Shanghai startup’s gene therapy restores hearing in five deaf children, running ahead of Lilly and Regeneron trials

A Shanghai startup has developed an experimental gene therapy that has successfully restored hearing in five out of six deaf children with congenital deafness. This groundbreaking achievement puts the Chinese company ahead of trials being conducted by Eli Lilly and Regeneron.

The gene therapy treatment involves injecting a functional copy of a gene called otoferlin into the inner ear. Mutations in the otoferlin gene are responsible for hearing loss due to defective hair cell function. Regeneron has also been working on a gene therapy for hearing loss, with preliminary results showing improvement in a child under two years old who received the treatment in one ear. However, the Shanghai startup's gene therapy has demonstrated significant progress, restoring hearing in five deaf children and outpacing the competition.