Skylark Bio has dosed the first patient in a phase 1/2 clinical trial evaluating SKY-GJB2, the company’s investigational inner-ear-targeted gene therapy for GJB2-mediated hearing loss, the most common genetic cause of pediatric deafness.
There are currently no approved disease‑modifying treatments for GJB2-mediated hearing loss. Current treatment options, including hearing aids and cochlear implants, can provide partial access to sound but do not restore natural hearing. SKY-GJB2 is designed as a potential first-in-class therapy to address the underlying cause of GJB2-related hearing loss by precisely delivering a functional copy of GJB2 to supporting cells in the cochlea that naturally express connexin 26, with the goal of restoring the gap-junction network essential for hearing.
The SONIX Phase 1/2 clinical trial is designed to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of a single unilateral intracochlear injection of SKY-GJB2 in children aged 9 months to 7 years. Preliminary data will be available at year-end 2026 with additional readouts anticipated in 2027.
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