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First Gene Therapy for Hearing Loss
April 27 , 2026
13 hrs 0 min
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- The Food and Drug Administration (FDA) of USA approved the first gene therapy for genetic hearing loss.
- The therapy named Otarmeni targets deafness caused by mutations in the OTOF (otoferlin) gene.
- It uses dual Adeno-Associated Virus (AAV) vectors to deliver a healthy copy of the gene into inner ear (cochlea) cells.
- The treatment restores the protein otoferlin, enabling proper transmission of sound signals from the ear to the brain.
- It is a one-time surgical therapy administered directly into the inner ear and has shown improved hearing in clinical trials.
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