STAT+: Skylark Bio kicks off new gene therapy trial for deafness
Get the Health newsletter
Daily health & science — research, biotech, public health, the studies worth knowing. Free.
- Skylark Bio emerged from stealth on Tuesday and dosed its first patient with a gene therapy aimed at restoring hearing in a child with a GJB2 mutation — the "holy grail" hearing-loss target.
- The FDA approved Regeneron's Otarmeni gene therapy in April for people with otoferlin-related mutations, two years after a landmark trial restored hearing in children who had been born deaf.
- Decibel Therapeutics was acquired by Regeneron in 2023; former SVP of research Joe Burns called otoferlin-related mutations a "Goldilocks" scenario because the ear stays completely intact in animal models — even geriatric ones.
- Startups in the US, France, and China are now racing to develop GJB2-targeted therapies, signaling a broader push beyond Regeneron's approved otoferlin indication.
Why it matters: Skylark's first-in-human dosing is a concrete step toward the GJB2 gene — the "holy grail" hearing-loss target that startups across three countries are now racing to crack. The move comes just months after the FDA approved Regeneron's Otarmeni for the narrower otoferlin-related patient group, establishing a regulatory precedent that could accelerate follow-on gene therapies.
Ask SkimNews
