New Gene Therapy Trial Restores Hearing and Speech in Children Born Deaf, Treated in Both Ears; Results Termed “Astounding”

Administering gene therapy in both ears of five children with DFNB9 led to additional benefits compared to previous trial in one ear, including sound source localization and hearing in noisy environments.

Dr. Yilai Shu examines young patient

Key Takeaways

  • A new clinical trial in Shanghai, China, in partnership with Mass Eye and Ear researchers, demonstrated the restoration of hearing in both ears in five children born with autosomal deafness caused by mutations in the OTOF.
  • All children gained ability to determine locations that sounds came from and improved speech perception in noisy environments.
  • The trial is the first to provide gene therapy for this form of inherited deafness to children in both ears, and the researchers hope to expand this work internationally.
A novel gene therapy designed to target a form of inherited deafness restored hearing function in five children who were treated in both ears. The children also experienced better speech perception and gained the ability to localize and determine the position of sound. The study, the world’s first clinical trial to administer a gene therapy to both ears (bilaterally), demonstrates additional benefits than what were observed in the first phase of this trial, published earlier this year, when children were treated in one ear. The research was led by investigators from Mass Eye and Ear (a member of the Mass General Brigham healthcare system) and Eye & ENT Hospital of Fudan University in Shanghai, and findings were published June 5,2024 in Nature Medicine. The open-access article is titled “Bilateral Gene Therapy in Children with Autosomal Recessive Deafness 9: Single-Arm Trial Result.”
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