StemCell Therapy

BOSTON, Sept. 15, 2020 /PRNewswire/ --GreenLight Bioscienceshas received a $3.3 million grant from the Bill & Melinda Gates Foundation to develop new mRNA-based gene therapies for Sickle Cell Disease and other global health challenges.

The funding will support GreenLight's research and testing of affordable therapies using the company's novel messenger RNA (mRNA) approach to gene editing. mRNA technology is already being used to develop vaccine candidates for infectious diseases, including the COVID-19 pandemic.

While initial research will focus on a cure for Sickle Cell Disease, GreenLight plans to develop a versatile gene editing platform to address a variety of diseases affecting underserved patient populations, such as treating HIV in developing countries.

Sickle Cell Disease is a group of inherited blood disorders in which red blood cells develop abnormally, causing pain and anemia. More than 4 million people currently suffer from the disease, with another 40+ million having the sickle cell trait, which can be passed on to future generations. The disease primarily targets people of African, Hispanic, or Middle Eastern descent. Current treatment regimens including blood transfusions and bone marrow transplants are costly, invasive, and impractical for treating large segments of affected patient populations.

"Funders are recognizing the potential of our innovative approach to gene editing that, in combination with our proprietary RNA manufacturing capability, has the potential to deliver accessible gene therapies and improve human health globally," said Marta Ortega-Valle, senior vice president of Human Health and Corporate Development at GreenLight Biosciences. "Finding a safe and effective therapy is critical, but equally important is the ability to produce it affordably for broader access. We are grateful for the Gates Foundation's support to advance novel gene editing approaches for populations in which those therapies are currently out of reach."

Gene editing therapies hold significant promise in the treatment of Sickle Cell Disease since it is a disorder caused by gene mutation. Using RNA as its core, GreenLight Biosciences is working to develop an in vivo gene therapy that could ultimately offer a cure to the disease.

Once the therapy candidate is validated and moves into clinical use, GreenLight Biosciences' biomanufacturing platform will accelerate production of affordable treatments at scale. "Manufacturing sufficient quantities of high-quality RNA at an accessible cost is critical for achieving the full potential of new therapies that aim to reach a global patient population. That capability does not yet exist in the market, but GreenLight's end-to-end, self-contained manufacturing platform aims to make that possible for all mRNA-based therapies and vaccines," Ortega-Valle added.

About GreenLight Biosciences, Inc.GreenLight is a bio-performance company with a unique, cell-free production platform that delivers high-performing RNA solutions to human, plant and animal challenges. GreenLight develops RNA products for plant and life science applications, and collaborates with industry leaders to advance vaccine development, pandemic preparation, crop management, and plant protection. The cutting-edge, natural platform delivers higher-quality RNA at a lower cost and higher speed than was ever before possible. The GreenLight team values diversity, inclusion, and equality and promises to use collaboration to remain scientifically imaginative and passionately focused on making a difference in the world. For more information, visithttps://www.greenlightbiosciences.com/.

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GreenLight Biosciences Receives $3.3 Million Grant to Develop Sickle Cell Disease Cure Using mRNA Gene Therapy - PRNewswire

JOHNSON CITY, Tenn., Sept. 10, 2020 /PRNewswire/ --LabConnect, the preeminent provider of clinical trial central lab services, today announced the expansion of its Johnson City facility to support its significant growth in cell and gene therapy and immuno-oncology studies. The company,which recentlyrelocated its headquarters to Tennessee, is doubling the capacity of its biorepository for sample storage and its clinical trial kit building capacity. LabConnect welcomed Tennessee Governor Bill Lee, Congressman Phil Roe, Economic Development Commissioner Bob Rolfe, elected officials, and other dignitaries to celebrate this milestone in the company's growth.

"While we have all had to adjust and adapt during these unprecedented circumstances, Tennessee's business climate has remained strong and welcoming to companies around the globe," said Gov. Lee. "I applaud LabConnect for continuing to invest and create jobs in our state and for choosing to bring its headquarters to Tennessee. I look forward to the many great things that will come from this facility in Johnson City."

"We are pleased that another company has chosen to expand its presence in Tennessee," Commissioner Rolfe said. "LabConnect is committed to leading the way in central laboratory services, which will have a global impact from its Tennessee=based facility. We appreciate LabConnect for its continued innovation and for creating high quality jobs."

"We are excited that the incentives with the State, Tennessee Valley Authority, and Northeast Tennessee Regional Economic Partnership have enabled LabConnect to expand its operations," said Tom Sellig, LabConnect CEO. "Our location offers several advantages which has allowed us to provide unique services to pharmaceutical and biotech clients. We are currently serving 200 leading biopharmaceutical clients and proud of the more than 20 products we have supported that are now FDA approved and used to treat patients around the world. The expanded capacity will allow us to scale our organization to meet LabConnect's growing demand for our clients' cell & gene therapy, rare and orphan diseases, and immuno-oncology projects."

For more information, visit http://www.labconnect.com.

