AAV-AIPL1

AAV-AIPL1 is an investigational gene therapy developed for the treatment of Leber congenital amaurosis 4 (LCA4), a severe inherited retinal dystrophy caused by mutations in the aryl hydrocarbon receptor-interacting protein-like 1 (AIPL1) gene. The therapy uses an adeno-associated viral (AAV) vector to deliver a functional copy of the AIPL1 gene directly to cone and rod photoreceptors in the retina via subretinal injection. This approach aims to restore vision and slow further degeneration in children with LCA4, who are typically blind from birth. Clinical data show that treated children experienced significant improvements in visual acuity and retinal structure compared to untreated eyes. The drug has received orphan drug designation from both FDA and European Commission, as well as rare pediatric disease designation from FDA[1][3][5][6].