StemCell Therapy

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, a patient-centric gene therapy company with a mission to eradicate monogenic CNS disease, today announced the appointment of its executive leadership team. This group has significant experience in gene therapy drug development and commercialization, and will enable Taysha to build the corporate culture and infrastructure necessary to advance its extensive pipeline of 18 gene therapy programs, with exclusive options to acquire four additional programs from UT Southwestern Gene Therapy Program. In addition, Sukumar Nagendran, M.D., former Chief Medical Officer of AveXis, and Phillip Donenberg, former Chief Financial Officer of AveXis, have joined the companys Board of Directors. Mr. Donenberg will also serve as the companys Audit Committee Chairman.

Joining the Taysha Board is a unique opportunity to contribute to scientific advancements in CNS gene therapy, said Sukumar Nagendran, M.D., Taysha Board of Directors. In partnership with UT Southwestern, Taysha has built an extensive pipeline of gene therapy candidates for life-threatening CNS diseases with significant unmet medical need.

It is a distinct pleasure to be reunited with many of my former AveXis colleagues that enabled the development and successful commercialization of Zolgensma, said Phillip Donenberg, Taysha Board of Directors. I am excited to contribute to Tayshas efforts to deliver therapies with the potential to improve the lives of patients with devastating CNS disease.

Each member of the Taysha leadership team has significant gene therapy expertise, with an unrelenting, patient-first focus guiding their individual areas of focus. Joining RA Session II, Founder, President and CEO of Taysha, on the management team are the following individuals:

From day one, we set out to build a team that has the passion, experience and talent to achieve our mission of eradicating monogenic CNS disease. Today, we are proud to announce a highly experienced team of CNS gene therapy experts, said Mr. Session. We are also pleased Dr. Nagendran and Mr. Donenberg have joined our Board and will contribute their several years of gene therapy expertise. Their experience in building AveXis will be invaluable as we continue to grow and advance several programs into the clinic.

About Taysha Gene Therapies

Taysha Gene Therapies is a patient-centric gene therapy company with a mission to eradicate monogenic CNS disease. We are focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the CNS in both rare and large patient populations. We were founded in partnership with The University of Texas Southwestern Medical Center, or UT Southwestern, to develop and commercialize transformative gene therapy treatments. Together with UT Southwestern, we are advancing a deep and sustainable product portfolio of 18 gene therapy product candidates, with exclusive options to acquire four additional development programs. By combining our management teams proven experience in gene therapy drug development and commercialization with UT Southwesterns world-class gene therapy research capabilities, we believe we have created a powerful engine to develop transformative therapies to dramatically improve patients lives. More information is available at http://www.tayshagtx.com.

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Taysha Gene Therapies Builds Experienced Executive Leadership Team to Advance Pipeline of Gene Therapies for Monogenic CNS Disease in Both Rare and...

Sept. 2, 2020 12:00 UTC

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, today announced the appointment of two new executives, Lisa M. Olson, Ph.D., as Chief Scientific Officer and Kevin B. Johnson, Ph.D., as Senior Vice President, Head of Regulatory and Quality. The Company also announced that Jason Ryan will transition from Chief Operating and Financial Officer to a consulting role for personal reasons while a search for his replacement is conducted.

With the additions of Lisa and Kevin to our team, Magenta continues to deepen our technical expertise, bolstering our strong discovery, research, development and regulatory leadership to further our goal of delivering curative immune reset to patients in need, said Jason Gardner, D.Phil., Chief Executive Officer and President, Magenta Therapeutics. We are delighted to welcome Lisa and Kevin on board and look forward to their many contributions to the Magenta mission.

As Chief Scientific Officer, Dr. Olson will provide strategic direction, oversight and execution for Magentas research and discovery efforts. This entails driving research strategy as Magenta continues to optimize its preclinical and clinical pipeline. She will join the executive team and will be a key member of the R&D leadership team.

Dr. Olson is an experienced senior-level pharmaceutical executive, with more than 20 years of experience in research and drug discovery. She comes to Magenta following 15 years in leadership positions at the AbbVie Bioresearch Center, most recently as Vice President, Immunology Discovery and Site Head, where she was responsible for all immunology discovery scientific and portfolio decisions, including new target approval, project advancement and licensing opportunities. Under her leadership, 15 molecules advanced into clinical development, including Upadacitinib that launched last year as Rinvoq. Prior to AbbVie, she served as a Research Fellow and Group Leader in Inflammation & Immunology at Pfizer, Inc. She began her career as an Assistant Professor at Washington University School of Medicine, following a post-doctoral cardiovascular fellowship at the University of Chicago.

Dr. Olson holds a Ph.D. from the University of Illinois at Urbana-Champaign and a Bachelor of Science from Iowa State University.

As Senior Vice President, Head of Regulatory and Quality, Dr. Johnson will lead Magentas global regulatory strategy for the Companys programs across multiple therapeutic areas. He will also be responsible for the oversight and accountability for all quality activities to enable Good Practice (GxP) functions across the portfolio. In this role, Dr. Johnson will provide strategic guidance and leadership to members of the R&D leadership team and the regulatory and quality teams for Magentas portfolio for all phases of product lifecycle.

Dr. Johnson bring years of regulatory, quality assurance and development leadership, coming to Magenta from Imara, Inc., where he served as Senior Vice President, Regulatory Affairs, Quality and Pharmacovigilance, leading successful requests for several regulatory designations with the U.S. Food and Drug Administration (FDA). Prior to his time at Imara, Dr. Johnson led global regulatory strategy and implementation for breakthrough therapy-designated rare disease development programs at Vtesse, later acquired by Sucampo. He also served as Director, Global Regulatory Affairs for Rare Diseases and Gene Therapies at GlaxoSmithKline, where he was part of on the international regulatory team for the European approval of the gene therapy Strimvelis for ADA-SCID, and subsequently secured Regenerative Medicine Advanced Therapy (RMAT) designation for a retinal gene therapy product.

Dr. Johnson holds a Ph.D. in Neurobiology from the University of North Carolina (UNC) School of Medicine; a Master of Business Administration from the Kenan-Flagler School of Business, UNC; and a Bachelor of Science in Chemistry from the University of South Florida.

Along with these leadership team additions, Magenta also announced today that Jason Ryan, Chief Operating and Financial Officer, will step down from that role at the end of September. He will continue to contribute to Magenta in a consulting capacity, and the Company has commenced a search for a replacement.

Jason has been a dynamic and reliable leader at Magenta since he joined us in 2019, leading finance and operations, contributing to our strategic planning efforts, and spearheading two financings during a period of significant growth, said Gardner. We are truly grateful for his contributions to the patients we seek to serve, our employees and business partners.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption Risk Factors in Magentas Annual Report on Form 10-K filed on March 3, 2020, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release 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/20200902005236/en/

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Magenta Therapeutics Appoints Lisa M. Olson as Chief Scientific Officer and Kevin B. Johnson as Senior Vice President, Head of Regulatory and Quality;...

REDWOOD CITY, Calif. and RESEARCH TRIANGLE PARK, N.C., Sept. 3, 2020 /PRNewswire/ --Kriya Therapeutics, a next generation gene therapy company focused on developing transformative treatments for highly prevalent diseases,announced today that its CEO, Shankar Ramaswamy, M.D., will present at multiple upcoming healthcare conferences in September and October. These include the following:

Citi's 15th Annual BioPharma Virtual ConferenceDate: Tuesday, September 8thTime: 3:30 PM ET / 12:30 PM PT

H.C. Wainwright & Co. 22nd Annual Global Investment ConferenceDate: Wednesday, September 16thTime: 9:30 AM ET / 6:30 AM PT

Cantor Fitzgerald Virtual Global Healthcare ConferenceDate: Thursday, September 17thTime: 3:20 PM ET / 12:20 PM PT

Chardan 4th Annual Genetic Medicines ConferenceDate: Tuesday, October 6thTime: 9:00 AM ET / 6:00 AM PT

About Kriya Therapeutics

Kriya Therapeutics is a next-generation gene therapy company focused on developing transformative treatments for highly prevalent serious diseases. With core operations in California and North Carolina, Kriya's technology-enabled platform is directed to the rational design and clinical translation of gene therapies for large patient populations. For more information, please visit http://www.kriyatx.com.

