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AAV-OTOF Gene Therapy Restores Hearing From Infancy to Adulthood

Single-dose delivery of a synthetic AAV vector through the cochlear round window has produced rapid, sustained hearing improvements in trial participants.

Image
Audiologist doing impedance audiometry or diagnosis of hearing impairment. (© Dusko - stock.adobe.com)
© PeopleImages.com - Yuri A via Shutterstock
A woman holding her ear, trying to hear.

Overview

  • All ten patients aged 1 to 24 experienced measurable hearing restoration following AAV-OTOF treatment.
  • Average sound thresholds improved from 106 decibels to 52 decibels by six months after therapy.
  • Most participants recovered some hearing within one month of receiving the inner-ear injection.
  • No serious adverse events were reported during six- to twelve-month safety monitoring.
  • Researchers will pursue five- to ten-year follow-up and adapt the AAV platform to other deafness genes such as GJB2 and TMC1.