated by these statements. Such forward-looking statements involve risks, uncertainties and other factors, some of which are beyond our control, which may cause our actual results, performance or achievements, or industry results, to be materially different from any future results, performance, or achievements expressed or implied by such forward-looking statements. These risks, uncertainties and factors include, but are not limited to, market and other conditions, as well as those risk factors discussed in Item 1A of our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission ("SEC") as well as other documents that may be filed by ThermoGenesis from time to time with the SEC, which are available atwww.sec.gov. We undertake no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. You are cautioned not to unduly rely on such forward-looking statements when evaluating the information presented in this press release.

Company Contact:Wendy Samford916-858-5191ir@thermogenesis.com

Investor Contact:Paula Schwartz,Rx Communications917-322-2216pschwartz@rxir.com

View original content:http://www.prnewswire.com/news-releases/thermogenesis-announces-its-immunecyte-joint-venture-acquires-key-technologies-to-develop-fully-human-polyclonal-and-monoclonal-antibody-therapeutics-for-covid-19-301039184.html

SOURCE ThermoGenesis Holdings, Inc.

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ThermoGenesis Announces Its ImmuneCyte Joint Venture Acquires Key Technologies to Develop Fully Human Polyclonal and Monoclonal Antibody Therapeutics...

GERMANTOWN, Md., April 13, 2020 (GLOBE NEWSWIRE) -- Orgenesis Inc. (NASDAQ: ORGS), a pioneering, global biotech company committed to lowering costs, accelerating commercialization, and transforming the delivery of cell and gene therapies (CGTs), announces it has entered into an agreement to acquire the assets of Tamir Biotechnology, Inc, including ranpirnase, TamirBios broad spectrum anti-viral platform. The acquisition will be completed for total stock and cash consideration valued at approximately $19 million, based on the value of the stock at closing. Orgenesis plans to combine ranpirnase with its co-developed Bioxome technology for enhanced payload delivery directly to cells.

TamirBio is a clinical stage anti-viral therapeutics company engaged in the discovery and development of a new class of prophylactic and therapeutic drugs for the treatment of viruses and other pathological conditions. TamirBios lead asset, ranpirnase, a ribonuclease (RNase), is a member of the superfamily of enzymes that catalyze the degradation of RNA, and mediate several essential biological activities, including the regulation of cell proliferation, maturation, differentiation, and cell death. Therefore, it is a potential candidate for the development of therapeutics for life-threatening diseases, including viral and autoimmune diseases, that require anti-proliferative and apoptotic properties.

TamirBios first target is the human papilloma virus (HPV), the worldwide leading cause of genital warts. The companys lead asset, topical ranpirnase, was evaluated in a phase I/II clinical trial targeting genital warts. The phase I/II study demonstrated clear clinical effect of ranpirnase. Additional clinical trials are currently being planned.

Orgenesis and TamirBio plan to combine ranpirnase with Bioxomes, which have demonstrated the ability to fuse with cell membranes and deliver an intracellular cargo, in a similar manner to natural exosomes. Bioxomes can carry selected therapeutic cargo inside the target cells when loaded with predesignated genetic material, proteins, signaling molecules and drugs, as these mimic the natural membrane fusion capacity of exosomes. Orgenesis and TamirBio believe the combination of the two platforms will result in enhanced efficacy and anti-viral results.

Vered Caplan, CEO of Orgenesis, said, Combining TamirBios broad antiviral platform, ranpirnase, with Bioxomes could result in an enhanced payload delivery into cells. In independent third-party testing, ranpirnase has shown anti-viral activity in multiple viruses. Additionally, over 1,000 patients have been dosed with ranpirnase in previous cancer/mesothelioma clinical trials. Ranpirnase demonstrated a strong safety and tolerability profile that should help accelerate the approval pathway. We believe combining ranpirnase with the Bioxome platform has the potential to become a potent and powerful combination given the natural intracellular trafficking abilities of Bioxomes. We look forward to testing a variety of additional anti-viral therapies in the near future.

We are honored to combine forces with Orgenesis to advance this powerful platform, stated, Jamie Sulley, President of TamirBio. Ranpirnase has already demonstrated preclinical antiviral activity in such viral diseases as HPV, HIV, Ebola, and SARS. Not only do we believe Orgenesis will help advance ranpirnase through the clinic using their global development platform, but by combining ranpirnase with the Bioxome technology, we believe we can deliver ranpirnase through a more effective delivery mechanism.

