Shanghai, China - June 5, 2024

In a groundbreaking advancement for the field of genetic hearing loss, a new gene therapy trial has shown remarkable success in restoring hearing and speech in children born deaf. Administering gene therapy in both ears of five children with DFNB9, a form of inherited deafness, led to unprecedented benefits, including restored hearing function, enhanced speech perception, and the newfound ability to localize sound sources.

The trial, hailed as the world's first clinical trial to administer gene therapy bilaterally, was led by investigators from Mass Eye and Ear and the Eye & ENT Hospital of Fudan University in Shanghai. Published in Nature Medicine, the findings unveil the transformative potential of this therapeutic approach in addressing congenital deafness.

Lead study author Yilai Shu, MD, PhD, emphasized the significance of treating children in both ears to achieve comprehensive hearing recovery. By targeting mutations in the OTOF gene, which impair the production of the otoferlin protein crucial for auditory function, the gene therapy yielded promising results.

Key outcomes from the trial include significant improvements in speech perception, sound localization, and even an appreciation for music in some participants. Despite initial concerns regarding adverse effects and immune responses, the trial demonstrated a favorable safety profile, with no serious events reported.