Regeneron Gene Therapy Dramatically Improves Hearing in Deaf Children

Regeneron Pharmaceuticals presented promising results for their investigational gene therapy, DB-OTO, at the 2024 American Society of Gene & Cell Therapy annual meeting in Baltimore. The therapy showed significant improvements in hearing for two children who were born with "profound genetic deafness."

One of the children, treated at 11 months old, returned to normal hearing levels within 24 weeks of receiving DB-OTO. This was measured using auditory brainstem response (ABR) and pure tone audiometry (PTA), both considered validated methods for assessing hearing function. Regeneron noted that this patient is among the youngest globally to receive gene therapy for genetic deafness. PTA assessments at the 24-week mark revealed an average improvement of 84 decibels (dB) across key speech frequencies, with one frequency achieving a hearing level of 10 dB.

The second child, treated at age four, showed initial hearing improvements at the six-week follow-up, responding to loud sounds. This patient's average improvement across key speech frequencies was 19 dB from baseline.

Dr. Lawrence Lustig, the study investigator and chairperson of Columbia University's Department of Otolaryngology-Head & Neck Surgery, described these initial outcomes as "impressive," highlighting the revolutionary potential of DB-OTO for treating otoferlin-related deafness.

These findings are part of the ongoing Phase I/II CHORD trial, an open-label, first-in-human study that is enrolling participants in the U.S., U.K., and Spain. Alongside efficacy, the trial also evaluated the safety of DB-OTO, finding it well-tolerated with no serious treatment-related adverse events.

DB-OTO is designed to be administered through a single ear injection. It is a cell-selective, AAV-delivered gene therapy that works by providing a functional copy of the otoferlin gene, which is mutated and non-functional in patients with genetic hearing loss. According to Regeneron, congenital hearing loss, affecting about 1.7 out of every 1,000 children born in the U.S., represents a significant unmet medical need. The otoferlin gene is often involved in these cases.

The encouraging early results from the CHORD trial place Regeneron among other innovators in the field of genetic hearing restoration. In January 2024, Eli Lilly reported that its investigational otoferlin gene therapy, AK-OTOF, restored hearing in an 11-year-old boy within 30 days of treatment. Similarly, a Chinese research group published findings showing hearing restoration in five out of six children treated with their own otoferlin gene therapy, as documented in The Lancet.

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