About LabConnectConnect with LabConnectthe preeminent provider of central laboratory support services for analytically and logistically complex studies such as immuno-oncology, cell and gene therapies, and rare & orphan diseases. The company offers unique and innovative services that have been specifically designed to meet the exacting demands of today's clinical trials. The worldwide scope of services includes routine and specialized testing, real-time sample tracking, data integration, biorepository, sample processing and specialized functional outsourcing. Leading the evolution in central laboratory services since 2002, LabConnect's services are customized to fit the unique needs of biopharmaceutical clients. Get connected by requesting a proposal at http://www.labconnect.com or via email at [emailprotected].

SOURCE LabConnect

http://www.labconnect.com

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LabConnect Announces Expansion to Support Cell & Gene Therapy Growth - PRNewswire

New Jersey contract manufacturer Catalent has been right in the mix in the COVID-19 response effort, signing pacts to help produce frontrunners in the vaccine hunt. Now, the company is fleshing out a Maryland facility to aid in that effortand position Catalent's cell and gene therapy offerings well into the future.

Catalent will infuse $130 million into its cell and gene therapy manufacturing facility in Harmans, Maryland, to broaden the CDMO's late-stage production capacity, the company said Wednesday.

The newest investment will add five late-stage clinical and commercial manufacturing suites to the Harmans site, expected to go online in the first half of 2022, Catalent said.

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That expansion will bring the total number of manufacturing suites to 15 at the planned 350,000-square-foot complexnear the Baltimore/Washington International airport. The Harmans facility recently received FDA approval for commercial production, and its initial 10 manufacturing suites are set to be fully operational by the first quarter of 2021.

The five new suites will be located in a second building at the site that will also house cold-storage warehousing and added office space, Catalent said. The Harmans complex is one of five Maryland sites for Catalent's cell and gene therapy manufacturing portfolio.

Adding capacity at its Harmans site is a future play for Catalent in the bustling cell and gene therapy space, but the facility could also benefit the CDMO's immediate COVID-19 response efforts.

RELATED:AstraZeneca ropes in Catalent gene therapy site to produce viral vectors for COVID-19 vaccine

Last month, British drugmaker AstraZeneca tappedCatalent to help produce bulk drug substance and viral vectors at the Harmans facility for the University of Oxford's adenovirus-based COVID-19 vaccine.

Catalent will start production there this quarter,buildingon its previous pact with AstraZeneca for fill-finish and packaging duties at its Anagni, Italy, site.

Catalent's work will include production of viral vectors for a genetically modified form of the adenovirus used in Oxford's shot, dubbed AZD1222. The modified virus, known as ChAdOx1 nCoV-19, aims to induce a lasting immune response to spike proteins added to the virus's surface.

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Catalent injects $130M into Maryland cell and gene therapy site drafted into COVID-19 vaccine hunt - FiercePharma

Sarepta has gone all-in on gene therapy over the last few years, racing with Pfizer and Solid Biosciences to be the first to develop a genetic fix for Duchenne muscular dystrophy, one of the most common rare diseases.

Sarepta has been comfortably in the lead, collecting the first positiveresults and snaring a $1.15 billion cash commercialization deal with Roche, but this week the company hit a snag. Late yesterday, Sarepta provided a program update for its gene therapy, revealing that in a scheduled meeting the FDA had raised concerns about the kinds of tests they would use to measure potency in the pivotal study and commercial supply for the gene therapy. The company has assays that might fit the criteria, they said, but needed additional dialogue with the agency to confirm.

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FDA knocks back Sarepta in Duchenne gene therapy race with Pfizer, but analysts urge caution - Endpoints News

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

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

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

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

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

Sarepta Forward-Looking Statements

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding Sareptas belief that its existing assays and data could be employed in response to OTATs request; the acceptability of Sareptas potency assay approach by the FDA; our plan to work with OTAT to potentially satisfy their requests and to obtain clarity on the timing of the commencement of our commercial supply study; and the potential of SRP-9001 to be a transformative therapy for DMD patients.

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

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

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

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

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

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

Collaboration partners,Neurocrine Biosciences (NASDAQ:NBIX) and Voyager Therapeutics (NASDAQ:VYGR) announced data from an early stage clinical trial with Parkinson's disease patients and NBIb-1817, an experimental gene therapy. Three years after a single administration, 14 out of 15 patients are still reporting motor function improvements.

At the moment, NBIb-1817 is in the middle of a phase 2 study that was put on hold in April due to the COVID-19 pandemic. If allowed to restart, the phase 2 RESTORE-1 trial will randomize patients to receive NBIb-1817 or a placebo, then measure for a change in "On" time without troublesome dyskinesia.

Image source: Getty Images.

This potential new treatment option uses a viral vector to deliver a gene encoding an enzyme that helps Parkinson's disease patients convert levodopa into the dopamine they need.

Administering NBIb-1817 requires magnetic resonance imaging (MRI) to guide a pair of infusions that deliver the therapy directly into the striatum, a structure deep in the center of the brain. Needles through the skull aren't anybody's idea of a good time, but the side effects that come with daily doses of levodopa aren't any fun either.

