VG901

VG901 is a first-in-class, clinical-stage gene therapy developed to treat retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene. It utilizes ViGeneron's proprietary next-generation adeno-associated virus vector (vgAAV) to deliver a functional CNGA1 gene directly to retinal photoreceptor cells via intravitreal injection, avoiding the need for subretinal administration and reducing the risk of retinal damage. The therapy aims to address the genetic root cause of CNGA1-associated RP by enabling functional gene replacement in affected cells. Preclinical studies have demonstrated safety, durable expression, and sustained tolerability. VG901 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA and is currently being evaluated in a Phase 1b clinical trial assessing its safety, tolerability, and preliminary efficacy as a one-time treatment.