Category: News

Jerry Mendell Award recognizes Dr. High for her outstanding work to bring cell and gene therapies to patients Research Triangle Park, N.C. – May 19, 2022 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that Dr. Katherine High, President of Therapeutics, received the Jerry Mendell award for Translational Science from the American Society of Gene and Cell Therapy (ASGCT).  Recently inducted as a member of the National Academy of Sciences (NAS), Dr. High has received this award in recognition of her significant contributions to the field of genetic medicine. “I am honored to receive this recognition from ASGCT,” said Dr. Katherine High. “I have dedicated my career to building the translational science that enables us to move scientific discoveries to new medicines for patients.  I look forward to furthering this work at AskBio and with our many partners to find the answers for devastating diseases that currently lack disease-modifying treatments.” Dr. High joined AskBio in 2021 and is internationally recognized for her pioneering bench-to-bedside research and clinical success for the clinical translation of genetic therapies for multiple inherited disorders.  Most notable, while serving as the Inaugural Director of the Center for Cellular and Molecular Therapeutics at Children’s Hospital of Philadelphia (CHOP), Dr. High led a multidisciplinary team of scientists and physicians to spin out Spark Therapeutics, where she led the team that achieved the first U.S. Food and Drug Administration approval of a gene therapy for a genetic disease. Today, at AskBio, Dr. High oversees a robust pipeline of gene therapeutics to treat various neuromuscular, central nervous system, cardiovascular and metabolic disorders, all at various stages of clinical development. “As a true pioneer in the field, author of more than 200 scientific papers with multiple patents in gene therapy, Dr. High is one of the most accomplished persons in gene therapy today,” added Sheila Mikhail, co-founder, and Chief Executive Officer of AskBio. “I am privileged to have her on our team and, on behalf of all of us at AskBio, we congratulate her for this esteemed award and her continued accomplishments in genetic medicine.” The Jerry Mendell Award for Translational Science award is named for Jerry Mendell, MD, the first person in collaboration with Askbio co-founder Dr. R. Jude Samulski, to study viral mediated gene therapy for muscular dystrophy in humans and recognizes the extensive work required to bring gene and cell therapies to clinical trial. Mendell was also the principal investigator who brought Zolgensmaâ, a gene therapy treatment for Spinal Muscular Atrophy that uses the recombinant adeno-associated virus (rAAV) vector technologies created by AskBio founder and Chief Scientific Officer, Dr. R. Jude Samulski. It is also the same technology that is used for Luxturnaâ, the FDA approved gene therapy brought to market by the team led by Dr. High while at Spark Therapeutics. Dr. High was acknowledged on May 17, 2022, as part of the ASGCT’s 25th annual meeting, the premier event bringing together over 4,800 noted professionals and experts in gene and cell therapy in the United States and

Presentations convey essential technological and scientific knowledge regarding AAV gene therapy and advancements across key disease areas  CEO Sheila Mikhail and President of Therapeutics, Katherine A. High will be featured speakers Research Triangle Park, N.C. – May 16, 2022 –Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that the Company will present 11 abstracts at the upcoming American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting being held May 16 – 19, 2022 at the Walter E. Washington Convention Center  in Washington, D.C.  The ASGCT Annual Meeting is the premier event for professionals in gene and cell therapy where noted industry professionals gather to learn from the latest advances in scientific and clinical research and cell and gene technology. Abstracts being presented by AskBio team members include new data and insights regarding Adeno Associated Virus (AAV) gene therapy, T-cell immune response to empty capsid technologies, inducible promoters and gene expression, and doggybone DNA (dbDNA) as well as data for AskBio’s key clinical development programs, including Pompe Disease, Parkinson’s Disease and Congestive Heart Failure.   AskBio will make seven oral presentations and four poster presentations. CEO Sheila Mikhail and President of Therapeutics, Katherine A. High will be featured speakers during the event. “Having 11 abstracts accepted for presentations reflects the significant progress made by our teams across a broad front,” commented Kathy High, President, Therapeutics for AskBio. “I am very proud of the groundbreaking work by our research and clinical teams as we continue to advance our therapeutic pipeline and AAV gene therapy research and manufacturing.”  Jude Samulski, Chief Science Officer and Co-Founder for AskBio said, “These presentations underscore our commitment to advancing the science of gene therapy to tackle many of the biggest challenges in the space today, including manufacturing, dosing, immune response and treatment efficacy. We hope that, together with our many colleagues in cell and gene therapy space, we can make a profound difference in the lives of patients around the world who are waiting for transformative gene therapies.”  AskBio’s presentations at ASGCT include:  Monday, May 16 Oral PresentationAbstract 37: Functional Assessment of T-cell Responses to AAV8 Empty Capsids in Healthy VolunteersSession: Immune Responses to AAV Vectors 10:30 am – 11:45 am, Room: 102 Oral PresentationAbstract 28: A First-in-Human Phase 1 Clinical Gene Therapy Trial for the Treatment of Heart Failure Using a Novel Re-Engineered Adeno-Associated Vector Session: Cardiovascular and Pulmonary Diseases 11:45 am – 12 PM, Room 206 Featured Speaker Launching Innovation Into Gene Therapy Companies Session: Part 2: Translating Science Into Medicine: Moving from Bench to Startup (Organized by the Bioindustry & Translational Science Committees) Sheila Mikhail, JD, CEO and Co-Founder, AskBio 1:30–2:18 PM Room: Salon G Tuesday, May 17 Oral Presentation: Abstract 434: Characterization of Alternative Reading Frame Proteins Generated from AAV Cassettes Session: Discoveries in Fundamental AAV Biology 4:00–4:15 PM, Ballroom A Poster Presentation: Abstract 796: Safety and preliminary efficacy of neurosurgical AAV2-GDNF delivery for Parkinson’s disease Session:  Gene and Cell Therapy Trials in Progress 5:30PM, Hall D            Poster Presentation: Abstract 711:

AskBio announced that it has been honored as one of the Triangle Business Journal’s (TBJ) 2021 Life Sciences Award winners in the Company of the Year category.