VAR002

VAR002 is an investigational **AAV-based gene therapy** developed by Variant for inherited retinal dystrophies caused by **CRX gene mutations**, including Leber congenital amaurosis, cone-rod dystrophy, and retinitis pigmentosa. It is a recombinant adeno-associated viral vector engineered to deliver an unmutated human **CRX** gene to retinal photoreceptors as a **gene supplementation** approach intended to restore functional CRX protein expression. The product is described as a mutation-agnostic therapy for CRX-associated disease and is administered as a sterile viral particle suspension by **subretinal injection**.