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AAV2-hRPE65v2,voretigene neparvovec-rzyl
AAV2-hRPE65v2,voretigene neparvovec-rzyl is a Gene therapy Biologic drug developed by Spark Therapeutics, Inc.. It is currently in Phase 3 development for Inherited retinal dystrophy caused by biallelic RPE65 mutations (Leber congenital amaurosis type 2). Also known as: AAV2-hRPE65v2, voretigene neparvovec, gene therapy vector, voretigene neparvovec-rzyl.
AAV2-hRPE65v2 is a gene therapy that delivers a functional copy of the human RPE65 gene into retinal pigment epithelium cells to restore vision in patients with inherited retinal dystrophy caused by RPE65 mutations.
AAV2-hRPE65v2 is a gene therapy that delivers a functional copy of the human RPE65 gene into retinal pigment epithelium cells to restore vision in patients with inherited retinal dystrophy caused by RPE65 mutations. Used for Inherited retinal dystrophy caused by biallelic RPE65 mutations (Leber congenital amaurosis type 2).
At a glance
| Generic name | AAV2-hRPE65v2,voretigene neparvovec-rzyl |
|---|---|
| Also known as | AAV2-hRPE65v2, voretigene neparvovec, gene therapy vector, voretigene neparvovec-rzyl |
| Sponsor | Spark Therapeutics, Inc. |
| Drug class | Gene therapy |
| Target | RPE65 gene |
| Modality | Biologic |
| Therapeutic area | Ophthalmology |
| Phase | Phase 3 |
Mechanism of action
The drug uses an adeno-associated virus serotype 2 (AAV2) vector to deliver the normal RPE65 gene into the eye. RPE65 encodes a protein essential for the visual cycle, which converts vitamin A into the light-sensitive form needed for photoreceptor function. By restoring RPE65 protein production, the therapy enables photoreceptors to respond to light again, thereby restoring or improving vision in patients with RPE65-mediated inherited retinal disease.
Approved indications
- Inherited retinal dystrophy caused by biallelic RPE65 mutations (Leber congenital amaurosis type 2)
Common side effects
- Conjunctival hyperemia
- Eye pain or discomfort
- Photopsia (flashing lights)
- Anterior chamber inflammation
- Cataract progression
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:
- AAV2-hRPE65v2,voretigene neparvovec-rzyl CI brief — competitive landscape report
- AAV2-hRPE65v2,voretigene neparvovec-rzyl updates RSS · CI watch RSS
- Spark Therapeutics, Inc. portfolio CI
Frequently asked questions about AAV2-hRPE65v2,voretigene neparvovec-rzyl
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Related
- Drug class: All Gene therapy drugs
- Target: All drugs targeting RPE65 gene
- Manufacturer: Spark Therapeutics, Inc. — full pipeline
- Therapeutic area: All drugs in Ophthalmology
- Indication: Drugs for Inherited retinal dystrophy caused by biallelic RPE65 mutations (Leber congenital amaurosis type 2)
- Also known as: AAV2-hRPE65v2, voretigene neparvovec, gene therapy vector, voretigene neparvovec-rzyl
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