SKY-GJB2

SKY-GJB2 is an investigational gene therapy developed by Skylark Bio Inc. for the treatment of pediatric patients with severe-to-profound sensorineural hearing loss caused by mutations in the GJB2 gene (Connexin 26), a condition known as DFNB1A. The therapy works by delivering a functional copy of the GJB2 gene directly into the inner ear to restore the production of Connexin 26, a gap junction protein essential for the maintenance of potassium homeostasis and hearing function. It is administered as a single-dose intracochlear infusion using the proprietary SKY-CAT delivery device via a transcanal endoscopic tympanotomy approach. SKY-GJB2 is currently being evaluated in the Phase 1/2 SONIX clinical trial (NCT07627971) in pediatric subjects aged 9 months to 7 years.