Oct. 27, 2023
TARRYTOWN, NEW YORK – Regeneron Pharmaceuticals, Inc. reported promising safety and efficacy outcomes from the first patient under the age of two enrolled in the Phase 1/2 CHORD trial. This trial focuses on otoferlin gene therapy (DB-OTO) for children with profound genetic hearing loss stemming from otoferlin gene mutations. Children with profound hearing loss due to otoferlin gene mutations face