Cautionary Note on Forward-Looking Statements

This press release includes forward-looking statements pertaining to our development programs and our proprietary platform. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. The forward-looking statements contained in this press release reflect Kriya's current views with respect to future events, and Kriya does not undertake and specifically disclaims any obligation to update any forward-looking statements.

ContactDan ChenChief Financial Officer[emailprotected]

SOURCE Kriya Therapeutics

https://www.kriyatx.com/

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Kriya Therapeutics To Present At Upcoming Healthcare Conferences - PRNewswire

New Phase 1b Data of Investigational Gene Therapy Compound, VY-AADC (NBIb-1817), Evaluating Three-Year Safety and Clinical Outcomes in Patients with Advanced Parkinsons Disease

Voyager to Participate in Upcoming September Investor Conferences

CAMBRIDGE, Mass., Sept. 03, 2020 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases, today announced data presentations at the International Parkinson and Movement Disorder Virtual Congress 2020 taking place on September 12-16, 2020. The presentations include new two- and three-year data related to its VY-AADC gene therapy treatment for Parkinsons disease being developed in collaboration with Neurocrine Biosciences:

Additionally, the company plans to participate in the following virtual investor conferences in September:

The webcast sessions may be accessed from the Investors & Media section of Voyagers website at http://www.voyagertherapeutics.com. Replays of the webcasts will be archived on the Company's website for at least 30 days.

About Voyager Therapeutics

Voyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyagers wholly owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinsons disease, Huntingtons disease, Friedreichs ataxia, and other severe neurological diseases. For more information, please visit http://www.voyagertherapeutics.com or follow @VoyagerTx on Twitter and LinkedIn.

Investor Relations: Paul CoxVP, Investor Relations857-201-3463pcox@vygr.com

Media: Sheryl Seapy W2Opure949-903-4750sseapy@purecommunications.com

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Voyager Therapeutics Announces Upcoming Presentations at the International Parkinson and Movement Disorder Society Virtual Congress 2020 -...

The NIH is pitching $14.6 million into a three for one HIV research program led by USC and the Fred Hutchinson Cancer Research Center that aims to strike the need for daily medication or even achieve a home run cure.

The five-year grant will back preclinical studies that combine gene editing with technology to improve bone marrow transplants. The potential therapy would engineer a patients own stem cells to fight HIV, and stimulate them to produce new immune cells once reintroduced to the patient.

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

Hans-Peter Kiem, the Stephanus Family Endowed Chair for Cell and Gene Therapy at Fred Hutch, is the co-director. Harvard University professor David Scadden and Magenta Therapeutics are also collaborating on the project.

The approach was inspired by three patients who appear to have been cured of the virus all of whom received blood stem cell transplants from donors who carried a mutation in the CCR5 gene. One of them, dubbed the Berlin patient, has been off antiretroviral drugs since 2007.

I think of the Berlin patient as proof of principle that replacing the immune system with one thats HIV-resistant by removing CCR5 is a possible way to treat somebody, Cannon said.

The program will study the use of gene editing to remove CCR5 from patients stem cells a process which is already in clinical trial for HIV treatment at City of Hope National Medical Center in Duarte, CA. The stem cells will also be engineered to release antibodies and antibody-like molecules that block HIV.

In addition, the grant will fund a Fred Hutch teams endeavor to adapt CAR-T cell therapy to create stem cells whose progeny target HIV-infected cells.

As for preparing a patient for the transplant,Magenta is working on antibody-drug conjugates to replace mild chemotherapy or radiotherapy typically given before the procedure. And Scadden is researching an injectable gel that could help immune cells repopulate more quickly, avoiding a delay.

HIV infection, which currently affects about 1.2 million Americans, has proved to be exceedingly difficult to cure. In July, Merck and Dewpoint inked a deal that allows the pharma to use the Boston-based biotechs biomolecular condensate technology to develop treatments, and potentially a cure, for the HIV virus. The NIH-funded group is hoping to at least control the virus enough to eliminate the need for daily meds. But at best, theyre also eyeing a long sought-after cure.

This grant funds a team with an overarching goal of developing what our perfect HIV gene therapy would look like, Cannon said. All of these pieces could happen separately, but the fact that the NIH has funded us as a team means that the sum will be so much bigger than the parts.

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Researchers teamed up to develop a 'three in one' HIV treatment and the NIH is throwing in $14.6M - Endpoints News

Fouad Namouni, a storied research exec who went from project leader on Opdivo and Yervoy to the top of the oncology research group at Bristol Myers Squibb, is joining the migration to biotech, picking up a new hat as president of R&D at Blueprint Medicines.

Once again, hes headed into a toe-to-toe showdown with a rival pharma organization.

Namouni will likely be coming on board just one step ahead of an approval for pralsetinib, Blueprints RET rival to Eli Lillys Retevmo, which got out in front with a May approval. Ironically, Lillys deal to buy into RET with its acquisition of Loxo also brought Josh Bilenker and his crew to the pharma giant, marking a rare career trajectory from a biotech into pharma, which has been bleeding talent for years now.

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Game change: A frontrunner in the cell therapy 2.0 field offers a first look at their lead therapy. And it's a doozy - Endpoints News

CAMBRIDGE, Mass., Sept. 3, 2020 /PRNewswire/ -- Obsidian Therapeutics, Inc., a biotechnology company pioneering controllable cell and gene therapies, today announced the appointment of Robert Ross, M.D., to its Board of Directors. Dr. Ross currently serves as the Chief Medical Officer of Surface Oncology.

"Rob brings strong clinical development experience, specifically in oncology, which will be invaluable as we advance towards the clinic," said Paul K. Wotton, Ph.D., Obsidian's Chief Executive Officer. "Rob has successfully advanced multiple programs from IND to pivotal trials, as well as led collaborations with industry and academic partners. His experience in progressing novel and innovative therapies, including cell and gene therapies, from the bench to the bedside will expedite the development of the first cytoDRiVE-based programs into human clinical trials to our ultimate goal of treating cancer patients with controllable living medicines."

Dr. Ross added, "Obsidian addresses a key unmet need in cell and gene therapy through the ability to regulate the biological activity of engineered cells, allowing the creation of highly effective, titratable and targeted immune-oncology therapies. I look forward to contributing to Obsidian's growth and clinical progress."

Dr. Ross serves as the Chief Medical Officer of Surface Oncology and oversees all clinical and regulatory operations and development efforts. He is responsible for advancing Surface Oncology's programs into the clinic. Rob has extensive clinical development experience, most recently at bluebird bio where he led the clinical development of genetically modified cellular therapies in betathalassemia and sickle cell disease. Rob was also the head of oncology at bluebird bio, building a multifaceted oncology program, led by an anti-BCMA chimeric antigen T cell therapy in collaboration with Celgene. Previously, he worked at Genentech and Infinity Pharmaceuticals on both small molecule and antibody programs from Phase I through pivotal trials, and was a faculty member at the Dana Farber Cancer Center, treating patients with genitourinary malignancies. Rob earned his bachelor's degree from Stanford University, his master's degree from Harvard Medical School and his medical degree from Columbia University Vagelos College of Physicians and Surgeons. Rob did his residency in internal medicine at the University of California, San Francisco and his fellowship in hematology/oncology at the combined program at the Dana Farber/Massachusetts General Hospital.

