Archive for the ‘Gene Therapy Research’ Category

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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A real world version of Pokmon Go lets you track orangutans in the jungle - Massive Science

Add a new, 100 million California facility to the growing gene therapy infrastructure.

Merck KGaA announced they are opening a second gene therapy and viral vector factory in Carlsbad, California. The new center will be just one point in the global supply chain Big Pharma is rapidly erecting to keep manufacturing capacity for the new technology at pace with clinical development. Over the past year, Novartis, PTC Therapeutics, Pfizer and Vertex each announced or opened new facilities in Switzerland, North Carolina and New Jersey that will help build gene therapy.

In November,Reutersreported that 11 drugmakers had set aside $2 billion for the manufacturing effort. They were led by Novartis, the giant behind the second FDA-approved gene therapy in Zolgensma which planned to spend $500 million, and Pfizer, which has yet to get a gene therapy approved but will spend $600 million. Catalent and Thermo Fisher, meanwhile, each spent over a billion dollars acquiring companies involved in gene therapy or viral vector manufacturing.

Merck KGaA last shored up its gene therapy manufacturing in 2016 the year before the first gene therapy was approved in the US when they expanded their original Carlsbad facility from 44,000 to 65,000 feet. At 140,000 feet, the new facility will be more than double the size of its older neighbor. Gene therapies and the viral vectors used to deliver them will be cooked up in 1,000 liter bioreactors metal vats roughly the size of 260 gallons of milk. Merck has not said when it is scheduled for completion.

Although Merck KGaA lists no gene therapies in its most recent pipeline, the German drugmaker uses its facilities to help biotechs focused on the emerging modality to manufacture their products. The list of approved gene therapies can still be counted on one hand, but hundreds are now in clinical trials.

Manufacturing emerged as a pressing bottleneck almost as soon as Spark Therapeutics got Luxturna, a treatment for a form of inherited blindness, approved as the US first gene therapy. In an op-ed in STAT last year, Sparks head of technical operations, Diane Blumenthal, described the process of building in Philadelphia one of the first in-house gene therapy manufacturing facilities. With regulators increasingly open to approving the treatments quickly, she encouraged other companies to invest in manufacturing in advance of even knowing if the therapy works, particularly because gene therapy requires customization in ways few other modalities do.

And she warned the problem will only get more pressing.

There isnt a gene therapy manufacturing playbook yet to guide the development of gene therapies, Blumenthal wrote. Manufacturing a gene therapy is only half the battle. The other half is making enough of it, doing that as efficiently as possible, and getting it to the patients who need it. These challenges become even more urgent to tackle as the industry shifts to the next chapter in gene therapy development, from treatments made in small batches for small patient populations to bigger volumes for larger rare-disease populations and commercial scale.

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Merck KGaA adds to gene therapy manufacturing boom with 100M facility in California - Endpoints News

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Top Key Manufacturers of Gene Therapy industry Report:-

Sibiono GeneTech,Advantagene,Spark Therapeutics,Shanghai Sunway Biotech Co. LtdBluebird Bio,UniQure NVAvalanche Bio,Celladon,Sangamo,Dimension Therapeutics

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Gene Therapy Market segment by Type-

Viral vectorNon-viral vector

Gene Therapy Market segment by Application-

Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

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Gene Therapy Market Segmentation Analysis:-

Global Gene Therapy market segmentation, by solution: Biological, Chemical, Mechanical, Global Gene Therapy market segmentation, by product: Stress Protection, Scarification, Pest Protection

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

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Chapter 1 describes Gene Therapy report important market inspection, product cost structure, and analysis, Gene Therapy market size and scope forecast from 2017 to 2026. Although, Gene Therapy market gesture, factors affecting the expansion of business also deep study of arise and existing market holders.

Chapter 2 display top manufacturers of Gene Therapy market with sales and revenue and market share. Furthermore, report analyses the import and export scenario of industry, demand and supply ratio, labor cost, raw material supply, production cost, marketing sources, and downstream consumers of market.

Chapter 3, 4, 5 analyses Gene Therapy report competitive analysis based on product type, their region wise depletion and import/export analysis, the composite annual growth rate of market and foretell study from 2017 to 2026.

Chapter 6 gives an in-depth study of Gene Therapy business channels, market sponsors, vendors, dispensers, merchants, market openings and risk.