About OrgenesisOrgenesis is a pioneering global biotech company which is unlocking the full potential of personalized therapies and closed processing systems through its Cell & Gene Therapy Biotech Platform, with the ultimate aim of providing life changing treatments at the Point of Care to large numbers of patients at low cost. The Platform consists of: (a) POCare Therapeutics, a pipeline of licensed cell and gene therapies (CGTs), and proprietary scientific knowhow; (b) POCare Technologies, a suite of proprietary and in-licensed technologies which are engineered to create customized processing systems for affordable point of care therapies; and (c) POCare Network, a collaborative, international ecosystem of leading research institutes and hospitals committed to clinical development and supply of CGTs at the point of care. By combining science, technologies and a collaborative network, Orgenesis is able to identify the most promising new therapies and provide a pathway for them to reach patients more quickly, more efficiently and at scale, thereby unlocking the power of cell and gene therapy for all. Additional information is available at: http://www.orgenesis.com.

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citybizlist : Washington DC : Orgenesis To Acquire Assets of Tamir Biotechnology - Citybizlist Real Estate

New York, April 14, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Bioinformatics Market Research Report by Product , by Application , by End User - Global Forecast to 2025" - https://www.reportlinker.com/p05881705/?utm_source=GNW

The Global Bioinformatics Market is expected to grow from USD 14,086.57 Million in 2019 to USD 32,672.24 Million by the end of 2025 at a Compound Annual Growth Rate (CAGR) of 15.05%.

Market Segmentation & Coverage:This research report categorizes the Bioinformatics to forecast the revenues and analyze the trends in each of the following sub-markets:

On the basis of Product, the Bioinformatics Market is studied across Bioinformatics Platform, Bioinformatics Service, and Knowledge Management Tool. The Bioinformatics Platform further studied across Sequence Alignment Platforms, Sequence Analysis Platforms, Sequence Manipulation Platforms, and Structural & Functional Analysis Platforms. The Bioinformatics Service further studied across Data Analysis Services, Database & Management Services, and Sequencing Services. The Knowledge Management Tool further studied across Generalized Knowledge Management Tools and Specialized Knowledge Management Tools.

On the basis of Application, the Bioinformatics Market is studied across Agriculture, Chemoinformatics, Genomics, Medicine, Metabolomics, Microbial Genome Applications, Molecular Phylogenetics, Proteomics, and Transcriptomics.

On the basis of End User, the Bioinformatics Market is studied across Academics, Agricultural Bioinformatics, Animal Bioinformatics, Clinical Diagnostics, Drug Development, Environmental Bioinformatics, Forensic Bioinformatics, Gene Therapy, Medical Bioinformatics, Molecular Medicine, and Reproductive Biotechnology.

On the basis of Geography, the Bioinformatics Market is studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas region is studied across Argentina, Brazil, Canada, Mexico, and United States. The Asia-Pacific region is studied across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, South Korea, and Thailand. The Europe, Middle East & Africa region is studied across France, Germany, Italy, Netherlands, Qatar, Russia, Saudi Arabia, South Africa, Spain, United Arab Emirates, and United Kingdom.

Company Usability Profiles:The report deeply explores the recent significant developments by the leading vendors and innovation profiles in the Global Bioinformatics Market including ABM Inc, Accelrys Inc., Agilent Technologies Inc, BGI Group, Biomax Informatics AG, DNASTAR, Eurofins Scientific, Illumina Inc., Partek Incorporated, Perkinelmer, Inc., QIAGEN Bioinformatics, Sophia Genetics, Thermo Fisher Scientific, Waters Corporation, and WuXi NextCODE..

FPNV Positioning Matrix:The FPNV Positioning Matrix evaluates and categorizes the vendors in the Bioinformatics Market on the basis of Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Competitive Strategic Window:The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies. The Competitive Strategic Window helps the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. During a forecast period, it defines the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth.

The report provides insights on the following pointers:1. Market Penetration: Provides comprehensive information on sulfuric acid offered by the key players2. Market Development: Provides in-depth information about lucrative emerging markets and analyzes the markets3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, and manufacturing capabilities of the leading players5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and new product developments

The report answers questions such as:1. What is the market size and forecast of the Global Bioinformatics Market?2. What are the inhibiting factors and their impact analysis shaping the Global Bioinformatics Market during the forecast period?3. What is the competitive position if vendors in the Global Bioinformatics Market?4. How Porters Five Forces define the Global Bioinformatics Market landscape?5. What are the technology trends and regulatory frameworks in the Global Bioinformatics Market?6. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Bioinformatics Market?7. What is the competitive strategic window for opportunities in the Global Bioinformatics Market?8. What are the modes and strategic moves considered suitable for entering the Global Bioinformatics Market?Read the full report: https://www.reportlinker.com/p05881705/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The Global Bioinformatics Market is expected to grow from USD 14,086.57 Million in 2019 to USD 32,672.24 Million by the end of 2025 at a Compound...

