FDA Approves First-Ever Gene Therapy for Deafness, Opening Door to a New Era

The FDA approved the first gene therapy for hereditary deafness, targeting OTOF mutations that cause a rare form of hearing loss.

In a small trial, most treated infants, children, and teens improved their hearing, and some reached normal hearing levels.

Side effects were minor and temporary, suggesting the treatment was generally well tolerated.

The decision could open a new treatment path for children born with genetic hearing loss.