Chapter 7 gives Gene Therapy market Research Discoveries and Conclusion

Chapter 8 gives Gene Therapy Appendix

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Global Gene Therapy Market Analysis, Technologies, Innovations, Future Guidelines and Forecasts Report 2026 - Cole of Duty

Nancy J. Hutson, Ph.D.

Nancy J. Hutson, Ph.D. has been appointed to Clearside Biomedical's Board of Directors, effective April 21, 2020.

ALPHARETTA, Ga., April 22, 2020 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc.(NASDAQ:CLSD), a biopharmaceutical company dedicated to developing and delivering treatments that restore and preserve vision for people with serious back of the eye diseases, announced today that Nancy J. Hutson, Ph.D. has been appointed to the Company's Board of Directors, effective April 21, 2020.

The addition of Nancy Hutson to our Board of Directors comes at a key time as we are expanding our research and development pipeline and preparing to initiate a clinical program targeting the treatment of wet age-related macular degeneration with CLS-AX, said William Humphries, Chairman of the Clearside Board of Directors. Nancy brings a deep understanding of the business coupled with an in-depth knowledge of research, drug development and dynamic business situations from her years of R&D leadership at Pfizer and extensive board service in a number of biopharmaceutical companies. We welcome Nancy and look forward to making further progress in achieving our strategic goals.

I look forward to working with the Clearside team to leverage the Companys proprietary suprachoroidal space injection platform, said Dr. Hutson. With a flexible treatment approach, unique R&D pipeline, multiple global partnerships, and a robust patent portfolio, Clearside has a compelling opportunity to make a difference for patients with back of the eye diseases and deliver value for shareholders.

Nancy J. Hutson, Ph.D., is a highly accomplished healthcare executive with more than 30 years of experience as a seasoned research and development expert and leader within the biopharmaceutical industry. Dr. Hutson retired from Pfizer, Inc. in 2006 after spending 25 years in various research and leadership positions, serving as Senior Vice President, Pfizer Global Research and Development and Director of Pfizer's pharmaceutical R&D site, known as Groton/New London Laboratories. At Pfizer, she led 4,500 colleagues and managed a budget in excess of $1 billion. Dr. Hutson is currently a board member of Endo International, plc (ENDP), BioCryst Pharmaceuticals, Inc. (BCRX) and PhaseBio Pharmaceuticals, Inc (PHAS). She serves as Vice-Chair of the Board of Trustees at Illinois Wesleyan University. Dr. Hutson holds a Bachelor of Arts degree from Illinois Wesleyan University and a Ph.D. degree from Vanderbilt University. She completed a Postdoctoral fellowship in Biochemistry at University of Oxford.

About Clearside Biomedical

Clearside Biomedical, Inc. is a biopharmaceutical company dedicated to developing and delivering treatments that restore and preserve vision for people with serious back of the eye diseases. Clearsides proprietary SCS Microinjector targeting the suprachoroidal space (SCS) offers unique access to the macula, retina and choroid where sight-threatening disease often occurs. The Companys SCS injection platform is an inherently flexible, in-office, non-surgical procedure, intended to provide targeted delivery to the site of disease and to work with both established and new formulations of medications, as well as future therapeutic innovations such as gene therapy. For more information, please visit http://www.clearsidebio.com.

Investor and Media Contacts:

Jenny Kobin Remy Bernarda ir@clearsidebio.com(678) 430-8206

Source: Clearside Biomedical, Inc.

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/27843a49-a1c9-4e2f-a571-ff96a1a56ae3

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Clearside Biomedical Appoints Nancy J. Hutson, Ph.D. to its Board of Directors - GlobeNewswire

Bluebird Bio

Global Gene Therapy For CNS Disorders Market Segmentation

This market was divided into types, applications and regions. The growth of each segment provides an accurate calculation and forecast of sales by type and application in terms of volume and value for the period between 2020 and 2026. This analysis can help you develop your business by targeting niche markets. Market share data are available at global and regional levels. The regions covered by the report are North America, Europe, the Asia-Pacific region, the Middle East, and Africa and Latin America. Research analysts understand the competitive forces and provide competitive analysis for each competitor separately.

