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AAV2-hCHM
Gene therapy for Leber congenital amaurosis
Gene therapy for Leber congenital amaurosis Used for Leber congenital amaurosis.
At a glance
| Generic name | AAV2-hCHM |
|---|---|
| Sponsor | Spark Therapeutics, Inc. |
| Drug class | Gene therapy |
| Target | RPE65 |
| Modality | Biologic |
| Therapeutic area | Ophthalmology |
| Phase | Phase 1 |
Mechanism of action
AAV2-hCHM is a recombinant adeno-associated virus vector that carries a normal copy of the RPE65 gene to replace the faulty gene in patients with Leber congenital amaurosis.
Approved indications
- Leber congenital amaurosis
Common side effects
- Vision impairment
- Eye inflammation
Key clinical trials
- Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations (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:
- AAV2-hCHM CI brief — competitive landscape report
- AAV2-hCHM updates RSS · CI watch RSS
- Spark Therapeutics, Inc. portfolio CI