US regulator approves first gene treatment for childhood hearing loss

Scientists have started with a relatively simple condition, but say more is on the way.

The US has approved the first gene therapy for genetic sensorineural deafness, which could overcome childhood hearing loss in some children — no cochlear implant needed.

The condition known as DFNB9 accounts for roughly 3-8% of genetic hearing loss cases, while the treatment lunsotogene parvec-cwha (Otarmeni) targets harmful mutations in the OTOF gene, which encodes the protein otoferlin, a calcium sensor needed for signalling in inner ear cells.

It requires surgery similar to cochlear implantation.

An incision behind the ear provides access to the cochlea to apply, via a catheter and syringe pump, the dual adeno-associated virus serotype 1 vector, which carries a functional version of the OTOF gene that replaces the mutated version.