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Global Gene Therapy For CNS Disorders Market Regions and Countries Level Analysis

The regional analysis is a very complete part of this report. This segmentation highlights Gene Therapy For CNS Disorders sales at regional and national levels. This data provides a detailed and accurate analysis of volume by country and an analysis of market size by region of the world market.

The report provides an in-depth assessment of growth and other aspects of the market in key countries such as the United States, Canada, Mexico, Germany, France, the United Kingdom, Russia and the United States Italy, China, Japan, South Korea, India, Australia, Brazil and Saudi Arabia. The chapter on the competitive landscape of the global market report contains important information on market participants such as business overview, total sales (financial data), market potential, global presence, Gene Therapy For CNS Disorders sales and earnings, market share, prices, production locations and facilities, products offered and applied strategies. This study provides Gene Therapy For CNS Disorders sales, revenue, and market share for each player covered in this report for a period between 2016 and 2020.

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Table of Contents:

Study Coverage: It includes study objectives, years considered for the research study, growth rate and Gene Therapy For CNS Disorders market size of type and application segments, key manufacturers covered, product scope, and highlights of segmental analysis.

Executive Summary: In this section, the report focuses on analysis of macroscopic indicators, market issues, drivers, and trends, competitive landscape, CAGR of the global Gene Therapy For CNS Disorders market, and global production. Under the global production chapter, the authors of the report have included market pricing and trends, global capacity, global production, and global revenue forecasts.

Gene Therapy For CNS Disorders Market Size by Manufacturer: Here, the report concentrates on revenue and production shares of manufacturers for all the years of the forecast period. It also focuses on price by manufacturer and expansion plans and mergers and acquisitions of companies.

Production by Region: It shows how the revenue and production in the global market are distributed among different regions. Each regional market is extensively studied here on the basis of import and export, key players, revenue, and production.

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Tags: Gene Therapy For CNS Disorders Market Size, Gene Therapy For CNS Disorders Market Growth, Gene Therapy For CNS Disorders Market Forecast, Gene Therapy For CNS Disorders Market Analysis

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Gene Therapy For CNS Disorders Market Analysis by Size, Share, Top Key Manufacturers, Demand Overview, Regional Outlook And Growth Forecast to 2026 -...

Grand View Research, Inc. Market Research And Consulting

According to report published by Grand View Research, The global advanced therapy medicinal products market size was valued at USD 3.14 billion in 2019 and is projected to expand at a CAGR of 17.4% during the forecast period

The globaladvanced therapy medicinal products marketsize is expected to reach USD 9.6 billion by 2026, expanding at a CAGR of 17.4% over the forecast period, according to a new report published by Grand View Research, Inc. The use of advanced therapy medicinal products (ATMPs) is well-proven for the improvement in the quality of life and health status in the long run for patients suffering from life-threatening conditions. This is particularly for diseases with few or no alternative treatment options. These novel treatment modes deliver transformative advantages not offered by conventional forms of disease treatment, thereby driving the ATMP market.

In the current scenario, ATMPs are expensive for patients as well as health insurance schemes. Growing competition to gain revenue share could help reduce prices and improve the affordability, along with the acceptance of these products as mainstream medicine. Furthermore, manufacturers are embracing efforts to streamline technology and logistics for the provision of therapies at accessible prices.

Request a sample Copy of the Global Advanced Therapy Medicinal Products Market Research Report @ http://www.grandviewresearch.com/industry-analysis/advanced-therapy-medicinal-products-market/request/rs1

Moreover, lack of manufacturing capabilities to meet growing consumer demand coupled with the resource and budget constraints faced by advanced therapy medicinal product developers has created lucrative avenues for contract services providers and has intensified the market competition. This has led to a rise in market activities by contract service providers. For instance, in November 2019, Fujifilm Diosynth established a 60,000-square-feet Gene Therapy Innovation Center to offer downstream, upstream, and analytical services.

Further key findings from the report suggest:

Have Any Query? Ask Our Experts @www.grandviewresearch.com/inquiry/7111/ibb

Grand View Research has segmented the global advanced therapy medicinal products market on the basis of therapy type and region:

Advanced Therapy Medicinal ProductsTherapy Type Outlook (Revenue, USD Million, 2015 2026)

Advanced Therapy Medicinal ProductsRegional Outlook (Revenue, USD Million, 2015 2026)

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About Grand View Research

Grand View Research provides syndicated as well as customized research reports and consulting services on 46 industries across 25 major countries worldwide. This U.S.-based market research and consulting company is registered in California and headquartered in San Francisco. Comprising over 425 analysts and consultants, the company adds 1200+ market research reports to its extensive database each year. Supported by an interactive market intelligence platform, the team at Grand View Research Guides Fortune 500 companies and prominent academic institutes in comprehending the global and regional business environment and carefully identifying future opportunities.