Latest Study on Industrial Growth ofGlobal Gene Editing Technologies Market2019-2025. A detailed study accumulated to offerLatest insights about acute features of the Gene Editing Technologies market. The report contains different market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market. It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary and SWOT analysis.

The Major Players Covered in this Report: Thermo Fisher Scientific, Merck, Sangamo Therapeutics, Lonza, Transposagen Biopharmaceuticals, Editas Medicine, CRISPR Therapeutics & Agilent Technologies

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This study also covers company profiling, specifications and product picture, sales, market share and contact information of various regional, international and local vendors of Global Gene Editing Technologies Market. The market opposition is frequently developing greater with the rise in scientific innovation and M&A activities in the industry. Additionally, many local and regional vendors are offering specific application products for varied end-users. The new merchant applicants in the market are finding it hard to compete with the international vendors based on reliability, quality and modernism in technology.

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The titled segments and sub-section of the market are illuminated below:

In-depth analysis of Global Gene Editing Technologies market segments by Types: , Double Strand Break Repair, Engineered Nucleases & Clustered Regularly Interspaced Short Palindromic Repeats

In-depth analysis of Global Gene Editing Technologies market segments by Applications: Gene Therapy & Eradicating Diseases

Major Key Players of the Market: Thermo Fisher Scientific, Merck, Sangamo Therapeutics, Lonza, Transposagen Biopharmaceuticals, Editas Medicine, CRISPR Therapeutics & Agilent Technologies

Regional Analysis for Global Gene Editing Technologies Market:North America (United States, Canada and Mexico)Europe (Germany, France, UK, Russia and Italy)Asia-Pacific (China, Japan, Korea, India and Southeast Asia)South America (Brazil, Argentina, Colombia etc.)Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

Furthermore, the years considered for the study are as follows:Historical year 2013-2017Base year 2018Forecast period** 2018 to 2023 [** unless otherwise stated]

**Moreover, it will also include the opportunities available in micro markets for stakeholders to invest, detailed analysis of competitive landscape and product services of key players.

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Guidance of the Global Gene Editing Technologies market report:

Detailed considerate of Gene Editing Technologies market-particular drivers, Trends, constraints, Restraints, Opportunities and major micro markets. Comprehensive valuation of all prospects and threat in the Global Gene Editing Technologies market. In depth study of industry strategies for growth of the Gene Editing Technologies market-leading players. Gene Editing Technologies market latest innovations and major procedures. Favorable dip inside Vigorous high-tech and market latest trends remarkable the Market. Conclusive study about the growth conspiracy of Gene Editing Technologies market for forthcoming years.

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Detailed TOC of Gene Editing Technologies Market Research Report-

Gene Editing Technologies Introduction and Market Overview Gene Editing Technologies Market, by Application [Gene Therapy & Eradicating Diseases]

Gene Editing Technologies Industry Chain Analysis Gene Editing Technologies Market, by Type [, Double Strand Break Repair, Engineered Nucleases & Clustered Regularly Interspaced Short Palindromic Repeats]

Industry Manufacture, Consumption, Export, Import by Regions (2013-2018) Industry Value ($) by Region (2013-2018)

Gene Editing Technologies Market Status and SWOT Analysis by Regions

Major Region of Gene Editing Technologies Marketi) Global Gene Editing Technologies Salesii) Global Gene Editing Technologies Revenue & market share Major Companies List Conclusion

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

About Author:HTF Market Report is a wholly owned brand of HTF market Intelligence Consulting Private Limited. HTF Market Report global research and market intelligence consulting organization is uniquely positioned to not only identify growth opportunities but to also empower and inspire you to create visionary growth strategies for futures, enabled by our extraordinary depth and breadth of thought leadership, research, tools, events and experience that assist you for making goals into a reality. Our understanding of the interplay between industry convergence, Mega Trends, technologies and market trends provides our clients with new business models and expansion opportunities. We are focused on identifying the Accurate Forecast in every industry we cover so our clients can reap the benefits of being early market entrants and can accomplish their Goals & Objectives.

