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Phase I/II study of LY-M001 in adults with type 1 Gaucher disease
ChiCTR2400091415Phase 2Recruiting
- Sponsor
- Institute of Hematology and Blood Diseases Hospital
- Start
- Apr 2024
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label trial of LY-M001 in Gaucher disease type 1 (GD1) (4 arms), sponsored by Institute of Hematology and Blood Diseases Hospital. Primary endpoints: Phase I: Incidence of dose-limiting toxicity events, Phase I: Incidence of adverse events and serious adverse events and 3 more. Started Apr 2024; status: recruiting.
02
Drugs
LY-M001
03
Conditions
Gaucher disease type 1 (GD1)
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