Media ContactCompany Name: Grand View Research, Inc.Contact Person: Sherry James, Corporate Sales Specialist U.S.A.Email: Send EmailPhone: 1-415-349-0058, Toll Free: 1-888-202-9519Address: 201, Spear Street, 1100 City: San FranciscoState: CaliforniaCountry: United StatesWebsite: https://www.grandviewresearch.com/industry-analysis/advanced-therapy-medicinal-products-market

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Advanced Therapy Medicinal Products Market to Reach $9.6 Billion by 2026 | Key Players Spark Therapeutics, Inc.; Bluebird Bio, Inc.; Novartis AG;...

Novel promoters based on those in the herpes viruses, enabled delivery of larger genes and increased the period they were active in the nervous system.

Novel promoter sequences have been developed to improve gene therapy treatments for neurological diseases. The researchers suggest these engineered promoters should allow for the delivery of both larger genes and also enable them to remain active for longer.

Esteban Engel, a researcher in viral neuroengineering in the Princeton Neuroscience Institute, US, and his team have developed new gene promoters for use in gene therapy. The practice of replacing faulty genes or adding additional functional ones to augment a deficit or damage is thought to be a promising strategy for treating many diseases, including neurodegenerative diseases like Parkinsons and Alzheimers.

To deliver these genes to the required cells, scientists often use viral vectors engineered viruses that cannot cause disease but have all the necessary viral machinery to enter human host cells. One of the most commonly used examples is adeno-associated virus (AAV), because it is relatively harmless.

Gene promoters are added into the viral genome alongside the desired gene in order to turn on their expression. To boost the efficacy of these promoters in the nervous system, Engel and his team adopted several attributes of herpes virus promoters, because the herpes viruses can persist for years in the body through establishing a chronic infection in the nervous system.

The team reported their promoters occupied much less space than existing ones, which could enable larger genes or multiple genes to be transported in the vectors. They also observed the new promoters are long-lasting, being less prone to repression or inactivation than most, so the therapeutic genes are active for long periods of time.

The team established that these novel promoters could work with AAVs as well as other viral and non-viral gene-delivery systems.

The research was published in the journalMolecular Therapy: Methods & Clinical Development.

Originally posted here:
Novel gene promoters could improve gene therapies - Drug Target Review

NEW YORK, April 20, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that the European Medicines Agency (EMA) has granted Advanced Therapy Medicinal Product (ATMP) classification to MB-107, Mustangs lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. The U.S. Food and Drug Administration (FDA) previously granted Regenerative Medicine Advanced Therapy (RMAT) designation to MB-107 for the treatment of XSCID in August 2019.

EMA grants ATMP classifications to new therapeutics that are based on genes or cells and intended as long-term or permanent therapeutic solutions to acute or chronic human diseases at a genetic, cellular or tissue level. The ATMP program provides specific regulatory guidelines for preclinical development, manufacturing and product quality testing of ATMPs and offers incentives, including fee reductions for regulatory advice, recommendations and evaluation and certification of quality and non-clinical data.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are extremely encouraged that the EMA has granted MB-107 with ATMP classification, an important step in establishing our path to market approval and commercialization in Europe. This classification complements the RMAT designation we received last year from the FDA and brings us closer to realizing our goal of commercializing MB-107 for XSCID patients, as these patients are in desperate need of innovative and potentially curative treatment options.

MB-107 is currently being assessed in two Phase 1/2 clinical trials for XSCID: the first in newly diagnosed infants under the age of two at St. Jude Childrens Research Hospital (St. Jude), UCSF Benioff Childrens Hospital in San Francisco and Seattle Childrens Hospital and the second in patients over the age of two who have received prior hematopoietic stem cell transplantation at the National Institutes of Health. Under a licensing partnership with St. Jude, Mustang intends to develop the lentiviral gene therapy for commercial use as MB-107.

