On April 22, 2024 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that new and updated data across its genetic medicines portfolio will be presented at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) annual conference in Baltimore, Maryland, from May 7 to 11, 2024 (Press release, Regeneron, APR 22, 2024, View Source [SID1234642201]). 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.

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"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 (Free ASGCT Whitepaper) 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 (Free ASGCT Whitepaper) 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 genetic medicines for more patients and diseases."

Regeneron presentations at ASGCT (Free ASGCT Whitepaper):

Abstract title Abstract Presenting/Lead
Author Presentation
date/time
(ET)
Oral Presentations
Intracochlear Administration of DB-OTO Gene Therapy in Pediatric Patients with Profound Hearing Loss Due to Otoferlin Mutations: The CHORD Phase 1/2 Open-Label Trial 10 Lawrence Lustig, Columbia University Wednesday, May 8, 9:30-9:45AM ET

Antibody-Based AAV Retargeting to Transferrin Receptor Mediates Efficient Blood Brain Barrier Crossing and In Vivo Gene Delivery to the CNS in Mice and Non-Human Primates 118 Kalyani Nambiar Wednesday, May 8, 4:15-4:30PM ET

Targeted Gene Insertion of Vectorized Monoclonal Antibodies in Non-Human Primates Overcomes AAV Genome Silencing in the Liver and Supports High, Sustained In Vivo Expression of Functional Antibodies 197 Rachel Sattler Thursday, May 9, 5:15-5:30pm ET
Retargeting of AAV Using Bispecific Antibodies 218 Sven Moller-Tank Thursday, May 9, 5:15-5:30PM ET
Tissue De-Targeting Abrogates Hepatotoxicity and Complement-Related Thrombotic Complications Associated with High-Dose AAV Gene Therapies 298 Andrew Baik Friday, May 10, 4:15-4:30PM ET
Orthogonal B Cell and Plasma Cell Immunosuppression Strategies Prevent and Suppress High-Titer Antibody Immunity to Enable AAV Vector Re-Dosing 353 Nicholas Giovannone Saturday, May 11, 8:15-8:30AM ET
Poster Presentations
AAV Conjugated to Antibodies Against p75NTR: A New Platform to Deliver Pain Therapeutics to Nociceptive Sensory Neurons 638 Adina Buxbaum Wednesday, May 8, 12:00-7:00PM ET
A Process for Identifying AAV and Transgene Integrations in Mouse and Human Genomes Using Long Read Oxford Nanopore Sequencing 897 Terrence Turner Wednesday, May 8, 12:00-7:00PM ET
DNA Leakage of rAAV Under Freeze/Thaw Stress and Analytical Method Development for Free DNA Characterization 898 Shuai Li Wednesday, May 8, 12:00-7:00PM ET
Identification of Degradation Pathways of rAAV8 to Aid Stable Drug Product Formulation Development 899 Ariel Chen Wednesday, May 8, 12:00-7:00PM ET
Lectures
Engineering CAR-T Cells with Novel Receptor Architectures N/A Philip Gregory Thursday, May 9, 10:55-11:25AM ET

Pressing Challenges in Gene Therapy N/A Jim Wang Saturday, May 11, 8:00-9:45AM ET