For patients with severe Parkinson's disease that has a diminishing response to levodopa, the temporary discomfort that comes with NBIb-1817 treatment seems like a trade-off most will be willing to accept. Three years after a single treatment with NBIb-1817, patients were able to reduce their daily levodopa doses from a baseline of 1500.9 milligrams per day to 1061.4 milligrams per day. Despite reducing their levadopa intake, patients receiving three different dosage strengths of NBIb-1817 improved average "On" time by up to 2.23 hours.

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Parkinson's Disease Patients Get Long-Term Benefits From Experimental Gene Therapy - The Motley Fool

CAMBRIDGE, Mass., Sept. 15, 2020 /PRNewswire/ --Obsidian Therapeutics, Inc., a biotechnology company pioneering controllable cell and gene therapies, today announced that Bristol Myers Squibb (NYSE:BMY) has exercised its option to an exclusive worldwide license to a cell therapy candidate based on Obsidian's cytoDRiVE technology for the controlled expression of the immunomodulatory factor CD40L. This announcement marks the first opt-in decision by Bristol Myers Squibbsince the companies announced their collaboration to develop novel cell therapies in January 2019. Under the terms of the agreement, Obsidian is eligible to receive potential future milestone and royalty payments.

"We are very interested in exploring innovative approaches to developing engineered cell therapies, including the cytoDRiVE platform," said Rupert Vessey, D. Phil., Executive Vice President, Research and Early Development, Bristol Myers Squibb. "By controllingthe expression of armed payloads like CD40L, Obsidian's cell therapy candidates may have the potential to overcome tumor microenvironment resistance and unlock the power of cell therapy in solid tumors and other malignancies."

"This announcement marks an important milestone validating Obsidian's cytoDRiVE platform, and we look forward to continuing to work with Bristol Myers Squibbto bring powerful new immunotherapies to patients," said Paul K. Wotton, Ph.D., Chief Executive Officer of Obsidian Therapeutics. "We are also pleased with the pace with which our own pipeline programs are progressing as we continue to advance our lead controllable tumor infiltrating lymphocyte (TIL) therapy to the clinic."

About Obsidian TherapeuticsObsidian Therapeutics is a biotechnology company pioneering controllable cell and gene therapies to deliver transformative outcomes for patients with intractable diseases. Obsidian's proprietary cytoDRiVE technology provides a way to control protein degradation using FDA-approved small molecules, permitting precise control of the timing and level of protein expression. The cytoDRiVE platform can be applied to design controllable intracellular, membrane and secreted proteins for cell and gene therapies as well as other applications. The Company's initial applications focus on developing novel cell therapies for the treatment of cancer. Obsidian is headquartered in Cambridge, Mass. For more information, please visit http://www.obsidiantx.com.

Media Contact:

Maggie BellerRusso Partners, LLC[emailprotected]646-942-5631

SOURCE Obsidian Therapeutics

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Obsidian Therapeutics Announces Bristol Myers Squibb Opt-In of cytoDRiVE Cell Therapy Candidate - PRNewswire

Pfizer has tipped its pipeline to deliver new products that add more than $15 billion to revenues by 2025. The anticipated pipeline contributions led Pfizer to predict it will weather the next wave of patent expirations that is set to start in 2026.

At its virtual investor day Tuesday, Pfizer predicted (PDF) that JAK1 inhibitor abrocitinib, antisense therapy vupanorsen and a 20-valent pneumococcal conjugate vaccine can generate peak annual revenues of more than $3 billion each. Pfizer also tipped its Duchenne muscular dystrophy gene therapy to rake in an excess of $2 billion and identified six other potential blockbusters.

With Pfizer tipping another five pipeline prospects to generate revenues of $500 million to $1 billion, the Big Pharma said sales of as-yet-unapproved experimental products could come to $15 billion by 2025.

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The peak years of the experimental medicines likely lie beyond 2025. The timing of the anticipated peaks of those products coincides with the years in which Pfizer expects to face another patent cliff.

In 2026 and 2027, the U.S. basic product patents on Prevnar 13, Eliquis and Xtandi are set to expire, potentially exposing products that generated sales of $6.3 billion in Pfizers home market last year to off-patent competition. All told, analysts expect Pfizer to lose up to $20 billion in sales due to patent expirations starting in 2026. Pfizers internal forecast is roughly in line with analyst estimates.

After Lyrica went off patent last year, Pfizer entered a window in which its portfolio is virtually free from the loss of exclusivity through to 2026. The lull gives Pfizer a period to get some of its pipeline prospects to market and start growing their sales, leading management to predict the current slate of experimental assets will at least replace the revenues lost to off-patent rivals starting in 2026.

That prediction rests on Pfizers ability to get key pipeline products to market. Pfizer used the virtual event to claim a growing effectiveness in that regard. As of 2015, 5% of the drugs Pfizer took into the clinic went on to win approval. By 2019, that figure had increased to 9%, causing Pfizers success rate to go from well below the industry average to slightly above the performance of its peers.

