Regeneron Pharmaceuticals, Inc. recently shared encouraging early results from the ongoing Phase I/II CHORD trial of DB-OTO, a gene therapy aimed at treating genetic deafness caused by variants of the otoferlin gene. The data was presented at the American Society of Gene and Cell Therapy (ASGCT) annual meeting in Baltimore, detailing significant hearing improvements in young patients.

The trial focuses on DB-OTO, designed to correct hearing loss linked to otoferlin deficiencies. Otoferlin is crucial for the transmission of sound information from the inner ear to the brain. Two young participants, one aged 11 months and the other 4 years at the time of treatment, exhibited remarkable hearing improvements after receiving a single intracochlear injection of the therapy.

The younger child achieved normal hearing levels across essential speech frequencies within 24 weeks of treatment, marking one of the youngest global instances of gene therapy application for genetic deafness. The second child also showed notable progress, with initial improvements observed just six weeks post-treatment.

Dr. Lawrence R. Lustig, from Columbia University’s Department of Otolaryngology–Head & Neck Surgery and a trial investigator, emphasized the significant potential of DB-OTO. According to him, the therapy could offer a comprehensive auditory experience to children with profound genetic deafness—a prospect previously considered unlikely within his professional lifetime.

The CHORD trial is a first-in-human, multicenter, open-label study assessing the safety, tolerability, and preliminary efficacy of DB-OTO. It includes participants from the United States, United Kingdom, and Spain, and involves a two-part protocol. Initially, subjects receive the gene therapy in one ear, followed by treatment in both ears based on the most effective dose identified in the first part.

DB-OTO has already garnered Orphan Drug, Rare Pediatric Disease, and Fast Track Designations in the U.S., with similar recognition from the European Medicines Agency, reflecting its potential as a critical intervention for a rare and challenging condition.

As Regeneron continues to enroll participants in the CHORD trial across various international sites, the early successes of DB-OTO offer hope for addressing the root cause of profound genetic deafness, potentially transforming the developmental trajectory and quality of life for affected children.

Treating Genetic Deafness
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