FDA official announcements and press releases

Bilateral Gene Therapy Restores Hearing in Five Deaf Children with OTOF Mutation

Jun 5, 2024, 01:31 PM

A pioneering gene therapy trial has restored hearing in five children who were born deaf, according to recent reports. The trial, conducted by researchers in China and the U.S., used an adeno-associated virus (AAV)-based therapy to deliver a normal copy of the OTOF gene, which is mutated in children with DFNB9, a condition responsible for 2-8% of all congenital hearing loss. The therapy has shown promising results, with children being able to hear and even dance to music after the treatment. The trial's success has raised hopes that this approach could be used to treat other forms of genetic or nongenetic deafness. Preliminary data, published in NatureMedicine and TheLancet, suggest that the bilateral therapy could enhance speech perception, sound source localization, and overall auditory experience. The therapy is described as groundbreaking and has been tested on children with autosomal deafness.