Press release
Regeneron to Highlight Advances in Genetic Medicine Research at American Society of Gene and Cell Therapy (ASGCT)
Oral presentations include updated results from clinical study of otoferlin gene therapy DB-OTO demonstrating restoration in children with profound genetic
About this update from Regeneron Pharmaceuticals, Inc.
[{"type":"text","content":"Oral presentations include updated results from clinical study of otoferlin gene therapy DB-OTO demonstrating restoration in children with profound genetic hearing loss Additional presentations cover progress in novel genetic medicine delivery systems and immune response modulation TARRYTOWN, N.Y., April 22, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that new and updated data across its genetic medicines portfolio will be presented at the American Society of Gene and Cell Therapy (ASGCT) annual conference in Baltimore, Maryland, from May 7 to 11, 2024. Data from 10 abstracts, including six oral presentations, provide insight on Regeneron’s approach to overcoming obstacles to clinical implementation of genetic medicines, from pre-dosing to delivery to long-term sustained expression. The company will also present updated data from the Phase 1/2 CHORD trial investigating DB-OTO in children with profound genetic hearing loss due to mutations of the otoferlin gene. “Genetic medicine approaches including gene therapy, gene editing and gene silencing hold incredible promise for people with serious, genetically driven diseases, but some common barriers to practical implementation remain, such as delivery to tissues beyond the liver and waning efficacy over time,” said Christos Kyratsous, Ph.D., Senior Vice President and Co-Head of Regeneron Genetic Medicines. “Regeneron continues to advance methods to overcome these obstacles through our proprietary delivery approaches utilizing next-generation viral vectors, particularly specific retargeting antibodies and innovative payloads. Our data at ASGCT also details efforts to sustain expression of treatment over time and better modulate immune response via adeno-associated virus delivery.” “We are continuing to dose patients in our clinical trial of DB-OTO gene therapy for profound hearing loss due to otoferlin deficiency and are advancing additional gene therapy programs toward the clinic. The ASGCT presentation will build on promising early results in the first patient,” said Aris Baras, M.D., Senior Vice President, Co-Head of Regeneron Genetic Medicines and Head, Regeneron Genetics Center®. “These results raise hope and enthusiasm for the field, and we believe that findings from the program will help us unlock paths forward for gene therapies and...