Guess the Therapy Answer March 25, 2026
Which gene therapy is currently under development by Regeneron for otoferlin-related hearing loss?
Answer: DB-OTO.
See below for further reading on DB-OTO:
October 13, 2025 — Regeneron’s DB-OTO, an investigational adeno-associated virus (AAV) dual-vector-based gene therapy intended to treat otoferlin (OTOF)-related hearing loss, has improved hearing and speech perception in pediatric patients treated in the phase 1/2 CHORD clinical trial (NCT05788536), according to updated data reported by thecompany at the annual American Academy of Otolaryngology-Head and Neck Surgery (AAO-HNSF) meeting, held October 11 to 14 in Indianapolis, Indiana, and simultaneously published in the New England Journal of Medicine (NEJM).
Among 12 patients treated in the pivotal CHORD study, 11 patients (14 of 15 treated ears) displayed clinically meaningful improvements in hearing, with 3 of the patients showing normal hearing levels. As such, the study met its primary end point, which was set at 9 patients showing improvements in hearing at a threshold of 70 decibels hearing level or less (dBHL), as evaluated at 24 weeks posttreatment by behavioral pure tone audiometry. Regeneron noted that the patients responded within weeks of receiving the gene therapy.
May 17, 2024 — Decibel Therapeutics and Regeneron are currently evaluating an AAV vector-based gene therapy, referred to as DB-OTO, for the treatment of OTOF) gene-related hearing loss in the first-in-human phase 1/2 CHORD clinical trial (NCT05788536). Data from the first 2 patients in the trial were presented by Lawrence R. Lustig, MD, the chair of the Department of Otolaryngology—Head and Neck Surgery at Columbia University College of Physicians, at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD.
Following his presentation, CGTLive® sat down with Lustig to learn more about the implications of the promising early findings. Lustig emphasized that these positive results may encourage more innovation in the field and eventually lead to more genetic testing in children with hearing loss and potentially spur on the development of gene therapies for other forms of genetic deafness.
