Last reviewed 2026-04-23 · How we verify

AAV2-hRPE65v2

AAV2-hRPE65v2 is a gene therapy that uses a viral vector to deliver a healthy copy of the RPE65 gene to the retina, treating inherited retinal dystrophy.

AAV2-hRPE65v2 is a gene therapy that uses a viral vector to deliver a healthy copy of the RPE65 gene to the retina, treating inherited retinal dystrophy. Used for Leber congenital amaurosis type 10, inherited retinal dystrophy.

At a glance

Mechanism of action

This gene therapy works by using an adeno-associated virus (AAV) to deliver a functional copy of the RPE65 gene to the retinal pigment epithelium (RPE) cells in the retina. The RPE65 gene is responsible for producing an enzyme that helps convert light into electrical signals in the retina. By delivering a healthy copy of this gene, AAV2-hRPE65v2 aims to restore vision in individuals with inherited retinal dystrophy caused by RPE65 gene mutations.

Approved indications

• Leber congenital amaurosis type 10, inherited retinal dystrophy

Common side effects

• Vision improvement

Key clinical trials

• A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US
• Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 (PHASE1, PHASE2)
• Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (PHASE3)
• Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
• Safety Study in Subjects With Leber Congenital Amaurosis (PHASE1)

Primary sources

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Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• AAV2-hRPE65v2 CI brief — competitive landscape report
• AAV2-hRPE65v2 updates RSS · CI watch RSS
• Spark Therapeutics, Inc. portfolio CI