About Obsidian Therapeutics Obsidian Therapeutics, Inc. is a biotechnology company pioneering controllable cell and gene therapies to deliver transformative outcomes for patients with intractable diseases. Obsidian's proprietary cytoDRiVE platform provides, for the first time, a technology to develop a new generation of cell and gene therapies in which the level and timing of protein activity are fully controlled in a dose-dependent platform comprises a therapeutic protein of interest fused to a drug-responsive domain (DRD). In the absence of the small molecule drug, the DRD-tagged protein is degraded before it becomes active. In contrast, when the small molecule drug is present, the DRD-tagged protein is stabilized and active, permitting precise control of the timing and level of protein expression. The platform can be applied to design controllable intracellular, membrane and secreted proteins for cell and gene therapies as well as other applications. The Company is headquartered in Cambridge, Mass. For more information, please visit http://www.obsidiantx.com.

Media Contact: Maggie Beller Russo Partners, LLC [emailprotected]646-942-5631

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Obsidian Therapeutics Appoints Rob Ross, MD, to Board of Directors - PRNewswire

LEIDEN, the Netherlands, September 03, 2020 / B3C newswire / -- Amarna Therapeutics, a privately held biotechnology company developing the next-generation SV40-based gene delivery vector platform named SVec that promises to transform gene-replacement and immunotherapy across many disease areas, today announced the appointment of Steen Klysner, Ph.D. as its new Chief Executive Officer (CEO) as per September 1. Founder and CEO Ben van Leent will become a member of Amarnas Supervisory Board.

I am very pleased to welcome Steen Klysner as our new CEO. Steen brings an extensive background as a biotech CEO to Swedish ExpreS2ion Biotech Holding AB & the Danish ExpreS2ion Biotechnologies ApS, preceded by an impressive track record in execution and value creation within the biotech industry. His leadership experience makes Steen an ideal candidate to lead Amarna into its next stage of growth and development. said Thomas Eldered, Chairman of Amarnas Supervisory Board. We are extremely grateful for Ben van Leents leadership and contributions to Amarna as both founder and CEO, and we are excited about the opportunity to focus his outstanding expertise as member of our Board.

I am incredibly honored to have been given the opportunity to lead Amarna, said Dr. Klysner. SVec has the potential to enable major medical breakthroughs, so that patients can be actually cured of life-threatening diseases for which, to date, effective treatment have not become available. Together with Amarnas highly qualified and experienced team, Im fully committed to advance the companys groundbreaking technology into the next important clinical development stages.

Steen Klysner comes to Amarna with over 30 years of experience in the life sciences industry. Prior to joining Amarna, Dr. Klysner served as CEO of the Swedish ExpreS2ion Biotech Holding AB in parallel with the Danish ExpreS2ion Biotechnologies ApS. Earlier, he was Senior Vice President (SVP) of preclinical R&D and SVP of Quality of Allergopharma, the Allergy Business Unit of Merck KGaA. He also served as CEO of Nordic Vaccine in Copenhagen, focusing on the development of non-invasive vaccination based on an integrated nanoparticle adjuvant and delivery platform. Prior to that he has also held positions at Pharmexa, Novo Nordisk and ALK.Dr. Klysner holds a Ph.D. from Technical University of Denmark combined with an Industrial Scientist Research Degree from the Danish Academy of Technical Sciences, a M.Sc. degree in Biochemistry from the University of Copenhagen and a B.Sc. in sports from the University of Copenhagen.Finally, Dr. Klysner is author/co-author of numerous patents and scientific publications in (inter)-national peer-reviewed medical journals.

I am deeply grateful to have had the opportunity to build Amarna to where it is today. In my new role as member of the Supervisory Board, my efforts will be towards helping raise the awareness of Amarna and its SVec gene delivery vector platform and using it to help build a robust pipeline, said Ben van Leent. I have full confidence that Steen, a very passionate and talented leader, will provide the leadership and expertise needed to guide Amarna through the next phases of growth. I look forward to working with Steen to drive forward Amarnas product candidates.

Caption: Steen KlysnerFor high resolution please click the image.

About SV40 vectors: A key to the success of gene therapyToday gene therapy enables the development of a next wave of treatments, with potential to not only treat but also to cure a number of major diseases. Key to the success of gene therapy is the efficient delivery of therapeutic genes into target cells, which is an ability that naturally evolved in viruses, rendering them ideally suited for gene delivery.The Simian virus 40 (SV40) strictly replicates in its natural host, macaque monkeys. The virus cannot replicate in humans and doesnt elicit an immune response, which makes it ideal for developing effective gene therapies. However, the clinical use of SV40 vectors has been hampered by production and safety issues. Amarna has solved this, by developing a novel proprietary SuperVero production cell line and the SVec gene delivery platform.Importantly, SVec can be used to efficiently induce immune tolerance to self-antigens driving degenerative, inflammatory and autoimmune human diseases. Amarna aims to develop SVec-based reverse vaccines for major indications such as neurodegenerative and psychiatric diseases (NDPs), atherosclerotic cardiovascular disease (ACD), obesity, diabetes mellitus (DM), arthritis and chronic obstructive pulmonary disease (COPD).

About Amarna TherapeuticsAmarna Therapeutics is a privately held Biotech company founded in 2008. Its head office is located in Leiden (The Netherlands), and its research facility in Seville (Spain). The company has developed a proprietary SuperVero cell line and SVec gene delivery platform for the development of safe and efficient immunotherapies for major indications within the degenerative, inflammatory and autoimmune disease areas.In October 2019, Amarna secured 10 million in new equity to progress its SVec platform towards clinical studies. The financing round was led by Flerie Invest AB, a Swedish investment company, together with existing shareholders and an innovation credit from the Netherlands Enterprise Agency (RVO.nl).

Contacts

Amarna TherapeuticsSteen Klysner, CEOThis email address is being protected from spambots. You need JavaScript enabled to view it.

LifeSpring Life Sciences Communication, AmsterdamLon Melens+31 6 538 16 427This email address is being protected from spambots. You need JavaScript enabled to view it.

Keywords: Humans; Simian virus 40; Netherlands; Genetic Therapy; Immunotherapy; Gene Transfer Techniques; Immune Tolerance

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Dutch Amarna Therapeutics Announces the Appointment of Steen Klysner as Chief Executive Officer - b3c newswire

PHILADELPHIA, PA, Sept. 03, 2020 (GLOBE NEWSWIRE) -- Spirovant Sciences, a gene therapy company developing treatments and cures for genetic lung diseases including cystic fibrosis, todayannounced that its CEO, Joan Lau, PhD, has been named finalist for the Ernst & Young LLP (EY US) Entrepreneur of the Year 2020 Award in the Greater Philadelphia area. The award honors entrepreneurial business leaders whose ambitions deliver innovation, growth and prosperity as they build and sustain successful businesses that transform our world. Award winners will be announced through a special virtual event in early October.

I am sincerely honored to be named a finalist for the EY Entrepreneur of the Year and to represent our terrific company, our talented team, our inspiring patients, and all the innovative entrepreneurs and scientists in this great region, said Lau. The successes and accomplishments of Spirovant, including being acquired twice in 2019, have resulted from the talent, steadfastness and dedication of our rapidly growing team. These truly exceptional individuals power our mission to deliver innovative gene therapy treatments to patients who have no other options. I thank EY for this honor and its support of entrepreneurialism in Greater Philadelphia and throughout the world.

About EY Entrepreneur of the Year

Entrepreneur of The Year is one of the preeminent competitive award programs for entrepreneurs and leaders of high-growth companies. The nominees are evaluated based on six criteria: overcoming adversity; financial performance; societal impact and commitment to building a values-based company; innovation; and talent management. Since its launch, the program has expanded to recognize business leaders in more than 145 cities in over 60 countries around the world.

Founded and produced by Ernst & Young LLP, the Entrepreneur of The Year Awards are nationally sponsored by SAP America and the Kauffman Foundation. In Greater Philadelphia, sponsors also include PNC Bank, DFIN, SolomonEdwards Group, Ballard Spahr LLP, Morgan, Lewis & Bockius LLP, Murray Devine & Company and Pepper Troutman LLP.