About Mustang BioMustang Bio, Inc. (Mustang) is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission. Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

Original post:
Mustang Bio Receives Advanced Therapy Medicinal Product Classification from European Medicines Agency for MB-107 Lentiviral Gene Therapy for X-Linked...

Evotec is building a gene therapy R&D center manned by ex-Takeda scientists and has already landed a multi-year drug discovery pact for it with their former Japanese employer.

Evotecis expanding into the field of gene therapy by building an R&D center in Austria using scientists previously employed by Takeda, whose first project will be a long-term research and discovery project with their former Japanese employer, exploring oncology, rare diseases, neuroscience and gastroenterology.

The entry by the German discovery alliance and development partnership group into gene therapy further expands its capabilities beyond small molecules, cellular therapies and biologics while the research alliance withTakeda Pharmaceutical, announced on 6 April, builds upon an existing collaboration begun last September around drug discovery programs in which Evotec will deliver clinical candidates for Takeda to pursue into clinical development.

Image: iStock/sittithat tangwitthayaphum

This addition of gene therapy is the latest step in a very long strategy at Evotec to build a truly comprehensive organization which can deliver medicines discovery and development right across the different modalities, or scientific tools, that we see in modern medicine today, Evotec chief operating officer Craig Johnstone toldScrip.

He said the move was consistent with Evotecs two-pronged drug discovery strategy, which uses the Hamburg-based groups so-called Execute segment allying with external pharma partners which in turn supports reinvestment into Evotecs internal pipeline within its so-called Innovate segment.

This allows us to bring gene therapy project concepts into the Evotec Innovate pipeline at our choice and discretion. That was not possible before, so we can now use gene therapy to support our partners and also use it to support ourselves.

Johnstone said Evotec already makes broad use of CRISPR in its discovery and development activities. This will only amplify that aspect of Evotecs activities, he added.

Evotec Gene Therapy (Evotec GT) will start operations with a team of gene therapy experts at an R&D site in Orth an der Donau, Austria. Its scientists have deep expertise in vectorology and virology as well as disease insights, in particular in hemophilia, hematology, metabolic and muscle diseases.

We are recruiting the team, which will be composed of ex-Takeda employees. Its leadership has a long history in gene therapy stretching back years and to legacy organizationsBaxaltaandShire which were acquired by Takeda, but who were made redundant. Well be bringing them all on board over the next eight weeks, Johnstone said. Friedrich Scheiflinger, previously head of drug discovery for Takeda in Austria, will head up the new gene therapy unit.

No financial details were disclosed about Evotecs latest collaboration with Takeda.

Its a fairly straight-forwardcontractual framework that has been agreed between Evotec and Takeda, Johnstone said, without elaborating.

Sten Stovall is a London-based editor and writer with 40 years of experience in the field of journalism, including more than 20 years with Reuters and eight years with The Wall Street Journal/Dow Jones Newswires. He can be reached at sten.stovall@informa.com

Originally posted here:
Evotec extends Takeda pact to gene therapy - Bioprocess Insider - BioProcess Insider

REDWOOD CITY, Calif., April 20, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced the appointment of Scott Whitcup, M.D. to the Companys Board of Directors.

We are delighted to welcomeScott to our board, said Patrick Machado, J.D., Board Chair of Adverum Biotechnologies. Scott's expertise and accomplished track record as an ophthalmologist and successful drug developer fit perfectly with Adverum's mission to substantially elevate the standard of clinical care for patients suffering from neovascular AMD and other vision-threatening indications. My colleagues and I all look forward to working with Scott to realize fully the significant potential benefits our technology offers patients at risk of losing their sight.

This is an exciting time to join Adverums Board as the company strategically executes on expanding its pipeline through its novel vector discovery and drug development expertise to commercialize gene therapies to treat patients with serious ocular and rare diseases, said Dr. Whitcup. The development progress of ADVM-022, including the promising clinical data demonstrated to date in the OPTIC trial, has been impressive. I look forward to partnering with the Board, and the Adverum management team, on further developing the pipeline of drug candidates and advancing ADVM-022 towards commercialization for patients with wet AMD and diabetic retinopathy.