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Gene Editing Technologies Market Major Technology Giants in Buzz Again | Merck, Sangamo Therapeutics, Lonza - Science In Me

University of Georgia Honors student Landon Clark is among 396 undergraduates across the nation to be recognized as Barry Goldwater Scholars, earning the highest undergraduate award of its type for the fields of the natural sciences, mathematics and engineering.

Clark, from Leesburg, is a third-year CURO Honors Scholar who is triple majoring in biochemistry and molecular biology, genetics and biology.

Since 1995, 61 UGA students have received the Goldwater Scholarship, which recognizes exceptional sophomores and juniors across the United States.

The University of Georgia is proud of Landon and pleased that he is extending our record of success in this prestigious competition, said President Jere W. Morehead. Already as an undergraduate student, he is helping UGA fulfill our commitment to conduct research that helps people lead longer, healthier lives.

This year, recipients were selected from a field of more than 5,000 college sophomores and juniors who were nominated by 461 academic institutions nationwide. Each awardee will receive up to $7,500 toward the cost of tuition, fees, books, and room and board.

Of this years Goldwater Scholars, 50 are mathematics and computer science majors, 287 are majoring in the natural sciences and 59 are majoring in engineering.

I am thrilled for Landon, who is absolutely one of our very best students, said David S. Williams, associate provost and director of the Honors Program, who serves as the UGA campus faculty representative for the Goldwater Scholarship. As a CURO Honors Scholar, Landon was recruited to come to UGA because of his promise as a researcher. This Goldwater award and recognition proves that his hard work is paying off. I look forward to watching his career continue to blossom in the future.

Clarks long-term plans include pursuing an MD/PhD in immunology, performing translational research on immune diseases using gene and immunotherapy techniques, and teaching at a university. As a translational researcher specializing in immunology, he plans to use gene therapy techniques to treat and cure immune disorders in humans.

Since his first weeks as a UGA freshman in August 2017, Clark has conducted research in the lab of Michael Terns, Distinguished Research Professor of Biochemistry and Molecular Biology. He researches the complex processes of CRISPR-Cas immune systems through a model archaeal organism, Pyrococcus furiosus. Now a junior, he has collaborated on three different projects, co-authored a paper, and mentors other undergraduates in this field.

Last summer, he worked in the translational research lab of H. Trent Spencer, a professor of pediatrics and director of the gene and cell therapy program at Emorys Winship Cancer Institute. There, Clark researched immune diseases and potential gene therapy techniques. In 2017, he also conducted research on sociological variables influencing educational inequality with Dawn Robinson, a professor of sociology at UGA.

Clark is a Crane Leadership Scholar, director of academic outreach for the Student Government Association, director of internal communications for UGA Red Cross, an exam writer for UGA Science Olympiad Outreach, co-president of the Biochemistry Undergraduate Society, co-president of the Omicron Delta Kappa National Honor Society, and treasurer of the Honors Program Student Council. He also volunteers in the neuroscience and cardiology units at Piedmont Athens Regional Medical Center.

Clark has presented his research at 10 conferences. He also studied neurology and cancer biology through UGAs Studies Abroad Program in Cortona, located in Tuscany, Italy.

The scholarship honoring Sen. Barry Goldwater was created to encourage outstanding students to pursue careers in the fields of mathematics, natural sciences and engineering. Since its first award in 1989, the Barry Goldwater Scholarship and Excellence in Education Foundation has bestowed 8,628 scholarships worth more than $68 million.

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UGA's Landon Clark named 2020 Goldwater Scholar - University of Georgia

Detailed market survey on the Global Hemophilia Gene Therapy Market Research Report 2020-2026. It analyses the vital factors of the Hemophilia Gene Therapy market supported present business Strategy, Hemophilia Gene Therapy market demands, business methods utilised by Hemophilia Gene Therapy market players and therefore the future prospects from numerous angles well. Business associatealysis could be a market assessment tool utilized by business and analysts to grasp the quality of an business. Hemophilia Gene Therapy Market report It helps them get a sense of what is happening in an industry, i.e., demand-supply statistics, Hemophilia Gene Therapy Market degree of competition within the industry, Hemophilia Gene Therapy Market competition of the business with different rising industries, future prospects of the business.