About Spirovant Sciences, Inc.Spirovant is a gene therapy company focused on changing the course of cystic fibrosis and other genetic lung diseases. The company's current investigational gene therapy technologies are designed to overcome the historical barriers that have prevented effective genetic treatments for cystic fibrosis. Spirovant is advancing programs for cystic fibrosis with both AAV and lentivirus vectors. Spirovant is a wholly owned subsidiary of Sumitovant Biopharma Ltd., which is itself a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. Spirovant is located inPhiladelphia, PA.More information is available athttps://www.spirovant.com/.

About Sumitovant BiopharmaLtd.Sumitovant is a global biopharmaceutical company with offices inNew York CityandLondon. Sumitovant is a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. Sumitovant is the majority shareholder of Myovant and Urovant, and wholly owns Enzyvant, Spirovant andAltavant. Sumitovant'spipeline is comprised of early- through late-stage investigational medicines across a range of disease areas targeting high unmet need. For further information about Sumitovant please visithttps://www.sumitovant.com/.

About Sumitomo Dainippon Pharma Co., Ltd.Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies inJapan, operating globally in major pharmaceutical markets, includingJapan, the U.S.,Chinaand the European Union. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 6,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website athttps://www.ds-pharma.com/.

Media ContactJennifer Guinan

Sage Strategic Marketing

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Spirovant CEO Joan Lau Named Finalist for EY Entrepreneur of The Year in Greater Philadelphia - GlobeNewswire

Breakthrough in delivery for cell and gene therapy products has led to a wave of M&A activity as big pharma aims not to miss out on the future of medicine

AIM healthcare index at the centre of innovation, has risen 6% YTD compared with the AIM all share, which has declined 7%

finnLife 50 index has also risen 6% in 2020 led by gains in Synairgen (+2,930%), Avacta (+654%), Omega Diagnostics (+322%) and Tiziana Life Sciences (+283%)

London 25 August 2020 Healthcare companies employing and developing cell and gene therapy (C>) are driving the next wave of innovation in the pharmaceutical industry, leading to increased M&A activity as big pharma aims not to miss out on the future of medicine. The AIM healthcare index has been at the centre of this innovation, rising 6% YTD compared with the AIM all share, which has declined 7%.

These are the findings of finnCaps new quarterly Life Sciences sector report, Rude Health.

Rather than just treating a disease and its symptoms, C> can target the underlying cause of a disease, with long-term benefits and curative potential. C> is now being realised on an applicable level, with many products already approved and the FDA expects to approve 10-20 products a year by 2025.

The financials of the sector are reflective of this rapid progress. In 2018, the market value of C> was $536 million - $1.07 billion; but by 2026 it is set to have a valuation of up to$35.4 billion. Given the high proportion of start-ups in the sector, M&A activity is on the rise, as evidenced by the $3 billion Astellas spent in January 2020 to acquire Audentes Therapeutics, specialists in genetics medicines.

In 2014/2015, M&A activity in the sector was $5 billion; by 2018/2019 it had surged 880% to $49 billion. Much of this is driven by big pharma firms not wanting to fall behind their smaller, more versatile competition, as they did with monoclonal antibody technology. Consequently, they have engaged with M&A to speed up and enhance their own R&D efforts.

The report notes that innovators in C> will be well placed to take part in the land grab that will follow as a result of continued advancements in the sector, and highlights now as a good time for investors and pharmaceutical companies to become involved as the sector is rapidly maturing past its high potential research and development stage with an established pipeline of therapies already being developed.

Some of the key reasons why the report considers the C> sector to be an attractive one for investment are:

Pharmas next wave of innovation. C>s can be potentially curative treatment options as they usually target the underlying cause of disease. In the long term, these therapies could become the backbone of treatment regimens, and solutions to various unmet needs.

Deals. Big Pharma had to play catch-up with monoclonal antibody technology and seems determined not to make the same mistake with C>, as reflected in the high deal activity and high deal values seen within this space.

Sector maturation. Advances in the sector mean that the C> sector is beginning to mature beyond the R&D stage and into commercialisation, with some products already approved, and with a very large future pipeline of therapies.

Revenue.Therapies in this space can command high prices, allowing for high revenue generation, even from rare diseases and limited patient populations.

Despite its vital role in the future of medicine, C> also comes with challenges. The report highlights that the manufacture of C>s is difficult given they are, by definition, personalised for the patient. This means they cannot be batch produced for distribution to multiple patients as more traditional medicines can. For example, Zolgensma, which treats those with motor neurone disease, is priced at $2.1 (1.6) million per therapy, making it the most expensive drug treatment ever.

The report also notes how the payment process for C> requires a reworking of reimbursement systems not used to outlaying so much money up front for a treatment with long-term benefits/curative potential versus continuous, and lower payments for ongoing medicine treatment.

The technologies the report shines a spotlight include CAR-T therapy, stem cell therapy, CRISPR, RNA therapies, among various others.

Arshad Ahad, Research Analyst, Life Sciences, at finnCap, commented:Few technologies in the life sciences sector hold as much promise as Cell and Gene Therapy, with its ability to provide long-term benefits and curative potential. These technologies have been seen as the future of medicine for many years, and now we are closer than ever to that future becoming a reality. If Cell and Gene therapy does become the backbone of treatment regimes in the future, similar to the rise of monoclonal antibodies, then the companies involved are developing expertise in a critical part of the life sciences industry, which should confer a significant competitive advantage as the sector matures further. Now is therefore a good time to invest in the future.

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FINNCAP'S LIFE SCIENCES REPORT INDICATES CELL AND GENE THERAPY SECTOR IS DRIVING THE NEXT WAVE OF INNOVATION IN PHARMA - PharmiWeb.com

The broker said the market value of the sector is expected to increase to up to US$35.4bn by 2026 from its 2018 value of between US$536mln-US$1.07bn

FinnCap Group PLC () has said healthcare companies employing and developing cell and gene therapy (C>) are driving the next wave of innovation in the pharmaceutical industry, which in turn is causing an uptick in mergers & acquisitions as pharma giants aim to be at the forefront of the next development in medicine.

In a report released on Tuesday, the brokerage firm highlighted that the trend was personified in the AIM healthcare index, which has risen 6% in the year to date as opposed to a 7% decline for the AIM All-Share index.

Small caps involved in the sector that have seen rapid share price rises this year such as (), () and (), have also led gains in finnCaps own finnLife 50 index.

The group also reported that the market value of the C> sector is expected to increase to up to US$35.4bn by 2026 from its 2018 value of between US$536mln-US$1.07bn.

M&A is also on the rise given the high number of startups in the sector, finnCap said, with acquisition activity valued at US$49bn in 2018/19 compared to US$5bn in 2014/15.

Much of this is driven by big pharma firms not wanting to fall behind their smaller, more versatile competition, as they did with monoclonal antibody technology. Consequently, they have engaged with M&A to speed up and enhance their own [research & development] efforts, the broker said.

Rather than just treating a disease and its symptoms, C> can target the underlying cause of a disease, with long-term benefits and curative potential. C> is now being realised on an applicable level, with many products already approved and the [US Food & Drug Administration] expects to approve 10-20 products a year by 2025, they added.

As a result, the report said that now was the time for investors to get involved as the C> sector is rapidly maturing past its high potential research and development stage with an established pipeline of therapies already being developed.

However, finnCap cautioned that there were challenges facing the sector including difficulty in manufacturing the therapies due to their personalised nature as well as a potential reworking of reimbursement systems that are not used to outlaying so much money up front for a treatment with long-term benefits/curative potential versus continuous, and lower payments for ongoing medicine treatment.

Few technologies in the life sciences sector hold as much promise as Cell and Gene Therapy, with its ability to provide long-term benefits and curative potential, said finnCap life sciences analyst Arshad Ahad.

