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GC310 AAV gene therapy in adults with Wilson disease
ChiCTR2500113213Phase 2Not yet recruiting
- Sponsor
- Peking Union Medical College Hospital
- Start
- Nov 2025
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label trial of GC310 in Wilson disease (WD) (2 arms), sponsored by Peking Union Medical College Hospital. Primary endpoints: Incidence of adverse events within 12 weeks after GC310 administration and Incidence of dose-limiting toxicity (DLT) events within 4 weeks after GC310 administration. Planned start Nov 2025; status: not yet recruiting.
02
Drugs
GC310
03
Conditions
Wilson disease (WD)
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