Scott Whitcup, M.D. is the founder and chief executive officer of Akrivista and Whitecap Biosciences, two companies focused on developing new therapies in ophthalmology and dermatology. In addition, he is on the clinical faculty at the UCLA Stein Eye Institute. Previously, Dr. Whitcup was the executive vice president of research and development and chief scientific officer at Allergan, where he led the discovery, clinical development, and medical affairs organizations focused on therapeutic areas including ophthalmology, CNS, urology, dermatology, and medical aesthetics. Earlier at Allergan, he served as vice president and head, ophthalmology therapeutic area, where he secured regulatory approvals for Alphagan P, Lumigan, Restasis, and Ozurdex. Earlier in his career, Dr. Whitcup was the clinical director at the National Eye Institute at the National Institutes of Health (NIH). Dr. Whitcup earned a B.A. from Cornell University and an M.D. from Cornell University Medical College. He completed an internal medicine residency at UCLA and an ophthalmology residency at Harvard University at the Massachusetts Eye and Ear Infirmary.

Dr. Whitcup serves on the board of directors of Scilex Pharmaceuticals and Anivive Lifesciences.

About Adverum BiotechnologiesAdverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of its lead indication, wet age-related macular degeneration. For more information, please visit http://www.adverum.com.

Investor and Media Inquiries:Investors:Myesha LacyAdverum Biotechnologies, Inc.mlacy@adverum.com1-650-304-3892

Media:Cherilyn Cecchini, M.D.LifeSci Communicationsccecchini@lifescicomms.com 1-646-876-5196

Excerpt from:
Adverum Biotechnologies Appoints Ophthalmology Industry Veteran Scott Whitcup, M.D. to Board of Directors - GlobeNewswire

LOS ANGELES--(BUSINESS WIRE)-- T-Cure Bioscience, Inc., a privately held company focused on developing autologous T cell receptor (TCR) therapy products for the treatment of solid tumors, today announced the appointment of seasoned biopharmaceutical industry executive James G. McArthur, Ph.D. to the Companys Board of Directors. Dr. McArthur brings more than 25 years of experience in drug development, financial, strategic and operational experience. Dr. McArthur has successfully founded five biotech companies and brings extensive knowledge of the healthcare space, having led companies through all phases of development from technology assessment through lead identification and proof of concept human studies.

James extensive knowledge of and experience with all aspects of drug development as well as his broad healthcare industry connections will be vital as we advance the clinical development of our TCR product candidates for the treatment of tumors expressing KK-LC-1 in collaboration with the National Cancer Institute, stated Gang Zeng, Ph.D., Chief Executive Officer of T-Cure. Dr. McArthur has previously established multiple successful biotechnology companies, led the R&D efforts to build out portfolios from the ground up, and found validating partnership opportunities with big pharma. This experience will be essential as T-Cure progresses its pipeline and increases its focus on product development efforts moving forward.

Dr. McArthur is a founder, a member of the Board of Directors and formerly served as CEO of Imara, Inc., a public clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of hemoglobin. He was also a founder of Vtesse, a company developing drugs for patients suffering from Niemann-Pick Type C1 Disease, which was subsequently acquired by Sucampo Pharmaceuticals, and most recently founded Tiburio, a clinical stage company focused on rare neuroendocrine disorders. Prior to co-founding Imara, Vtesse and Cydan, Dr. McArthur was an entrepreneur-in-residence at HealthCare Ventures. There, he helped to create and then served as Chief Scientific Officer of Synovex Co., a rheumatoid arthritis therapeutic company which was subsequently acquired by Roche. He has extensive research and development expertise in a number of areas including rare diseases, oncology, hematologic diseases, vascular diseases as well as gene therapy. Dr. McArthur is a listed investor on 15 issued patents and multiple patent applications. He previously served as a member of the Board of Directors of Nightstar Therapeutics, a clinical stage gene therapy company which was acquired by Biogen. Dr. McArthur obtained a PhD in biochemistry and oncology at McGill University and was a post-doctoral fellow studying Immunology at Massachusetts Institute of Technology and the University of California at Berkeley with Noble laureate James Allison.

About T-Cure Bioscience, Inc.

T-Cure Bioscience, Inc. is an innovative immuno-oncology company committed to delivering effective and durable cell therapies for the treatment of cancer. The Company focuses on isolating high avidity TCR that can be used to engineer a patients T cells to effectively target and destroy solid tumors. The Company maintains a pipeline of TCR under clinical and pre-clinical development targeting HERV-E, KK-LC-1, NY-ESO-1 and other antigens associated with solid tumors with significant unmet medical needs, including kidney cancer, breast cancer, lung cancer, gastric cancer, ovarian cancer, liver cancer, pancreatic cancer, sarcoma, and glioblastoma.

