Skylark Bio Begins First Human Trial of Gene Therapy for GJB2-Related Deafness

Skylark Bio, a newly unveiled biotech firm, disclosed that it has dosed its first patient with a gene-therapy designed to correct a GJB2 mutation that causes congenital deafness. The trial follows the FDA’s April clearance of Regeneron’s Otarmeni, a therapy for a distinct otoferlin-related mutation that demonstrated robust hearing gains without safety concerns. Experts note that otoferlin mutations act like a “Goldilocks” scenario, preserving ear anatomy even in aged animal models, a point emphasized by former Decibel Therapeutics research leader Joe Burns.

The GJB2 gene is widely regarded as a coveted target for broader hearing-loss treatments, prompting a race among startups in the United States, France and China. Skylark’s entry signals the next phase of translating genetic breakthroughs into clinical options for children born deaf. The company’s move underscores growing momentum in the field of auditory gene therapy, building on prior successes and expanding the pipeline of potential cures.