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AAV RPE65
Gene therapy for RPE65-mediated inherited retinal dystrophy
Gene therapy for RPE65-mediated inherited retinal dystrophy Used for Leber congenital amaurosis, Inherited retinal dystrophy.
At a glance
| Generic name | AAV RPE65 |
|---|---|
| Sponsor | MeiraGTx UK II Ltd |
| Drug class | Gene therapy |
| Target | RPE65 |
| Modality | Biologic |
| Therapeutic area | Ophthalmology |
| Phase | Phase 1 |
Mechanism of action
AAV RPE65 is a gene therapy that uses an adeno-associated virus (AAV) to deliver a healthy copy of the RPE65 gene to the retina, replacing the faulty gene responsible for inherited retinal dystrophy.
Approved indications
- Leber congenital amaurosis
- Inherited retinal dystrophy
Common side effects
- Vision impairment
- Intraocular pressure increase
Key clinical trials
- Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (PHASE3)
- Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)
- Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) (PHASE1, PHASE2)
- Safety Study in Subjects With Leber Congenital Amaurosis (PHASE1)
- Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis (PHASE1, PHASE2)
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:
- AAV RPE65 CI brief — competitive landscape report
- AAV RPE65 updates RSS · CI watch RSS
- MeiraGTx UK II Ltd portfolio CI