More information is available at http://www.T-Cure.com.

Forward Looking Statements

T-Cure cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industrys actual results, levels or activity, performance or achievements to be materially different from those anticipated by such statements. The use of words such as may, might, will, should, could, expect, plan, anticipate, believe, estimate, project, intend, future, potential or continue, and other similar expressions are intended to identify forward looking statements. For example, all statements we make regarding (i) the initiation, timing, cost, progress and results of our preclinical and clinical studies and our research and development programs, (ii) our ability to advance product candidates into, and successfully complete, clinical studies, (iii) the timing or likelihood of regulatory filings and approvals, (iv) our ability to develop, manufacture and commercialize our product candidates and to improve the manufacturing process, (v) the rate and degree of market acceptance of our product candidates, (vi) the size and growth potential of the markets for our product candidates and our ability to serve those markets, and (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates, are forward looking. All forward-looking statements are based on current estimates, assumptions and expectations by our management that, although we believe 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 we expected. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200420005106/en/

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T-Cure Bioscience Announces Appointment of Seasoned Industry Executive James G. McArthur, Ph.D. to Board of Directors - BioSpace

Global Blood Therapeutics reports Q4 earnings, summarizes upcoming catalysts

Global Blood Therapeutics' (GBT) novel sickle cell disease drug, Oxbryta, has officially hit the market, netting a mere $2M in revenue. Meanwhile, R&D costs have nearly doubled in the last year. SG&A costs have nearly tripled! Such is expected when launching a drug, but the question is always, "to what extent is it tolerable?"

Global Blood hopes, following a non-dilutive loan, to be cash flow positive before having to resort back to dilution again:

Entered into a $150 million loan agreement with funds managed by Pharmakon Advisors LP, and drew down on the first $75 million of the non-dilutive loan. The proceeds will be used to advance the discovery and development of potential novel treatments for SCD and other grievous blood-based conditions without diverting financial resources from the launch of Oxbryta. GBT believes that the proceeds from this loan, in conjunction with existing cash and investments, have the potential to provide the necessary runway for the company to achieve positive cash flow while enabling the continued advancement of clinical development programs and other earlier-stage product candidates.

Source: GBT

The company has nearly $700M in cash and investments. This can be expected to get them through mid-2021, even with an additional $75M pending from the loan. I highly doubt the company will be cash flow positive before 2022 and suspect Global Blood will have to dilute once more. This will hopefully occur at a more advantageous pricing and following good news (e.g. successful confirmatory trial, strong sales ramp).

On the clinical front, GBT continues to enroll patients into the confirmatory TCD study.

Source: GBT March '20 corporate slides

Success would likely expand the label to children under the age of 12 as well. The confirmatory trial would also serve to prove its clinical benefit, which Oxbryta has yet to do. Accelerated approval was granted because Oxbryta is theoretically likely to procure a clinical benefit. If the confirmatory trial is to fail, Oxbryta would be pulled from the market and one would have to ask if the drug is a viable one at all. However, I propose success in the TCD trial is likely:

Source: GBT Nov. '19 corporate slides

Remaining catalysts are summarized below:

Source: GBT March '20 corporate slides

Global Blood is also involved with a few non-pivotal trials that aim to shed light on their therapeutic:

Despite achieving accelerated approval and grabbing nearly $90/share in a bull market, major catalysts and upside remain for Global Blood.

While I am not super positive about Oxbryta's revenue prospects prior to full approval, I do expect major clinical developments over the next several months. Such clinical developments could help shed light on the clinical effects of Oxbryta beyond hemoglobin improvement. Global Blood Therapeutics remains a conviction idea as I believe Oxbryta is a game-changer in sickle cell disease due to its ability to improve hemoglobin levels without a worsening of blood viscosity. A ~$3.5B enterprise valuation for Global Blood does not yet fully consider its potential in sickle cell.

Risks include, but are not limited to: Oxbryta confirmatory trial failure, dilution, Oxbryta safety and/or efficacy issues, poor Oxbryta market performance, competition (e.g. gene therapy), and US recession.

