Regeneron’s gene therapy restores hearing in children with genetic deafness

Regeneron’s experimental gene therapy, DB-OTO, significantly improved hearing in children with a rare genetic form of deafness, with most regaining moderate to normal hearing.

Why it matters: If approved, DB-OTO could become the first gene therapy for congenital deafness, potentially replacing lifelong reliance on cochlear implants for thousands of children worldwide.

Backstory: Some babies are born profoundly deaf due to mutations in a gene crucial for hearing. Regeneron’s therapy delivers functional copies of that gene directly into the inner ear via the same procedure used for surgical implants.

Zoom in: In a study of 12 children, 9 reached moderate to normal hearing levels within 6 months. One toddler progressed to normal hearing, identifying spoken words without visual cues. The therapy proved effective across different ages, not just in very young children, as previously thought. Despite the drug’s efficacy, minor side effects were typical of ear surgeries; two serious events resolved without lasting issues.

Big picture: The success of DB-OTO signals a major leap in gene therapy, particularly for neurological and sensory disorders. It also reflects Regeneron’s growing push into genetic medicine, amid a competitive but cooling biotech market.

What’s next: Regeneron plans to file for FDA approval by year-end, facing competition from companies like Eli Lilly's Akouos. A possible obstacle could be the still unknown long-term durability that insurers are concerned about, given the expected high price tag.