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LY-M003 gene therapy in adult Wilson's disease (Phase I/II, single-dose)
ChiCTR2600126269Phase 2Not yet recruiting
- Sponsor
- First Affiliated Hospital, Zhejiang University School of Medicine
- Start
- Jun 2026
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label trial of LY-M003 in Wilson disease (WD) (3 arms), sponsored by First Affiliated Hospital, Zhejiang University School of Medicine. Primary endpoints: Incidence of dose-limiting toxicity (DLT) within at least 28 days after LY-M003 infusion (Phase I) and Safety profile within 52 weeks after LY-M003 infusion (AEs, AESIs, SAEs, and clinical/lab abnormalities). Planned start Jun 2026; status: not yet recruiting.
02
Drugs
LY-M003
03
Conditions
Wilson disease (WD)
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