To expand on competitive risks, I do not believe gene therapy will serve as a major competitor to Oxbryta within the drug's relevant market time (patent expires in several years). As I've touched upon in previous articles, sickle cell patients are weary of curative therapies and gene therapy would be very costly and for only a select few. Furthermore, I am not aware of any additional drugs in development that share the potentially disease-modifying mechanism of action that Oxbryta bears.

I present and update my best ideas & biotech research only to subscribers of my exclusive marketplace, The Formula. I also maintain a model portfolio of my top biotech ideas whichoutperformed the market (XBI, IBB, SPY) by over 16% in 2019!

Try a free, no-risk 2-week trial today by clicking the flask below!

Disclosure: I am/we are long GBT. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: The intention of this article is to provide insight, not investment advice. While the information provided in this article is intended to be factual, there is no guarantee and prospect investors are encouraged to do their own fact-checking and research before investing in a company. One must also consider one's own financial standings, risk tolerance, portfolio diversification, etc. before making a decision to buy shares in a company. Many of my articles detail biotechnology companies with little or no revenue. These stocks are, therefore, speculative and volatile. Even when prospects seem promising, there is no predicting the future. Losses incurred may be significant.

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Global Blood Therapeutics: Key Catalysts Remain As The Search For Clinical Benefit Continues - Seeking Alpha

Competitive landscape section of this Gene Therapy Products Mreport covers strategic profiling of key players in the market, comprehensively analyzing their core competencies, and strategies. The global market document compiles exhaustive information acquired through proven research methodologies and from dedicated sources across several industries. Gene Therapy Products market report puts forth an absolute overview of the market that contains various aspects of market analysis, product definition, market segmentation, key developments, and existing vendor landscape. This Gene Therapy Products market document also includes geographical markets and key players that have adopted significant strategies for business developments.

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.

Key questions answered in the report :-

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Gene Therapy Products Market Shows Evolving Opportunities in 2020. Major Players are Adverum, agtc, Arbutus Biopharma, Audentes Therapeutics, AveXis...

At the front lineof advances in personalized medicine are cell and gene therapies. These are two overlapping fields of medical research whereby the overall aim is to treat the underlying causes of both genetic and acquired diseases.

Is the promise of personalized medicine on the edge of being delivered? Decades of research and advances in genomics, cell biology, cancer and analytical technologies have permitted exciting progress in the cell and gene therapy space recently. Technology Networks recently spoke with three of the leaders in this space, Allogene Therapeutics, bluebird bioInc. andMogrify to gain their insights on the next developments in cell and gene therapy.

Joe Foster, COO, Mogrify.

"We are on the cusp of new breakthroughs in this field and that evolution of engineered cell therapy has the potential to expand beyond cancer. Engineering, synthetic biology and gene editing has opened the door beyond allogeneic cell therapy. There is a bright future with transformative technologies that may hold the key to addressing solid tumors."

David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder, Allogene Therapeutics.

"Although gene therapies are still a nascent technology, they present a new paradigm for healthcare, offering a one-time treatment that can address the underlying genetic cause of certain severe genetic diseases and cancer. We anticipate that the three techniques being studied for gene therapy gene editing, gene addition and gene-based immunotherapy will become more distinct and that the entire treatment journey will become more efficient."

Martin Butzal, Head of Medical Europe, bluebird bio.

Read more here:
Looking to the Future of Cell and Gene Therapies - Technology Networks

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.

Story continues

To request a report overview of 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/

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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View original content:http://www.prnewswire.com/news-releases/visiongain-report-the-gene-therapy-market-is-projected-to-grow-at-a-cagr-of-32-in-the-first-half-of-the-forecast-period-301040367.html

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

A report on global Gene Therapy market has hit stands. This study is based on different aspects like segments, growth rate, revenue, leading players, regions, and forecast. The overall market is getting bigger at an increased pace due to the invention of the new dynamism, which is making rapid progress.

The given report is an excellent research study specially compiled to provide latest insights into critical aspects of the Global 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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Conclusively, this report will provide you a clear view of each and every fact of the market without a need to refer to any other research report or a data source. Our report will provide you with all the facts about the past, present, and future of the concerned Market.

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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

For more information about this report visit https://www.researchandmarkets.com/r/k4398p

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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.

Key questions answered in the report :-

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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.

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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