In what scientists are calling a watershed moment for genetic medicine, a clinical trial has demonstrated that a single gene therapy treatment can restore functional hearing in children born with a specific form of hereditary deafness, with five of six participants achieving hearing within the normal range after treatment.
The trial, conducted across four research centers in the United States and United Kingdom, targeted mutations in the OTOF gene, which encodes a protein essential for transmitting sound signals from hair cells in the inner ear to the auditory nerve. Children without functional copies of the gene are born profoundly deaf.
The treatment uses a modified virus to deliver a functional copy of the OTOF gene directly to hair cells in the cochlea. The procedure is performed under anesthesia and takes approximately two hours. Side effects were mild and transient in the trial population, all of whom were under 18 months of age at the time of treatment.
The Food and Drug Administration has granted the therapy Breakthrough Designation, a status that accelerates the review process for treatments addressing serious conditions. Researchers estimate a full regulatory submission could come within 18 months if ongoing follow-up data remains consistent.