Pfizer also noted an improved phase 2 success rate. In 2017, 17% of Pfizers phase 2 trials succeeded. Since then, Pfizer has averaged 46%. The success rate so far this year stands at 53%. Pfizer claims to be moving faster, too, stating it is on course to reduce development timelines by 2.5 years between 2017 and 2021. Automation of processes accounts for the biggest anticipated time saving.

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Pfizer tips pipeline to add $15B to sales in coming years - FierceBiotech

TEL AVIV, Israel, Sept. 15, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT) will present today a corporate overview, including an update on the OVAL pivotal study, at the H. C. Wainwright 22nd Annual Global Investment Conference being held virtually on September 14-16, 2020.

"Our OVAL trial of VB-111 in ovarian cancer continues to progress well, with over a third of the study participants already enrolled," said Dror Harats, MD, Chief Executive Officer of VBL Therapeutics. "We are pleased that the high response rate seen in our interim analysis in March, continues to be high in the total patient population to date. With blinded data becoming more mature, we currently see a good correlation between the CA-125 and RECIST responses, as well as with preliminary PFS and OS data. So far, OVAL blinded data recapitulate what we have seen in our positive Phase 2 study, which is very encouraging."

Presentation Details:

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

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

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

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VBL Therapeutics to Provide an Update on the OVAL Study Today at the H. C. Wainwright 22nd Annual Global Investment Conference - GlobeNewswire

As the global COVID-19 situation is rapidly changing, staying abreast with the latest news can be challenging. In this article, Sheraz Gul provides an overview of the broad range of potential treatments in development and discusses how the regulatory landscape can shift at any time.

IN EARLY AUGUST 2020, the World Health Organization (WHO) reported the number of confirmed cases of COVID-19 has exceeded 20 million including over 700,000 deaths. These staggering figures have come about despite global lockdown measures being in place for the past few months. The resulting flattening of confirmed cases and deaths is now being jeopardised because, as the lockdown measures are being relaxed, we are seeing a resurgence of cases in some locations. In order to prevent a possible second wave of COVID-19 cases and deaths, the wearing of masks in public places is now mandatory in many countries. At the start of August 2020, New Zealand, which had gone 102 days without recording a locally transmitted case of COVID-19, saw new cases emerging. This highlights that we are still vulnerable to the spread of the virus.

Thus far, no US Food and Drug Administration (FDA)-approved therapy for use to treat COVID-19 is available. In order to provide some respite, on 1 May 2020 the FDA, as part of its Coronavirus Treatment Acceleration Program (CTAP), issued an Emergency Use Authorization (EUA) for the investigational antiviral drug remdesivir to treat suspected or laboratory-confirmed COVID-19 in adults and children hospitalised with severe disease. Although there is limited information known about the safety and effectiveness of using remdesivir to treat people in the hospital with COVID-19, this investigational drug has been shown in a clinical trial to shorten the time to recovery in some patients. It is noteworthy that prior to this, on 28 March 2020, the FDA also issued an EUA for chloroquine and hydroxychloroquine. This was subsequently revoked on 15 June 2020, as the FDA considered that chloroquine and hydroxychloroquine no longer met the statutory criteria for the EUA. In conclusion, the use and revocation of COVID-19 drugs can change at any time, so referring to official guidance is essential.

A recent update of the US ClinicalTrials. gov website listed around 2,000 COVID-19 interventional studies that are underway, around 90 of which are now completed. These cover a broad range of potential treatments including: 1) antiviral drugs to prevent viruses from multiplying; 2) immunomodulators aimed at reducing the bodys own immune reaction to the virus; 3) neutralising antibody therapies to fight the virus (manufactured, animalsourced and blood-derived from people who have previously had COVID-19); 4) cell therapy products (cellular immunotherapies and other types of both autologous and allogeneic cells, such as stem cells, and related products); and 5) gene therapy products that modify or manipulate the expression of a gene or alter the biological properties of living cells for therapeutic use. This diversity of therapeutic approaches is important as it allows opportunities for all types of treatments to be discovered, whilst expanding our understanding of the disease. Bearing in mind the timescale to discover a therapy for any given disease, the scientific community has made major advances towards developing a COVID-19 therapy. We eagerly await the output of various clinical trials over the next few months with the expectation that some of these yield sufficiently efficacious and safe therapies that gain approval by the regulators.

As the global COVID-19 situation is rapidly changing, it is advisable to stay abreast with the latest news only from reputable sources (see suggested reading below).

Sheraz Gul is an expert in drug discovery with experience gained in academia (University of London), industry (GlaxoSmithKline Pharmaceuticals) and the largest applied research organisation in Europe (Fraunhofer Institute). He is also an adjunct lecturer at NUI-Galway, Ireland and scientific co-founder of Transcriptogen Ltd. He has co-ordinated work packages in drug discovery projects, which have attracted more than 7 million funding and has organised 42 drug discovery workshops since 2011 across the globe and trained 780 scientists

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COVID-19 therapies in the pipeline - Drug Target Review

-Parkinsons Disease R&D Day to be hosted by Gavin Corcoran, MD, Chief R&D Officer, on October 30th, 2020, with leading movement disorder specialists

NEW YORK and BASEL, Switzerland, Sept. 08, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that the company will provide a corporate overview and participate in 1:1 meetings at upcoming virtual investor and patient conferences in September. Additionally, the company announced plans for their 1st Parkinsons Disease R&D Day with key opinion leaders.