These technologies have been seen as the future of medicine for many years, and now we are closer than ever to that future becoming a reality. If Cell and Gene therapy does become the backbone of treatment regimes in the future, similar to the rise of monoclonal antibodies, then the companies involved are developing expertise in a critical part of the life sciences industry, which should confer a significant competitive advantage as the sector matures further. Now is therefore a good time to invest in the future, the research analyst added.

FinnCap shares were steady at 23p in late-morning trading on Wednesday.

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FinnCap touts cell and gene therapy as next wave of innovation in pharma industry - Proactive Investors UK

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apic Bio Inc., a gene therapy company developing novel treatment options for patients with rare genetic diseases, today announced the appointment of Jorge A. Quiroz, MD, MBA, as Executive Vice President, Chief Medical Officer. Dr. Quiroz brings over 20 years of scientific, clinical, and regulatory experience helping to develop and evolve gene therapies and small molecule drug programs from preclinical to regulatory filing. Most recently, Dr. Quiroz served as Chief Medical Officer of Solid Biosciences where he was responsible for leading the clinical advancement of its gene therapy program.

We are excited to welcome Jorge to Apic during this critical period for the company and our pipeline. His extensive expertise in the clinical and regulatory development of gene therapies for rare diseases will support the advancement of both our SOD1 amyotrophic lateral sclerosis (ALS) and alpha-1 antitrypsin deficiency (Alpha-1) programs, said John Reilly, MS, MBA, Co-founder and Chief Executive Officer of Apic. As CMO, Jorge will also lead the development and build out of our early-stage gene therapy programs derived from our THRIVE platform. We look forward to working with Jorge during this next chapter for Apic as we rapidly advance our therapies into the clinic on behalf of patients and families in need.

I am delighted to join Apic during an exciting period of expansion as we prepare to submit an Investigational New Drug (IND) application for APB-102 for the treatment of patients with SOD1 ALS this year and enter the IND-enabling stage for APB-101 for the treatment of patients with alpha-1 antitrypsin deficiency, said Dr. Jorge Quiroz, EVP and Chief Medical Officer of Apic. The Companys mission, deep scientific foundation, clinical approach, and manufacturing expertise puts us in an excellent position to bring new gene therapy treatments to patients living with rare and monogenic disorders.

Dr. Quiroz previously served as the Head of Neurodevelopment & Psychiatry, Translational Medicine Neurosciences at F. Hoffmann-La Roche AG. He has also served as a Director at Johnson & Johnson Pharmaceutical Research & Development, LLC. He received an MD from the Pontifical Catholic University of Chile and completed his medical training as a Research Fellow at the Laboratory of Molecular Pathophysiology, at the National Institute of Mental Health. Dr. Quiroz is board certified in Psychiatry and also holds an MBA dual degree from Columbia University and London Business School.

About Apic Bio

Apic Bio is a gene therapy company focused on developing novel treatment options for rare, undertreated neurological and liver diseases. The Company's lead program is an adeno-associated (AAV)-based gene therapy for the treatment of the copper zinc superoxide dismutase 1 (SOD1) ALS, a genetic form of the disease. Preclinical studies of additional genetic forms of ALS (C9Orf72) and alpha-1 antitrypsin deficiency (Alpha-1) are ongoing. The Company is also advancing discovery programs for two undisclosed CNS indications that leverage its proprietary silence and replace THRIVE platform. The Company is backed by leading and disease-centric investors, including Morningside Ventures, ALS Investment Fund, and The Alpha-1 Project (TAP). For more information please visit http://www.apic-bio.com.

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Apic Bio Announces Appointment of Jorge A. Quiroz, MD, MBA, as Executive Vice President and Chief Medical Officer - Business Wire

The addition of cryogenic freezer systems firm MVE Biological Solutions will boost Cryoports position in the temperature-controlled cell and gene therapy logistics space.

Cryoport, a temperature-controlled supply chain services firm for the life sciences, has agree to buy MVE Biological Systems for $320 million with the deal expected to close by the end of the year.

MVE is a subsidiary of Chart Industries and will bring Cryoport a greater presence in the cell and gene therapy services space, broadening its portfolio to include manufactured vacuum insulated products and cryogenic freezer systems. The firm has three manufacturing sites two in the US and one in China and more than 300 customers, including Cryoport itself.

Image: iStock/Zhanna Hapanovich

According to Jefferies analyst Brandon Couillard, MVE has an estimated 55% share of the vacuum insulated products and cryogenic equipment market and the acquisition will bolster Cryoports strategy of becoming the top life sciences supply chain solutions provider, further augmenting its exposure to the rapidly growing cell & gene therapy space.

The deal comes days after Cryoport announced it is buying French logistics firm CRYOPDP for 49 million ($58 million).

Cryoport has fundamentally transformed the company via the CRYOPDP and MVE deals, Couillard said in a note, adding the acquisition spree solidifies Cryoports status as one of the best ways to play the coming cell and gene therapy boom.

Speaking on the deal, Jerrell Shelton, CEO of Cryoport, said: The acquisition of MVE Biological Solutions represents an important step in carrying out Cryoports mission. It further entrenches us in the cell and gene therapy supply chain ecosystem at a time when cell and gene therapies are experiencing rapid and sustained growth, and with an even more exciting growth story ahead.

He added: Bringing MVE Biological Solutions under the Cryoport umbrella, which will include Cryoport Systems, Cryogene, and the recently announced agreement to acquire CRYOPDP is expected to increase our revenue run rate to over$160 millionand to be immediately accretive.

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Cryoport strikes second acquisition in a week - Bioprocess Insider - BioProcess Insider

KUALA LUMPUR, Malaysia, Aug. 26, 2020 /PRNewswire/ --Sunway University's Professor Jeff Tan Kuan Onn of the Department of Biological Sciences and Professor Poh Chit Laa from the Centre for Virus and Vaccine Research, along with their research collaborators from Harvard Medical School's Center for Stem Cell Therapeutics and Imaging (USA) as well as University of Tennessee Health Science Centre (USA) have completed a study that has demonstrated the efficacy of molecular gene therapy as a new strategy for cancer treatment.

Professor Jeff Tan Kuan Onn

The research could potentially contribute to shorter treatment time for cancers, reduce treatment costs and minimise the adverse effects of current chemo-drugs in cancer patients such as susceptibilities toward microbial infections, hair-loss and other side effects of chemo-drugs that drastically affect the quality of life of cancer patients undergoing therapy.

Principal Investigator Professor Jeff Tan explained, "Currently, chemo-drugs are relatively ineffective against cancer cells that have developed drug-resistance resulting in the need for high doses of chemo-drugs or a combination of chemo-drugs to be administered to patients with cancer cells. Chemo-drug resistant cancer cells also can spread quickly and that drastically reduce the survival rate of cancer patients".

"Our research utilises molecular gene therapy which is the introduction of genetic materials into cancer cells to promote the sensitivity of cancer cells to chemo-drugs. By genetically engineering the cancer cells, we find that we can induce the cancer cells to produce activated pro-death and tumour suppressor proteins that cause cell death and growth arrests in cancer cells. The weakened cancer cells can then be killed relatively easily by the administration of chemo-drugs in smaller doses. Ultimately, the research could contribute to increasing the survival rates of cancer patients undergoing cancer treatments," he added.

Co-Investigator Professor Poh Chit Laa said that the effectiveness of the strategy has been demonstrated in mice implanted with human breast cancer cells. "In the mice that weretreated with the gene therapy, the tumours obtained from the treated mice showed significant tumour cell death and the tumours were 20 times smaller and 32 times lighter in volume and weight, respectively, when compared to the tumours obtained from the untreated mice. The results indicated that the gene therapy was able to shrink the tumours significantly, even without treatment with chemo-drugs. Small doses of market-available anti-cancer drugs could then be used to kill the cancer cells effectively. We hope to see our research contribute to better survival rates of cancer patients, and minimise the side-affects associated with anti-cancer drugs," said Professor Poh.

