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LY-M001 in Adult Gaucher Disease Type I
NCT06162338Phase 1Recruiting
- Start
- Nov 2023
- Primary completion
- Dec 2026
- Modality
- Gene Editing, Gene Silencing, AAV Vectors
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of LY-M001 in Gaucher disease type I (GD1). Primary endpoints: Incidence of treatment-related adverse events at 38 weeks and Incidence of events adjudicated as dose limiting toxicity (DLT) at 28 days. Started Nov 2023; status: recruiting.
02
Drugs
LY-M001
03
Conditions
Gaucher disease type I (GD1)
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