Investor and Patient Conference Presentation details:

A live webcast of the presentations will be available in the Events section of Axovant's website at http://www.axovant.com. A replay will be available for approximately 30 days following the conference.

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 target GM1 gangliosidosis, GM2 gangliosidosis (also known as 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.

Contacts:

Media & Investors

Parag MeswaniAxovant Gene Therapies Ltd.(212) 547-2523media@axovant.cominvestors@axovant.com

Source:Axovant Gene Therapies

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Axovant Announces First Parkinson's Disease Gene Therapy R&D Day and Participation in Upcoming September Conferences" - GlobeNewswire

One of the major challenges facing gene therapy a way to treat disease by replacing a patients defective genes with healthy ones is that it is difficult to safely deliver therapeutic genes to patients without the immune system destroying the gene, and the vehicle carrying it, which can trigger life-threatening widespread inflammation.

Three decades ago researchers thought that gene therapy would be the ultimate treatment for genetically inherited diseases like hemophilia, sickle cell anemia and genetic diseases of metabolism. But the technology couldnt dodge the immune response.

Since then, researchers have been looking for ways to perfect the technology and control immune responses to the gene or the vehicle. However, many of the strategies tested so far have not been completely successful in overcoming this hurdle.

Drugs that suppress the whole immune system, such as steroids, have been used to dampen the immune response when administering gene therapy. But its difficult to control when and where steroids work in the body, and they create unwanted side effects. My colleague Mo Ebrahimkhani and I wanted to tackle gene therapy with immune-suppressing tools that were easier to control.

I am a medical doctor and synthetic biologist interested in gene therapy because six years ago my father was diagnosed with pancreatic cancer. Pancreatic cancer is one of the deadliest forms of cancer, and the current available therapeutics usually fail to save patients. As a result, novel treatments such as gene therapy might be the only hope.

Yet, many gene therapies fail because patients either already have pre-existing immunity to the vehicle used to introduce the gene or develop one in the course of therapy. This problem has plagued the field for decades, preventing the widespread application of the technology.

Traditionally scientists use viruses from which dangerous disease-causing genes have been removed as vehicles to transport new genes to specific organs. These genes then produce a product that can compensate for the faulty genes that are inherited genetically. This is how gene therapy works.

Though there have been examples showing that gene therapy was helpful in some genetic diseases, they are still not perfect. Sometimes, a faulty gene is so big that you cant simply fit the healthy replacement in the viruses commonly used in gene therapy.

Another problem is that when the immune system sees a virus, it assumes that it is a disease-causing pathogen and launches an attack to fight it off by producing antibodies and immune response just as happens when people catch any other infectious viruses, like SARS-CoV-2 or the common cold.

Recently, though, with the rise of a gene editing technology called CRISPR, scientists can do gene therapy differently.

CRISPR can be used in many ways. In its primary role, it acts like a genetic surgeon with a sharp scalpel, enabling scientists to find a genetic defect and correct it within the native genome in desired cells of the organism. It can also repair more than one gene at a time.

Scientists can also use CRISPR to turn off a gene for a short period of time and then turn it back on, or vice versa, without permanently changing the letters of DNA that makes up or genome. This means that researchers like me can leverage CRISPR technology to revolutionise gene therapies in the coming decades.

But to use CRISPR for either of these functions, it still needs to be packaged into a virus to get it into the body. So some challenges, such as preventing the immune response to the gene therapy viruses, still need to be solved for CRISPR-based gene therapies.

Being trained as a synthetic biologist, I teamed up with Ebrahimkhani to use CRISPR to test whether we could shut down a gene that is responsible for immune response that destroys the gene therapy viruses. Then we investigated whether lowering the activity of the gene, and dulling the immune response, would allow the gene therapy viruses to be more effective.

CRISPR can precisely remove even single units of DNA. KEITH CHAMBERS/SCIENCE PHOTO LIBRARY/Getty Images

A gene called Myd88 is a key gene in the immune system and controls the response to bacteria and viruses, including the common gene therapy viruses. We decided to temporarily turn off this gene in the whole body of lab animals.

We injected animals with a collection of the CRISPR molecules that targeted the Myd88 gene and looked to see whether this reduced the quantity of antibodies that were produced to specifically fight our gene therapy viruses. We were excited to see that the animals that received our treatment using CRISPR produced less antibody against the virus.

This prompted us to ask what happens if we give the animal a second dose of the gene therapy virus. Usually the immune response against a gene therapy virus prevents the therapy from being administered multiple times. Thats because after the first dose, the immune system has seen the virus, and on the second dose, antibodies swiftly attack and destroy the virus before it can deliver its cargo.

