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LY-M003 gene therapy single-arm study in Wilson's disease
ChiCTR2500096888Phase 2Recruiting
- Sponsor
- First Affiliated Hospital, Zhejiang University School of Medicine
- Start
- Sep 2024
- 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) (5 cohorts), sponsored by First Affiliated Hospital, Zhejiang University School of Medicine. Primary endpoints: Incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks following LY-M003 infusion and Incidence of dose-limiting toxicity (DLT) events within at least 28 days following LY-M003 infusion. Started Sep 2024; status: recruiting.
02
Drugs
LY-M003
03
Conditions
Wilson disease (WD)
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