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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The FDA gives the green light to the first gene therapy for deafness
The Food and Drug Administration approved the first gene therapy to restore hearing for people who were born deaf.The decision, while only immediately affecting people born with a very rare form of genetic deafness, is being hailed as a milestone in the quest to treat hearing loss.“It’s the first time in history there’s a new drug for hearing loss,” says Zheng-Yi Chen, an associate scientist at Mass Eye and Ear in Boston who was not involved in …
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