We saw that animals receiving more than one dose did not show an increase in antibodies against the virus. And, in some cases, the effect of gene therapy improved compared with the animals in which we had not paused the Myd88 gene.

We also did a number of other experiments that proved that tweaking the Myd88 gene can be useful in fighting off other sources of inflammation. That could be useful in diseases like sepsis and even COVID-19.

While we are now beginning to improve this strategy in terms of controlling the activity of the Myd88 gene. Our results, now published in Nature Cell Biology,provide a path forward to program our immune system during gene therapies and other inflammatory responses using the CRISPR technology.

Samira Kiani, Associate Professor of Pathology, University of Pittsburgh

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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CRISPR Can Help Combat the Troubling Immune Response Against Gene Therapy - Gizmodo Australia

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

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

Gene Therapy Market, By Product

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

Gene Therapy Market, By Indication

Neurological Diseases Cancer Hepatological Diseases Duchenne Muscular Dystrophy Other Indications

Gene Therapy Market, By Delivery Method

Ex vivo In vivo

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

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

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

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

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

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

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

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

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

Gene therapy is utilized for treating neurodegenerative disorders like Alzheimer, amyotrophic lateral sclerosis, and spinal muscular atrophy. Gene therapy is one of the key treatment kinds that will propel the market growth over the forecast period. Moreover, gene therapy also finds lucrative applications in precision medicine. In addition to this, a rise in the occurrence of cancer is prompting the demand to treat the disease through gene therapy.

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Based on the type, the market can be segregated into Germ Line Gene Therapy and Somatic Gene Therapy. In terms of vector type, the gene therapy industry can be divided into Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome. On the basis of therapy area, the market for gene therapy can be classified into Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others.

The key players included in this market are Advanced Cell & Gene Therapy, Audentes Therapeutics, Benitec Biopharma, Biogen, Blubird Bio, Inc., Bristol-Myers Squibb Company, CHIESI Farmaceutici SPA, Eurofins Scientific, Geneta Science, Genzyme Corporation, Gilead, GlaxoSmithKline PLC, Human Stem Cells institute, Novartis AG, Orchard Therapeutics, Pfizer Inc., Sangamo therapeutics, Spark therapeutics, and Voyager Therapeutics.

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Gene Therapy Market By Industry Type, By Brand And Major Players 2020-2027 - The Daily Chronicle

TEL AVIV, Israel, Sept. 08, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT) announced today the successful completion of a Type B pre-IND meeting with the U.S. Food and Drug Administration (FDA) regarding the Company's development plan for VB-601. VB-601 is the Companys lead anti-MOSPD2 antibody for immune-inflammatory indications, for which a briefing package for the pre-IND meeting was submitted in June. Based on the FDA's feedback, VBL plans to advance its IND-enabling activities for VB-601 as planned.

We are very pleased with the outcome of the pre-IND meeting with the FDA, said Dror Harats, M.D., CEO of VBL Therapeutics. VB-601 is a first-in-class product candidate that has a completely novel mechanism of action. It can block the ability of monocyte to migrate to inflammatory sites, no matter which molecules try to attract them in. Therefore, it is important that we have reached alignment with the FDA on the path forward for VB-601. With this green light, we plan to continue to advance VB-601, aiming to start a first-in-human study in the second half of 2021.

About VB-601VBL is advancing a drug development program that is exploring the potential of MOSPD2 (motile sperm domain-containing protein 2), a protein that VBL has identified as a key regulator of cell motility, as a therapeutic target for inflammatory diseases. VB-601 is a proprietary anti-MOSPD2 monoclonal antibody which has potential for treatment of multiple chronic inflammatory diseases.

About VBLVascular Biogenics Ltd., operating asVBL Therapeutics, is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class treatments for cancer. VBLs lead oncology product candidate, ofranergene obadenovec (VB-111), is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. It is conveniently administered as an IV infusion once every two months. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in a VBL-sponsored all comers phase 1 trial as well as in three VBL-sponsored tumor-specific phase 2 studies. Ofranergene obadenovec is currently being studied in a VBL-sponsored phase 3 potential registration trial for platinum-resistant ovarian cancer.

Forward Looking StatementsThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. These forward-looking statements may include, but are not limited to, statements regarding VBLs programs, including VB-601, including their pre-clinical and clinical development, therapeutic potential and clinical results. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals, the risk that historical clinical trial results may not be predictive of future trial results, that our financial resources do not last for as long as anticipated, and that we may not realize the expected benefits of our intellectual property protection. A further list and description of these risks, uncertainties and other risks can be found in our regulatory filings with theU.S. Securities and Exchange Commission, including in our annual report on Form 20-F for the year endedDecember 31, 2019, and subsequent filings with theSEC. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. VBL Therapeutics undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

INVESTOR CONTACT:

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

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VBL Therapeutics Announces Successful Completion of Pre-IND Meeting with FDA Regarding Proposed Clinical Development of VB-601 - BioSpace

Seeing a child debilitated by illness is never easy.