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The Global Hemophilia Gene Therapy market worth about xx billion USD in 2020 and it is expected to reach xx billion USD in 2026 with an average growth rate of x%. United States is the largest production of Hemophilia Gene Therapy Market and consumption region in the world, Europe also play important roles in global Hemophilia Gene Therapy market while China is fastest growing region.

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Spark TherapeuticsFreeline TherapeuticsSangamo TherapeuticsUltragenyxuniQureShire PLCBioMarinBioverativ

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Research on Hemophilia Gene Therapy Market (impact of COVID-19) 2020-2026 Spark Therapeutics, Freeline Therapeutics, Sangamo Therapeutics, Ultragenyx...

Welcome to the latest edition of our weekly EuroBiotech Report. We start this week with AstraZeneca, which revealed it aims to have a COVID-19-neutralizing antibody in the clinic in the next three to five months. AstraZeneca's compatriot GlaxoSmithKline also shared an update on its COVID-19 activities. GSK added Xiamen Innovax Biotech to the list of organizations it is working with on a COVID-19 vaccine. While those are just two of the many initiatives targeting COVID-19, Wellcome is worried about a funding shortfall. Wellcome wants businesses to commit $8 billion this month to keep programs moving forward quickly. Researchers working outside of COVID-19 are faring worse still, with Cancer Research UK responding to the pandemic with deep cuts. Away from COVID-19, Evotec entered the gene therapy space through a deal with Takeda. And more. Nick Taylor1. AstraZeneca targets summer start for COVID-19 antibody trial

AstraZeneca has outlined its multipronged approach to the development of antibodies against the pandemic SARS-CoV-2 virus. The Big Pharma is applying its own discovery capabilities to the task and helping to evaluate candidates identified by academic partners in China and the U.S.2. GSK allies with Innovax for COVID-19 vaccine R&D project

GlaxoSmithKline has teamed up with Xiamen Innovax Biotech to evaluate a vaccine against the novel coronavirus behind the COVID-19 pandemic. The agreement gives Innovax access to a GSK adjuvant to enhance the immune response triggered by its recombinant protein-based vaccine.3. Wellcome targets $8B raise in weeks to fix COVID-19 funding gap

Virtual Clinical Trials Online

This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.

Wellcome is calling on businesses to invest in an $8 billion (7 billion) fund focused on reducing cases of COVID-19 to zero as soon as possible. Almost half the money is earmarked for the development of drugs and vaccines that Wellcome sees as the worlds best exit strategy from the pandemic.4. COVID-19 claims Cancer Research UK funding cuts, forecasting major shortfall

Cancer Research UK (CRUK) said it has been forced to take the difficult decision to make deep funding cuts across its grants and institutes as the fallout from the pandemic continues to affect all areas of biomedical research.

5. Evotec allies with Takeda to move into gene therapy R&D

Evotec has allied with Takeda to expand into gene therapy research. The move sees Evotec establish a 20-person team in Austria, the focal point of Takedas gene therapy operation, and sign up to work on programs for its Japanese partner.And more articles of note>>

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EuroBiotech ReportAZ, GSK in COVID-19, $8B funding call, CRUK cuts and Evotec - FierceBiotech

Niche players in the global gene therapy market must leverage greater government expenditure and upgrade their existing infrastructure along with expanding their gene therapy centers for sustaining their market hegemony.

ROCKVILLE, MD / ACCESSWIRE / April 8, 2020 / Global gene therapy market is poised for robust growth with net revenue pool set to exceed approximately US$ 5 Bn by 2026 end. The market is receiving tailwinds from advancements in synthetic biology. On that premise, the gene therapy market will expand 3X through over the forecast period, projects Fact.MR (2020-2026).

"Certain types of cancer such as Diffuse Large B-cell Lymphoma (DLBCL) and lymphoblastic leukemia are contributing to high mortality rates across the world. Gene therapy is gaining increasing recognition in its immense potential for treating rare diseases. Continued research and development in the area of gene therapy is supporting market growth as well," states Fact.MR.

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Gene Therapy Market - Key Findings

Gene Therapy Market - Key Driving Factors

Explore 59 tables and 79 figures of the study. Request TOC of the report at-

https://www.factmr.com/report/4648/gene-therapy-market

Gene Therapy Market - Key Restraints

Impact of COVID-19 on Gene Therapy Market

In view of ongoing onslaught of COVID-19 pandemic, the focus of major healthcare authorities of the world has gravitated towards developing vaccines for the deadly respiratory disease. Gene therapy is one such area of research which could help boost antibodies required to treat patients infected with Coronavirus. For instance, Generation Bio is exploring the potential role of gene therapy in treating COVID-19 patients. Hence, the global gene therapy market will benefit from the outbreak in that market players are rushing to develop multiple therapeutic approaches for SARS-CoV-2. Growing fears of similar Coronavirus outbreaks in the future will continue accelerating the development of gene therapy as well.

