Skylark Bio doses first patient in hearing-loss trial
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- Skylark Bio emerged from stealth and announced it has dosed its first patient with a gene therapy designed to restore hearing in a child carrying a GJB2 mutation, the gene researchers call the 'holy grail' of hearing-loss targets.
- Regeneron received FDA approval in April for Otarmeni, the first gene therapy for deafness, which addresses Otoferlin-related mutations and was enabled by the 2023 acquisition of Decibel Therapeutics.
- Joe Burns, former SVP of research at Decibel Therapeutics, framed Otoferlin mutations as a 'Goldilocks' target because the ear remains completely intact in animal models even into geriatric age.
- Startups in the United States, France, and China are racing to develop GJB2-targeted therapies, positioning the mutation as the next frontier after Otoferlin.
- The earlier breakthrough two years ago allowed children born deaf to hear for the first time, turning indistinct murmurs into audible whispers and demonstrating robust gains without safety concerns.
Why it matters: GJB2 is the most common cause of genetic hearing loss, so a successful therapy would dramatically expand the addressable population beyond the narrow Otoferlin group served by Regeneron's newly approved Otarmeni. With startups in the US, France, and China all pursuing the same target, Skylark's first-in-human dosing marks the opening move in what is shaping up to be a multi-company race to treat the dominant genetic form of deafness.
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