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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 hearing loss

Additional presentations cover progress in novel genetic medicine delivery systems and immune response modulation

New York, US -- 22 April 2024 -- 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, US from 7 to 11 May 2024. Data from ten 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 PhD, senior vice president and co-head of Regeneron Genetic Medicines. “Regeneron continues to advance methods to overcome these obstacles through our proprietary delivery approaches utilising 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 programmes toward the clinic. The ASGCT presentation will build on promising early results in the first patient,” said Aris Baras MD, head, Regeneron Genetics Center and senior vice president, co-head of Regeneron Genetic Medicines. “These results raise hope and enthusiasm for the field, and we believe that findings from the programme will help us unlock paths forward for gene therapies and genetic medicines for more patients and diseases.”

Regeneron presentations at ASGCT:

About Regeneron

Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, many of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, hematologic conditions, infectious diseases and rare diseases.

Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, including VelociSuite® which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases.

For more information, please visit or follow Regeneron on LinkedIn, Instagram, Facebook or X.