US regulator approves first gene treatment for childhood hearing loss
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.