Key points from article :
Regeneron’s experimental gene therapy, DB-OTO, has shown promising results in improving hearing in children with a rare genetic form of deafness. In a phase 1/2 trial known as CHORD, 10 out of 11 treated children experienced significant hearing improvements. The therapy, designed for children with otoferlin-related hearing loss, delivers a functional gene via an intracochlear injection, a procedure similar to cochlear implantation.
Early results indicate that DB-OTO can restore hearing to nearly normal levels in some cases, with one child developing speech recognition after previously being completely deaf. The therapy has been well tolerated, with no serious safety concerns directly linked to DB-OTO, though some participants experienced temporary post-surgical effects like dizziness and nausea.
Regeneron’s findings offer new hope for treating genetic hearing loss, a condition that currently has limited options beyond cochlear implants. The FDA has recognized DB-OTO’s potential by granting it multiple fast-track designations. While further studies are needed, researchers believe this marks the beginning of a new era in precision medicine for hearing loss, with future applications potentially extending to age-related and noise-induced deafness.
