Some deaf children are hearing again because of a new gene therapy
A new gene therapy called Otarmeni, approved by the FDA in April 2026, has restored hearing in children with severe-to-profound hearing loss caused by OTOF gene mutations. Clinical trials showed 80% of patients gained measurable hearing, with effects lasting over two and a half years. The success marks a resurgence in gene therapy, a field once derailed by safety concerns following a 1999 trial death.
- ▪The FDA granted accelerated approval to Otarmeni, a gene therapy developed by Regeneron, on April 23, 2026.
- ▪In clinical trials, 80% of patients treated with Otarmeni gained measurable hearing, and 42% could hear whispers.
- ▪The therapy targets mutations in the OTOF gene, which cause congenital hearing loss.
- ▪The treatment uses adeno-associated viruses (AAV) to safely deliver functional genes, avoiding the immune reactions seen in earlier gene therapies.
- ▪Dr. Yilai Shu of Fudan University co-led the trial, which included children who began responding to sound within weeks of treatment.
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Future PerfectSome deaf children are hearing again because of a new gene therapyA medical field that almost died is quietly fixing one disease at a time.by Bryan WalshMay 2, 2026, 11:45 AM UTCShareGift This is what gene therapy can do in 2026. Svetlana Repnitskaya/Getty ImagesBryan Walsh is a senior editorial director at Vox overseeing the climate teams and the Unexplainable and The Gray Area podcasts. He is also the editor of Vox’s Future Perfect section and writes the Good News newsletter. He worked at Time magazine for 15 years as a foreign correspondent in Asia, a climate writer, and an international editor, and he wrote a book on existential risk.In a lab room, a toddler, deaf from birth, sits while a tone plays. There’s no reaction.
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