Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy

Russell et al. (2017). Lancet DOI: 10.1016/S0140-6736(17)31868-8 Citations: 1968

Parts used

AAV2 Serotype

Key findings

  • Pivotal Phase 3 trial leading to Luxturna approval. First FDA-approved gene therapy using AAV2 for inherited retinal disease. Established AAV2 as gold standard for retinal applications.