"We are currently working on investigations to optimise the delivery of the gene therapy and anti-cancer drugs to human tumours with hopes that this will result in tangible clinical outcomes," said Professor Jeff Tan.

The research project was recently published in the peer-review Journal of Cancer Research and Clinical Oncology. Collaborators for the research include Lee Yong Hoi, Pang Siew Wai and Samson Eugin Simon from the Department of Biological Sciences, Sunway University; Esther Revai Lechtich and Khalid Shah, of the Center for Stem Cell Therapeutics and Imaging, Brigham and Women's Hospital, Harvard Medical School (USA); Suriyan Ponnusamy and Ramesh Narayanan from the Department of Medicine, Centre of Cancer Drug Discovery, College of Medicine, University of Tennessee Health Science Centre (USA).

Story continues

The research is a result of a collaboration agreement between Harvard Medical School and Sunway University aimed at developing new cancer therapies targeting drug resistant cancer cells. In 2016, Professor Jeff Tan visited Harvard University on the Jeffrey Cheah Travel Grant which enabled him to better understand how cancer research projects are conducted as well as examining experimental models used to study cancer biology at Harvard University, Massachusetts General Hospital (MGH), a hospital affiliated with Harvard Medical School, and the Dana-Farber Cancer Institute.

To read the jointly published article: https://link.springer.com/article/10.1007/s00432-020-03231-9

Photo - https://photos.prnasia.com/prnh/20200825/2898392-1

SOURCE Sunway University

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Sunway University's Collaboration with Harvard Medical School Makes Advances in Potential Cancer Treatment using Gene Therapy - Yahoo Finance

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Newly-hired analyst Danielle Brill launched coverage at Raymond James this week with a 490-page survey of her favorite biotech stocks. Atop the list are hematology drugmaker Acceleron Pharma and the gene therapy developer uniQure.

Brill thinks Acceleron (ticker: XLRN) could rise at least 60%, while uniQure (QURE) could nearly double. Shes also partial to the neurology stock Acadia Pharmaceuticals (ACAD), the hematology stock Global Blood Therapeutics (GBT), and Sarepta Therapeutics (SRPT).

Acceleron has two potential blockbusters, writes Brill in her Monday report. In partnership with Bristol Myers Squibb (BMY), the company recently launched sales of an anemia drug Reblozyl. An investigational drug called sotatercept did well in Phase 2 trials for pulmonary arterial hypertension, a serious congenital disease that can require lung transplants.

After its November 2019 launch, sales of Reblozyl rose to $55 million in the June 2020 quarter as a treatment for beta thalassemia and for a particular type of blood cancer. Brill thinks that the drugs annual sales could exceed $3 billion by the end of the decade, yielding annual royalties of over $750 million for Acceleron. As for sotatercept, she thinks its annual sales could hit $2 billion.

So Raymond James rates Acceleron a Strong Buy, and Brill believes its $92 stock could hit $155.

She also has a Strong Buy on uniQure, saying that its stock could rise more than 90% from its recent price of $39, to at least $75. The shares sold off this year after uniQure licensed its investigational treatment for a kind of hemophilia at a price some investors thought was too low. But Brill thinks the deal made commercial sense and gave uniQure cash to advance its just-started clinical trials of a gene therapy for the deadly neurological disorder Huntingtons disease, which killed songwriter Woody Guthrie, among others.

The first couple of Huntingtons patients were dosed with the gene therapy in June and suffered no safety problems. Brill expects uniQure to have efficacy data in 2021. If the Huntingtons gene therapy succeeds, she predicts a $7 billion annual market for uniQure.

Acadia Pharmaceuticals gets an Outperform rating from Brill. The stock sank from $57 to its current level of $38, after the June news that Acadias drug Nuplazid failed a pivotal trial as a treatment for depression. Nuplazid is already approved as a treatment for psychosis from Parkinsons disease and it allowed Acadia to have 2019 revenue of $340 million, with a loss of $235 million, or $1.60 a share.

By April of next year, the U.S. Food and Drug Administration will decide whether to approve Nuplazid for the large market of dementia patients who suffer psychosis. If approved, the added sales could ultimately exceed $3 billion a year, predicts Brill, while bringing Acadia to profitability by 2022. Good news on Nuplazid would lift Acadia stock to her target of $65.

Global Blood is one of a number of companies bringing long-overdue treatments to the blood disorder known as sickle cell disease. Brill rates its stock an Outperform. In December 2019, the company launched the drug Oxbryta and sales have taken off nicely. If the drugs adoption continues, Brill predicts that it could accrue more than 30,000 patients worldwide, brining annual sales close to $2 billion. That would bring in more than $9 a share in earnings by 2025, she says, and merits her price target of $115 for Global Bloods stock, which currently goes for $64.

Another Outperform rating goes to Sarepta Therapeutics, which aims to add a gene therapy to its approved treatments for Duchenne muscular dystrophy. Data on the gene therapys Phase 2 study will come out in the first quarter of 2021. Brill is optimistic, based on improvements seen in the three of the four patients treated in the Phase 1 trial. The stock market reaction to next years Phase 2 results will be dramatic, she predicts, sending Sarepta shares up or down by $100. Shes a believer and has a $200 price target on the $149 stock.

Write to Bill Alpert at william.alpert@barrons.com

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5 Biotech Stocks That Could Be Worth a Look After an Analyst Started Coverage - Barron's

ATLANTA, Aug. 24, 2020 /PRNewswire/ -- Primary HLH is a family of devastating primary immune deficiencies with limited treatment options and no gene therapies under clinical testing. Expression Therapeutics has developed a promising and potentially curative gene therapy candidate for familial hemophagocytic lymphohistiocytosis (HLH) type 3 (FHL3). Untreated, FHL3 presents as a hyper-inflammatory state with multi-organ damage leading to premature death. Expression Therapeutics expects to rapidly progress candidates for other common forms of primary HLH as well.

"We are excited to announce this expansion of our gene and cell therapy pipeline beyond our lead stem cell lentiviral gene therapy candidate for hemophilia A that is entering Phase 1 clinical testing. Through ongoing research and development incorporating our core technology platforms, Expression Therapeutics has been able to rapidly generate promising therapeutic candidates for our HLH portfolio and several other critical disease areas with significant unmet clinical need," said Christopher Doering, Ph.D., Chief Scientific Officer of Expression Therapeutics.

Proof of concept for stem cell lentiviral gene therapy of FHL3 was demonstrated using primary patient cells and a genetic mouse model of FHL3. A key component in this success was the integration of one of Expression Therapeutics' core technology platforms that facilitates the rapid generation of transgenes with superior potency. Our lead candidate successfully restored exocytosis and cytolytic function to primary patient cells as well as a murine disease model strongly supporting the advancement of this pipeline product candidate.

"We believe there are three key aspects to FHL3 that make it a strong candidate for hematopoietic stem and progenitor cell (HSPC) lentiviral vector (LV) gene therapy. First, preclinical and clinical studies suggest that less than 20% genetically corrected cells are required to reverse most FHL3 disease symptoms. Second, because of the autologous nature of stem cell-based lentiviral gene therapy, the grave risk of graft vs host disease is eliminated. Third, with stem cell-based lentiviral gene therapy there will be no wait time to find a sufficiently human leukocyte antigen-matched donor," said Trent Spencer, Ph.D., President of Expression Therapeutics.

According to Deanna Fournier, Executive Director of the Histiocytosis Association, "We are very excited about the possibilities this work offers. Our HLH community, and the entire histiocytosis community, is very hopeful and excited about the future!"

Expression Therapeutics is a biotechnology company based in Atlanta and Cincinnati. The current therapeutic pipeline includes advanced gene therapies for hemophilia, neuroblastoma, T-cell leukemia/lymphoma, acute myeloid leukemia (AML), and primary immunodeficiencies such as hemophagocytic lymphohistiocytosis (HLH).

