Regeneron, with ‘game-changing’ new data, to seek approval of hearing loss gene therapy

Regeneron Seeks Approval for Hearing Loss Gene Therapy

Regeneron will seek approval for a gene therapy that has shown promising results in treating a rare, genetic form of deafness.

A study published in The New England Journal of Medicine found that the therapy significantly improved hearing in nearly a dozen children with this condition.

The gene therapy targets a genetic form of hearing loss that affects a small number of babies born each year, who often rely on cochlear implants for their entire lives.

The gene normally makes a protein that the hairs in our inner ears need to relay sound signals to the brain.

According to the study results, most of the children who received the gene therapy are now hearing well enough to not need help from implants.

Author's summary: Regeneron's gene therapy shows promise in treating rare hearing loss.

more

BioPharma Dive BioPharma Dive — 2025-10-12