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voretigene neparvovec-rzyl
Voretigene neparvovec-rzyl is a gene therapy that delivers a functional copy of the RPE65 gene into retinal cells to restore vision in patients with inherited retinal dystrophy caused by RPE65 mutations.
Voretigene neparvovec-rzyl is a gene therapy that delivers a functional copy of the RPE65 gene into retinal cells to restore vision in patients with inherited retinal dystrophy caused by RPE65 mutations. Used for Inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations (Leber congenital amaurosis type 2).
At a glance
| Generic name | voretigene neparvovec-rzyl |
|---|---|
| Also known as | AAV2-hRPE65v2 |
| Sponsor | Spark Therapeutics, Inc. |
| Drug class | Gene therapy |
| Target | RPE65 gene |
| Modality | Biologic |
| Therapeutic area | Ophthalmology |
| Phase | FDA-approved |
Mechanism of action
The drug uses an adeno-associated virus (AAV) vector to introduce a working RPE65 gene into the retinal pigment epithelium. RPE65 encodes a protein essential for the visual cycle; its absence or dysfunction causes progressive vision loss. By restoring RPE65 function, the therapy enables photoreceptors to regenerate visual pigment and respond to light, thereby halting or improving vision loss.
Approved indications
- Inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations (Leber congenital amaurosis type 2)
Common side effects
- Conjunctival hyperemia
- Eye pain
- Photopsia (flashing lights)
- Anterior chamber inflammation
- Cataract
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
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- voretigene neparvovec-rzyl CI brief — competitive landscape report
- voretigene neparvovec-rzyl updates RSS · CI watch RSS
- Spark Therapeutics, Inc. portfolio CI