For inquiries, please contact:

Ashley WalshDirector of Corporate DevelopmentExpression Therapeutics 1860 Montreal RoadTucker, Georgia 30084[emailprotected]+1 312.637.2975www.expressiontherapeutics.com

SOURCE Expression Therapeutics

http://www.expressiontherapeutics.com

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Expression Therapeutics Announces Success in Developing a Stem Cell Lentiviral Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH) - PRNewswire

Audentes Therapeutics has indefinitely delayed plans to seek regulatory approval for its rare disease gene therapy after the death of a third patient involved in a clinical trial of the drug.

This was the third death of a patient involved in the Audentes ASPIRO clinical trial, which is evaluating its AT132 gene therapy in patients with X-linked myotubular myopathy (XLTM), a rare neuromuscular disease.

XLTM mainly affects males and causes muscle weakness that ranges in severity from mild to life-threatening. In severe forms of the disease, the weakened muscle make breathing difficult and can lead to respiratory failure. The genetic disorder has a mortality rate of around 25% by the age of ten.

AT132 is an aden-associated virus (AAV) based gene therapy that is designed to deliver the MTM1 gene deficient in XLMTM. According to Audentes, preliminary findings show that the immediate cause of death of the third patient was gastrointestinal bleeding.

The patient ws one of three study participants who had received AT132 at a dose of 3x1014 vg/kg, the higher dose, who subsequently began to demonstrate signs of liver dysfunction within three to four weeks after initial dosing.

The three patients also demonstrated evidence of pre-existing hepatobiliary disease, although over half of the patients enrolled in the study also showed evidence of the same pre-existing conditions.

Audentes, which was bought by Japanese pharma company Astellas last year, was forced to put the ASPIRO trial on hold in June after disclosing the first two patient deaths.

Audentes, together with the ASPIRO investigators and independent Data Monitoring Committee, continues to closely monitor all patients enrolled in the study. Additionally, Audentes investigation into why these three patients developed progressive liver dysfunction is ongoing, the company said in a statement.

Although the study is currently on hold, Audentes maintained that there are no other patients involved in the trial that are known to be experiencing similar liver disfunction.

The company plans to provide further information on the ASPIRO programme based on both ongoing data collection and future regulatory status updates.

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Audentes' rare disease gene therapy programme indefinitely delayed after third patient death - PMLiVE

Dublin, Aug. 24, 2020 (GLOBE NEWSWIRE) -- The "Global Gene Therapy Market (by Cell Type, Vector Type, Application, End-User & Region): Insights & Forecast with Potential Impact of COVID-19 (2020-2024)" report has been added to ResearchAndMarkets.com's offering.

The global gene therapy market is expected to reach US$ 6.42 billion in 2024, witnessing growth at a CAGR of 19.29%, over the period 2020-2024.

Growth in the gene therapy market has accrued due to the increasing prevalence of chronic diseases, rising healthcare expenditure, expanding urbanization, growth of gene therapy clinical trials and upsurge in economic growth. The market is anticipated to experience certain trends like rapid adoption of personalized medicine, growing occurrence of genetic disorders, advancements in gene therapy and increasing R&D funding. The growth of the market would be challenged by side effects of gene therapy and ethical and safety concerns and high cost of the treatment.

The global gene therapy market has been segmented on the basis of cell type, vector type, application, end-user and region. Depending on the cell type, the market can be bifurcated into somatic cell gene therapy and germ cell gene therapy. According to the vector type, the global gene therapy market can be categorized into retrovirus & gammaretrovirus, adeno-associated viruses (AAV), lentivirus, adenovirus, modified herpes simplex virus and non-viral plasmid vector. Whereas, on the basis of application, the market can be split into oncological disorders, neurological disorders, infectious diseases, cardiovascular diseases, rare diseases and others. Further, in terms of end-user, the global gene therapy market can broadly be segmented into hospitals, specialty treatment centers and other end-users.

The fastest-growing regional market is North America due to the rising incidence of cancer and other target diseases, increasing favorable reimbursement scenario in the region and improvements in healthcare infrastructure. Further, the sudden outbreak of COVID-19 is causing an adverse disruption on the overall economy and society, affecting the rate of gene therapy procedures and clinical trials, which is expected to negatively impact the growth of the global gene therapy market during the forecasted period.

Scope of the report:

Key Topics Covered:

1. Market Overview

2. Impact of COVID-192.1 Economic Impact2.2 Decline in Global GDP2.3 Decline in Industrial Production2.4 Impact on Gene Therapy2.5 Impact on Clinical Trials of Gene Therapy

3. Global Market Analysis3.1 Global Gene Therapy Market by Value3.2 Global Gene Therapy Market Forecast by Value3.3 Global Gene Therapy Market by Cell Type3.3.1 Global Somatic Cell Gene Therapy Market by Value3.3.2 Global Somatic Cell Gene Therapy Market Forecast by Value3.3.3 Global Germ Cell Gene Therapy Market by Value3.3.4 Global Germ Cell Gene Therapy Market Forecast by Value3.4 Global Gene Therapy Market by Vector Type3.4.1 Global Retrovirus & Gammaretrovirus Gene Therapy Market by Value3.4.2 Global Retrovirus & Gammaretrovirus Gene Therapy Market Forecast by Value3.4.3 Global Adeno-Associated Viruses Gene Therapy Market by Value3.4.4 Global Adeno-Associated Viruses Gene Therapy Market Forecast by Value3.4.5 Global Lentivirus Gene Therapy Market by Value 3.4.6 Global Lentivirus Gene Therapy Market Forecast by Value3.4.7 Global Adenovirus Gene Therapy Market by Value3.4.8 Global Adenovirus Gene Therapy Market Forecast by Value3.4.9 Global Modified Herpes Simplex Virus Gene Therapy Market by Value 3.4.10 Global Modified Herpes Simplex Virus Gene Therapy Market Forecast by Value3.4.11 Global Non-Viral Plasmid Vector Gene Therapy Market by Value3.4.12 Global Non-Viral Plasmid Vector Gene Therapy Market Forecast by Value3.5 Global Gene Therapy Market by Application3.5.1 Global Oncological Disorders Gene Therapy Market by Value3.5.2 Global Oncological Disorders Gene Therapy Market Forecast by Value3.5.3 Global Neurological Disorders Gene Therapy Market by Value3.5.4 Global Neurological Disorders Gene Therapy Market Forecast by Value3.5.5 Global Infectious Disease Gene Therapy Market by Value3.5.6 Global Infectious Disease Gene Therapy Market Forecast by Value3.5.7 Global Cardiovascular Diseases Gene Therapy Market by Value3.5.8 Global Cardiovascular Diseases Gene Therapy Market Forecast by Value3.5.9 Global Rare Diseases Gene Therapy Market by Value 3.5.10 Global Rare Diseases Gene Therapy Market Forecast by Value3.6 Global Gene Therapy Market by End-User3.6.1 Global Hospitals & Clinics Gene Therapy Market by Value3.6.2 Global Hospitals & Clinics Gene Therapy Market Forecast by Value3.6.3 Global Specialty Treatment Centers Gene Therapy Market by Value3.6.4 Global Specialty Treatment Centers Gene Therapy Market Forecast by Value3.7 Global Gene Therapy Market by Region

4. Regional Market Analysis4.1 North America4.1.1 North America Gene Therapy Market by Value4.1.2 North America Gene Therapy Market Forecast by Value4.2 Europe4.2.1 Europe Gene Therapy Market by Value4.2.2 Europe Gene Therapy Market Forecast by Value4.2.3 Europe Gene Therapy Market by Indication4.2.4 Europe Large B-Cell Lymphoma Gene Therapy Market by Value4.2.5 Europe Large B-Cell Lymphoma Gene Therapy Market Forecast by Value4.2.6 Europe Inherited Retinal Disease Gene Therapy Market by Value4.2.7 Europe Inherited Retinal Disease Gene Therapy Market Forecast by Value4.2.8 Europe ADA-SCID Gene Therapy Market by Value4.2.9 Europe ADA-SCID Gene Therapy Market Forecast by Value4.2.10 Europe Acute Lymphoblastic Leukemia Gene Therapy Market by Value4.2.11 Europe Acute Lymphoblastic Leukemia Gene Therapy Market Forecast by Value4.3 Asia Pacific4.3.1 Asia Pacific Gene Therapy Market by Value4.3.2 Asia Pacific Gene Therapy Market Forecast by Value4.4 RoW4.4.1 RoW Gene Therapy Market by Value4.4.2 RoW Gene Therapy Market Forecast by Value

