The short version
- A Cincinnati brother and sister born deaf from a faulty otoferlin gene gained hearing after a single gene therapy given in a clinical trial.
- The therapy, Otarmeni, won accelerated FDA approval in April after 16 of 20 trial participants reached 70 dB hearing or better.
- It treats only one rare cause of deafness, about 1% of congenital cases, and Regeneron offers it free in the US.
Abdu was 3 when he received the gene therapy at Cincinnati Children’s Hospital in 2025, in the trial’s second group. Within four weeks he went from hearing almost nothing to detecting sounds, and the hospital says he now hears in the normal range and is learning to speak. His sister Sumaya was treated at 1, and her hearing has, in the hospital’s words, “dramatically improved”. “For us, it was a moment of real amazement, because it’s something that we would have never really expected to happen in our career, for kids to be able to hear without the need of devices,” said John Greinwald Jr., MD, of the hospital’s otolaryngology division.
Both children carry two faulty copies of the OTOF gene, which makes otoferlin, a protein the inner ear’s hair cells need to pass signals to the hearing nerve. The therapy, Regeneron’s Otarmeni, is the first approved gene therapy for a genetic form of hearing loss. The hospital cites the first 12 participants reported in the New England Journal of Medicine, of whom 9 reached 70 dB or better by 24 weeks. The FDA’s accelerated approval on April 24 rested on a larger count: 16 of 20. Regeneron must keep collecting data as a condition of that approval.
The release does not say which ears were treated. Regeneron makes the therapy available free in the US, but it treats one cause only: OTOF variants account for about 1% of congenital deafness.
