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Regeneron wins FDA approval for first gene therapy for genetic hearing loss

Clinical trial data showed 90% of participants improved, and some children later learned to speak, researchers said.

  • Researchers reported Wednesday in the journal Nature that experimental gene therapy successfully restored hearing in people born with a rare form of deafness caused by OTOF gene mutations.
  • The OTOF gene encodes otoferlin, a protein critical for translating sound vibrations into electrical signals the brain can interpret; without it, children are born completely deaf.
  • About 90 percent of recipients saw hearing improvements, with the study involving participants aged nine months to 32 years and reporting no serious side effects or dose-related toxicity.
  • Recipients showed improved speech perception, with some children learning to speak for the first time; gene therapy may eventually prove superior to mechanical Cochlear implants requiring maintenance.
  • Regeneron Pharmaceuticals plans to file for regulatory approval, potentially becoming the first gene therapy for deafness to clear the Food and Drug Administration within the next year.
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Nature broke the news in United Kingdom on Wednesday, April 22, 2026.
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