When doctors tell youthere is nothing they nor you can do to help ease yourbaby's suffering, well, parents who know that type of helplessness often find it hard to describe.

We were told to take my son home and love him, becausehe probably wouldnt live past his second birthday, said Pace father Todd Hamrick. But, were way past that birthday now.

Hamricks son, Alek, was diagnosed at six months old with spinal muscular atrophy and not expected to live long enough to toddle. ButAlek, now 3, has beaten the odds and outlived that initial, bleak prognosis.

His parents attribute much of his success to his doctor, Richard Finkel, who entered Alek into a clinical trial for what they believe has been a wonder drug for their little boy, Evrysdi, which was recently approved by the Food and Drug Administration.

Whats hard to even get around is that people even bothered to research it, Todd Hamrick said. Its just a small amount of the population that has SMA. Its not like researching a blood pressure medication.

Spinal muscular atrophy, or SMA,is a genetic disorder caused by a loss of nerve cells that effect human motor function.

Essentially, those afflicted by SMA are made weak. Their muscleswaste away. In many cases, eventually, a person loses their ability to walk, to eat andevento breathe, and they die.

Aleks mother, Iwona Hamrick, is a nurse at a local hospital and wellremembers the moment she heardher sons diagnosis.

It was unimaginable. His pregnancy was normal, she said. We did genetic testing and it was negative. Unfortunately, at that time, they were not screening for his disease. So, from a healthy baby to a dying baby, you know?

The parents felt they had to travel, in more ways than one, to find the places and help that they could for their Alek.

Todd Hamrick said that he and wife decided to move from Gulf Breeze to Pace after Aleks diagnosis after feeling ostracized by many of their former acquaintances. The parents felt like some people who they used to know were made uncomfortable by their son's illness.

The area is very in-the-dark when it comes to children with issues, Todd Hamrick said. If your kids are healthy and you'rehealthy, its a great area. But, it doesnt attract the greatest talent or best and biggest facilities or endowments.

So, Alek traveled with his family to meet his future doctor, Finkel, at the Nemours Children's Hospital in Orlando.

Alekwas first treated with a gene therapy drug, and he made some response with that, Finkel told the News Journal. But more recently, he started on a second drug, which seems to be having an enhanced effect, I must say.

Finkel, an expert in the field of pediatric neurologic disorders, left Florida in March for a position leading the new Center for Experimental Neurotherapeutics at St. Jude Childrens Research Hospital in Memphis, Tennessee. Buthe has continued to monitor Aleks progress via video.

Both of the drugs he has received, the gene therapy and this new drug, Evrysdi, are designed to increase a certain protein in Aleks body that is deficient because of his genetic disorder, he explained. But they do it in different ways. The potential advantage of his new drug, Evrysdi because its an oral drug; you take it by mouth it goes into the stomach and into the bloodstream, and from there, it goes to all the tissues of the body.

And, we think that there is an enhanced effect, becauseit gets into the muscle tissue, Finkel continued. "These are very, very, early daysin trying to make assessments, soI dont want to say that we can come to any kind of conclusions yet."

However the cutting-edge drug works doesnt matter to a mother, whos just glad that it isworking.

It gave us hope. Thats for sure, Iwona Hamrick said. Becausewe felt helpless.

Since Alek started his new treatment last November, his strength has increased tremendously.

His muscle tone got better, Iwona Hamrick said. He is much stronger in the upper body, so much so, he is pushing his little wheelchair.

Alek can now cruise around his Pace home in an extraordinarily lite-weight wheelchair designed by a Swedish inventor who alsohas a child with SMA.

Alek had a lot of trouble before starting this medication even pushing it, Iwona Hamrick said. Sonowhe is just rolling around the house. Also, he is barring more weight on his legs.

Recently, Alek has started to be able to walk in a pool a huge milestone for the toddler.

But some worries remain the same.

Every day, Alek must use a type of breathing machine.

Its a cough assist machine, Todd Hamrick explained. We use it two times a day, when hes healthy. Becausewhere we can just clear our throats when we cough, he doesnt have that strength no lung strength.

Alek attends physical therapy, aqua-therapy,hippotherapy, occupational therapy and speech therapy sessions every week to try and ensure he remains healthy and continues to properly develop.

My worries have changed a lot, Todd Hamrick said. I used to worry my child was going to die. Now, Im worrying about if other kids will bully him at school.

"But that'sa great worry to have," he continued. "Compared to how it use to be, getting picked on is a great thing to worry about.

Colin Warren-Hicks can be reached at colinwarrenhicks@pnj.com or 850-435-8680.

Read or Share this story: https://www.pnj.com/story/news/2020/09/08/pace-florida-toddler-spinal-muscular-atrophy-helped-drug-evrysdi/5681766002/

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Pace toddler wasn't supposed to survive. Thanks to wonder drug, parents say he's thriving - Pensacola News Journal

NEW YORK and CLEVELAND, Sept. 08, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the Company will participate in the following upcoming investor conferences.

