Skylark Bio

Skylark Bio is a clinical-stage gene therapy company focused on developing targeted genetic medicines to address the underlying causes of hereditary hearing loss. The company's lead program, SKY-GJB2, targets GJB2-related pediatric deafness — the most common genetic cause of childhood hearing loss worldwide — using an AAV vector engineered for precise, cell-specific delivery to the inner ear. In 2026, Skylark received FDA IND clearance for SKY-GJB2, enabling initiation of its first-in-human Phase 1/2 SONIX clinical trial. The company is also advancing a second program, SKY-PEN, targeting Pendred Syndrome (SLC26A4 mutations), for which preclinical data demonstrated dose-dependent hearing rescue across multiple mouse models. Skylark's gene therapy approach leverages targeted local delivery to cochlear supporting cells, avoiding systemic exposure. The company has raised $40 million in funding and partnered with Forge Biologics for AAV development and cGMP manufacturing to support clinical advancement.