5. Market Dynamics5.1 Growth Drivers5.1.1 Increasing Prevalence of Chronic Diseases5.1.2 Rising Healthcare Expenditure5.1.3 Expanding Urbanization 5.1.4 Growth of Gene Therapy Clinical Trials5.1.5 Upsurge in Economic Growth5.2 Key Trends and Developments5.2.1 Rapid Adoption of Personalized Medicines5.2.2 Growing Occurrence of Genetic Disorders5.2.3 Advancements in Gene Therapy5.2.4 Increasing R&D Funding5.3 Challenges5.3.1 Side Effects of Gene Therapy5.3.2 Ethical and Safety Concerns5.3.3 High Cost of Treatment

6. Competitive Landscape6.1 Global Market6.1.1 Revenue Comparison of Key Players6.1.2 Market Capitalization Comparison of Key Players6.1.3 R&D Comparison of Key Players

7. Company Profiles7.1 Roche Holding AG7.1.1 Business Overview7.1.2 Financial Overview7.1.3 Business Strategies7.2 Pfizer Inc.7.2.1 Business Overview7.2.2 Financial Overview7.2.3 Business Strategies7.3 Novartis International AG7.3.1 Business Overview7.3.2 Financial Overview7.3.3 Business Strategies7.4 GlaxoSmithKline PLC7.4.1 Business Overview7.4.2 Financial Overview7.4.3 Business Strategies7.5 Bristol Myers Squibb Co. (Celgene Corporation)7.5.1 Business Overview7.5.2 Financial Overview7.5.3 Business Strategies7.6 Gilead Sciences, Inc.7.6.1 Business Overview7.6.2 Financial Overview7.6.3 Business Strategies

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

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Outlook on the Worldwide Gene Therapy Industry to 2024 - Insights & Forecast with Potential Impact of COVID-19 - GlobeNewswire

It may be possible to protect Parkinson's patients' brains from further damage by turning off a "master regulator" gene, researchers report.

"One of the biggest challenges in treating Parkinson's, other than the lack of therapies that impede disease progression, is that the disease has already laid waste to significant portions of the brain by the time it is diagnosed," said researcher Viviane Labrie, an associate professor at the Van Andel Institute, in Grand Rapids, Michigan.

"If we can find a way to protect critical brain cells from Parkinson's-related damage early on, we could potentially delay or even prevent symptom onset," she suggested in an institute news release.

Deadly for brain cells

Labrie and her colleagues compared the brains of Parkinson's patients and people without the neurodegenerative disease and found that a master regulator gene called TET2 was overactive in the brains of those with Parkinson's. That resulted in a heightened immune response and reactivation of the cell cycle.

While restarting the cell cycle is normal for many types of cells, it's deadly for brain cells, the study authors explained.

The researchers also found that reducing TET2 activity in mouse brains protects brain cells from inflammatory damage and the resulting neurodegeneration seen in Parkinson's disease patients.

These and other findings suggest that lowering TET2 activity could provide a new way to preserve brain cells in Parkinson's patients, according to the authors of the study published in the journal Nature Neuroscience.

A complex disease

For example, reducing TET2 activity might be used after a patient has a major inflammatory event, such as an infection, to relieve residual inflammation without interfering with its normal, healthy role in the body.

"Parkinson's is a complex disease with a range of triggers. Temporarily reducing TET2 activity could be one way to interfere with multiple contributors to the disease, especially inflammatory events, and protect the brain from loss of dopamine-producing cells," Labrie said.

"More work is needed before a TET2-based intervention can be developed, but it is a new and a promising avenue that we already are exploring," she concluded.

Image credit: iStock

Excerpt from:
Could gene therapy stem the damage of Parkinson's? - Health24

The Food and Drug Administrations rejection of a gene therapy for hemophilia A on Wednesday surprised many hematology researchers and Wall Street watchers who expected speedy approval for the one-time treatment to end the inherited bleeding disorder.

For one family in Indianapolis active in the hemophilia patient community, the decision was disappointing, but also appreciated.

Its a sad day because for a lot of people, they were ready to go, Michelle Rice, whose two sons have severe forms of hemophilia A, told STAT. But its also a good day, said Rice, who has a mild case of the disease and serves as chief external affairs officer for the National Hemophilia Foundation, because I think this community has fought long and hard for safety to be a priority.

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Hemophilia A is a genetic disorder in which the body fails to produce a protein called Factor VIII that is crucial for blood clotting. It affects about 20,000 people in the U.S., almost all men. The bleeding episodes and joint damage it causes are kept under control by frequent infusions costing about $300,000 per year.

BioMarins gene therapy, called Roctavian, is designed to fix that inherited defect, but the therapys impact on Factor VIII levels seems to wane over time. A year after treatment, patients in BioMarins clinical trial had Factor VIII levels of 64.3 international units per deciliter on average. After two years, that number fell by more than half. Four years after treatment, the average was 24.2 IU/dL, a 63% decline from the first year.

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The FDA has asked for two more years of data to better establish the durability of the gene therapy. That means the one-time treatment, if approved, will be delayed until at least 2022.

Roctavian is one of three gene therapies for hemophilia A now being developed. Roche, which acquired Spark Therapeutics gene therapy program when it bought the biotech earlier this year, and the partnership of Pfizer and Sangamo Therapeutics are at work on similar one-time therapies. Each is a year or more away from filing for FDA approval.

Len Valentino, CEO of the National Hemophilia Foundation, applauded the FDAs push for more durability data. He is a hematologist and former vice president, strategy lead in hematology, at Spark.

I think its good for the patient community because we need to protect our patients at all costs, he said. This is a community thats been through a tremendous amount of grief in the past with first HIV and AIDS and then hepatitis C. So I think protecting our community is of the utmost importance. And at this point, theres just too many unknowns that we dont understand the answers to.

In the 1970s and 1980s, about half of all people with hemophilia became infected with HIV after being transfused with contaminated blood products. Many patients with severe hemophilia developed AIDS, and thousands died. Infection with hepatitis C was also common when patients relied on infusions of clotting factors from human plasma, before the availability of recombinant factor VIII and IX made their treatments safer.

In the 80s, our population was decimated by HIV. Then in the late 80s, early 90s, a lot of them got hepatitis C, Rice said. My older son was one of them.

The more than a dozen products now available are safer, she said, and the treatments have made a huge difference for her older son, now 30, preventing the bleeding episodes that used to mean he was in the hospital every 7 to 10 days. Her younger son, who is 25, has fared better. Spontaneous bleeds are rare and prophylactic infusions several times a week have become as routine as brushing your teeth, Rice said.

Right now, were comfortable with where we are, she said. Were comfortable with the way our bodies feel.

Valentino called current therapies to treat hemophilia good, safe, and effective. At this point, the unmet medical need for hemophilia A is not great because I think the standard of care is quite good at this point.

There are two other sticking points for Rice and her sons, who arent pinning their hopes on any gene therapy: It doesnt change the genes you pass down to your children. And she wonders how long its benefits will last.

Everybody wants to see gene therapy. I can tell you that from the time my oldest was born, I was hearing gene therapy is going to be here in 10 years, she said. Its not that people in the community are not excited about it. I think they very much are excited about it. I feel like were probably closer than weve ever been getting here. And my kids have said were not going to rule it out forever.

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Wary hemophilia patients are willing to wait longer for a safe gene therapy - STAT