Competitive Landscape

Prominent players profiled in this Fact.MR study include, but are not limited to, Orchard Therapeutics Limited, CELGENE CORPORATION, Spark Therapeutics, Inc., Sibiono GeneTech Co. Ltd., Spark Therapeutics Inc., Gilead Sciences Inc., and Novartis AG. Developed regions remain the key focus area of major stakeholders in the global gene therapy market. Existing gene therapy centers are being prioritized by market players in order to utilize the full extent of their resources. Moreover, they are benefitting from success rates associated with gene therapy and faster drug approvals. Gilead Sciences Inc. expanded their gene therapy centers to a total of 90 recently.

About the Report

This 170-page study offers in-depth commentary on the gene therapy market. The study provides compelling insights on the gene therapy market on the basis of product (yescarta, kymriah, luxturna, strimvelis, and gendicine), application (ophthalmology, oncology, Adenosine Deaminase Deficiency- Severe Combined Immunodeficiency) across three regions (The United States, Europe, and Rest of the World).

Explore Fact.MR's Comprehensive Coverage on Healthcare Landscape

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About Fact.MR

Expert analysis, actionable insights, and strategic recommendations of the veteran research team at Fact.MR helps clients from across the globe with their unique business intelligence requirements. With a repository of over thousand reports and 1 million+ data points, the team has scrutinized the Healthcare industry across 50+ countries for over a decade. The team provides unmatched end-to-end research and consulting services. Fact.MR's healthcare market reports and industry analysis help businesses navigate challenges and take critical decisions with confidence and clarity amidst breakneck competition.

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Fact.MR11140 Rockville PikeSuite 400Rockville, MD 20852United StatesEmail: sales@factmr.comWeb: https://www.factmr.com/PR- https://www.factmr.com/media-release/1409/global-gene-therapy-market

SOURCE: FactMR

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Gene Therapy Market Set for 3X Expansion Between 2020 and 2026; COVID-19 Stimulating Development of Multiple Therapeutic Approaches: Fact.MR -...

Global Gene Therapy Market report underlines the specific study of the Biotechnology industry which explains what the market definition, classifications, applications, engagements, and global industry trends are. The market data analyses and evaluated in this Gene Therapy market report makes you achieve the business goals and objectives in preset time frame. It highlights a wide-ranging evaluation of the markets growth prospects and restrictions. This report is very useful to all sizes of business which makes it simpler to take informed decisions regarding different facets of industry. Gene Therapy market report truly acts as a backbone to the business.

A finest market research report acts as an innovative solution for the businesses in todays changing market place and hence this Gene Therapy report is generated. Key players in the market, major collaborations, merger and acquisitions along with trending innovation and business policies are also reviewed in this Gene Therapy report. The report has a list of key competitors with the required specifications and also provides the strategic insights and analysis of the key factors influencing the industry. The data and information of the Gene Therapy report not only helps business make data-driven decisions but also guarantees maximum return on investment (ROI).

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MARKET INTRODUCTION

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

Key Competitors In Market are Sangamo Therapeutics, Inc., bluebird bio, Inc., uniQure N.V., AveXis, Vineti, Solid Biosciences., Spark Therapeutics, Inc., CHIMERON BIO, RENOVA THERAPEUTICS, and HORAMA S.A.

MARKET SCOPE

The Global Gene Therapy Market Analysis to 2025 is a specialized and in-depth study of the gene therapy industry with a focus on the global market trend. The report aims to provide an overview of global gene therapy market with detailed market segmentation by cell type, application, and geography. The global gene therapy market is expected to witness high growth during the forecast period. The report provides key statistics on the market status of the leading market players and offers key trends and opportunities in the market.

Market segmentation:

By Cell Type (Somatic Gene Therapy, Germline Gene Therapy);

By Application (Genetic Disorder, Cancer, Neurological Disorder, and Others)

By Geography North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America. And 13 countries globally along with current trend and opportunities prevailing in the region.

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Gene Therapy Market 2020: Competitive Landscape and Recent Industry Development Analysis by Sangamo Therapeutics, Inc., bluebird bio, Inc., uniQure...