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona’s clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company’s portfolio of AAV-based gene therapies also features ABO-201 for CLN3 disease. Abeona’s novel, next-generation AIM capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona’s fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit http://www.abeonatherapeutics.com.

Investor Contact: Greg Gin VP, Investor Relations Abeona Therapeutics +1 (646) 813-4709 ggin@abeonatherapeutics.com

Media Contact: Scott Santiamo Director, Corporate Communications Abeona Therapeutics +1 (718) 344-5843 ssantiamo@abeonatherapeutics.com

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Abeona Therapeutics to Participate in Upcoming Investor Conferences - Stockhouse

Roots Analysis has done a detailed study on Gene Therapy Market (3rd Edition), 2019-2030, covering key aspects of the industrys evolution and identifying potential future growth opportunities.

Key Market Insights

For more information, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

Table of Contents

4.2.1 Types of Viral Vectors

11.2 Analysis by Special Designations Awarded

11.3.1. Analysis by Target Indication

11.3.2. Analysis by Type of Vector Used

11.4.1. Analysis by Target Indication

11.4.2. Analysis by Type of Vector Used

11.5.1. Analysis by Target Indication

11.5.2. Analysis by Type of Vector Used

11.6.1. Analysis by Target Indication

11.6.2. Analysis by Type of Vector Used

11.7.1. Analysis by Target Indication

11.7.2. Analysis by Type of Vector Used

11.8.1. Analysis by Target Indication

11.8.2. Analysis by Type of Vector Used

11.9.1. Analysis by Target Indication

11.9.2. Analysis by Type of Vector Used

11.10.1. Analysis by Target Indication

11.10.2. Analysis by Type of Vector Used

11.11.1. Analysis by Target Indication

11.11.2. Analysis by Type of Vector Used

11.12.1. Analysis by Target Indication

11.12.2. Analysis by Type of Vector Used

11.13.1. Analysis by Target Indication

11.13.2. Analysis by Type of Vector Used

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The Gene Therapy Market is projected to grow at an annualized rate of 45%, till 2030 - Scientect

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

Gene Therapy Market Report: Industry Insights, 2020-2026

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

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

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

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The FREE PDF Sample Report Includes:

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

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

2020 Gene Therapy Market: COVID-19 Impact Analysis

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

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

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Gene Therapy Market 2020: Research Scope & Coverage

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

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

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Competitive Landscape

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

Key Takeaways from this Gene Therapy Market Report

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Know Top Players and Growing Trends in Gene Therapy Market 2020 Research Report and Industry Forecast till 2026 - Galus Australis

MarketsandResearch.biz has published the latest market research study on Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020 by Company, Type and Application, Forecast to 2025 which investigates a few critical features of the market such as industry condition, division examination, market insights. The report studies the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market share, competition landscape, market share, growth rate, future trends, market drivers, opportunities and challenges, sales channels. The report has referenced down to earth ideas of the market in a straightforward and unassuming way in this report. The research contains the categorization of the market by top players/brands, region, type, and end-user. The report exhaustive essential investigation of current market trends, opportunities, challenges, and detailed competitive analysis of the industry players in the market.

The research report has comprehensively included numbers and figures with the help of graphical and pictorial representation which embodies more clarity on the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market. Then the report delivers key information about market players such as company overview, total revenue (financials), market potential, global presence, as well as market share, prices, production sites and facilities, products offered, and strategies adopted by them. Market status and outlook of global and major regions, from angles of players, countries, product types, and end industries have been analyzed.

NOTE: Our report highlights the major issues and hazards that companies might come across due to the unprecedented outbreak of COVID-19.

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Key strategic manufacturers included in this report: BioReliance, Richter-Helm, UniQure, Cobra Biologics, MassBiologics, Oxford BioMedica, Lonza, MolMed, FinVector, FUJIFILM Diosynth Biotechnologies, Brammer Bio, bluebird bio, Aldevron, Spark Therapeutics, VGXI, Biovian, Eurogentec, Novasep, PlasmidFactory, Cell and Gene Therapy Catapult, Vigene Biosciences

Market Potential:

Key market vendors have been predicted to obtain the latest opportunities as there has been an increased emphasis on spending more on the work of research and development by many of the manufacturing companies. Also, many of the market contenders are forecasted to make a foray into the emerging economies to find new opportunities. The global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market has gone through rapid business transformation by good customer relationships, drastic and competitive growth, significant changes within the market, and technological advancement in this market.

Geographically, this report is segmented into several key countries, with market size, growth rate, import and export of in these countries from 2015 to 2020, which covering: North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), MENA (Saudi Arabia, UAE, Turkey and South Africa)

The market can be segmented into product types as: AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA, Other Vectors

The market can be segmented into applications as: Cancers, Inherited Disorders, Viral Infections, Others

ACCESS FULL REPORT: https://www.marketsandresearch.biz/report/83910/global-viral-vectors-non-viral-vectors-and-gene-therapy-manufacturing-market-2020-by-company-type-and-application-forecast-to-2025

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Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020 | Scope of Current and Future Industry 2026 - Good Night, Good...