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AAV2hAQP1
AAV2-mediated gene therapy targeting aquaporin-1
AAV2-mediated gene therapy targeting aquaporin-1 Used for Leber congenital amaurosis 10 (LCA10).
At a glance
| Generic name | AAV2hAQP1 |
|---|---|
| Sponsor | MeiraGTx, LLC |
| Drug class | Gene therapy |
| Target | Aquaporin-1 |
| Modality | Biologic |
| Therapeutic area | Ophthalmology |
| Phase | Phase 1 |
Mechanism of action
AAV2 vector delivers the hAQP1 gene to the retina to treat Leber congenital amaurosis 10 (LCA10)
Approved indications
- Leber congenital amaurosis 10 (LCA10)
Common side effects
Key clinical trials
- Safety of a Single Administration of AAV2hAQP1, an Adeno-Associated Viral Vector Encoding Human Aquaporin-1 to One Parotid Salivary Gland in People With Irradiation-Induced Parotid Salivary Hypofunction (PHASE1)
- Long-Term Follow-Up Study of AAV2hAQP1 for Radiation Induced Xerostomia
- A Phase 1 Open-Label, Dose Escalation Study to Determine the Optimal Dose, Safety, and Activity of AAV2hAQP1 in Subjects With Radiation-Induced Parotid Gland Hypofunction and Xerostomia (PHASE1)
- Effect of AdhAQP1 on Salivary Flow in Patients Treated With Radiation for Head and Neck Cancer (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:
- AAV2hAQP1 CI brief — competitive landscape report
- AAV2hAQP1 updates RSS · CI watch RSS
- MeiraGTx, LLC portfolio CI