Skylar Bio dosed its first patient in a new gene therapy effort for childhood hearing loss linked to GJB2 mutations, building on the field’s momentum after FDA approval of Regeneron’s Otoferlin-related Otarmeni. The company said the new program targets restoration of hearing in a pediatric population with this genetic cause of deafness. The announcement arrives amid growing competition across the U.S., France, and China to address additional hearing-loss targets beyond the first wave of results tied to Otoferlin. Skylar Bio framed the start of dosing as a key operational milestone for a therapy aimed at restoring auditory function. Regulators approved Otarmeni for children and others with specific Otoferlin-related mutations earlier this year, and the article highlighted prior preclinical rationale around the ear remaining intact in animal models, supporting safety and feasibility for expansion to other genetic etiologies.
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