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AAV2-hCHM — Competitive Intelligence Brief
phase 1
Gene therapy
RPE65
Ophthalmology
Biologic
Live · refreshed every 30 min
Target snapshot
AAV2-hCHM (AAV2-hCHM) — Spark Therapeutics, Inc.. Gene therapy for Leber congenital amaurosis
Comparator set (6 drugs)
| Drug | Generic | Sponsor | Phase | Class | Target | Approval / PDUFA |
|---|---|---|---|---|---|---|
| AAV2-hCHM TARGET | AAV2-hCHM | Spark Therapeutics, Inc. | phase 1 | Gene therapy | RPE65 | |
| voretigene neparvovec-rzyl | voretigene neparvovec-rzyl | Spark Therapeutics, Inc. | marketed | Gene therapy | RPE65 gene | |
| AAV2-hRPE65v2,voretigene neparvovec-rzyl | AAV2-hRPE65v2,voretigene neparvovec-rzyl | Spark Therapeutics, Inc. | phase 3 | Gene therapy | RPE65 gene | |
| Onasemnogene Abeparvovec-brve | Onasemnogene Abeparvovec-brve | Novartis Pharmaceuticals | marketed | Gene Therapy | SMN1 gene | |
| p53 with chemotherapy | p53 with chemotherapy | Shenzhen SiBiono GeneTech Co.,Ltd | marketed | Gene therapy; recombinant adenovirus vector | p53 (tumor suppressor protein) | |
| p53 gene therapy | p53 gene therapy | Shenzhen SiBiono GeneTech Co.,Ltd | marketed | Gene therapy | p53 (tumor suppressor protein) | |
| chemotherapy plus p53 | chemotherapy plus p53 | Shenzhen SiBiono GeneTech Co.,Ltd | marketed | Combination chemotherapy with p53 gene therapy | p53 tumor suppressor protein |
Recent regulatory actions (last 90 days)
Upcoming PDUFA dates (next 180 days)
Patent timeline (forward window)
Sponsor landscape (Gene therapy class)
- Spark Therapeutics, Inc. · 4 drugs in this class
- MeiraGTx UK II Ltd · 3 drugs in this class
- Novartis Gene Therapies · 2 drugs in this class
- Kolon TissueGene, Inc. · 2 drugs in this class
- Krystal Biotech, Inc. · 2 drugs in this class
- Abeona Therapeutics, Inc · 2 drugs in this class
- Larimar Therapeutics, Inc. · 1 drug in this class
- 4D Molecular Therapeutics · 1 drug in this class
- Medeor Therapeutics, Inc. · 1 drug in this class
- MeiraGTx, LLC · 1 drug in this class
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Cite this brief
Drug Landscape (2026). AAV2-hCHM — Competitive Intelligence Brief. https://druglandscape.com/ci/aav2-hchm. Accessed 2026-05-13.
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