STAT+: Skylark Bio kicks off new gene therapy trial for deafness

Two years ago, scientists achieved a breakthrough by using gene therapy to restore hearing in children born deaf due to otoferlin mutations, with some hearing for the first time. The robust gains and lack of safety concerns led the FDA to approve Regeneron's Otarmeni therapy in April for people with this rare genetic mutation. According to Joe Burns, former senior vice president of research at Decibel Therapeutics (acquired by Regeneron in 2023), otoferlin-related mutations are a 'Goldilocks' case because the ear remains intact in animal models even into geriatric models.

Researchers are now targeting other causes of deafness, including the GJB2 gene, considered the holy grail. Startups across the US, France, and China are racing to develop solutions. Skylark Bio emerged from stealth on Tuesday to announce it has dosed its first patient with a therapy aimed at restoring hearing in a child with a GJB2 mutation.