Skylark Bio Advances Pendrin Gene Therapy for Hearing Loss with New Preclinical Data
Skylark Bio, a biotechnology company, is set to present new preclinical data for its Pendrin gene therapy program, SKY-PEN, at the American Society of Gene & Cell Therapy (ASGCT) 2026 Annual Meeting. The program targets SLC26A4-related hearing loss, a significant genetic cause of hearing impairment. The preclinical data demonstrate dose-dependent improvements in hearing across multiple mouse models, with preservation of critical inner-ear structures and precise expression in cochlear cells. Skylark Bio's approach involves a proprietary capsid for targeted, micro-dose delivery, aiming to provide durable benefits in non-dividing cells. The company is also advancing its GJB2 program for pediatric deafness, with first-in-human studies planned for mid-2026.