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LY-M001
- Development stage
- Phase 1
- Lead developer
- Lingyi Biotech
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
LY-M001 is an investigational gene therapy developed for the treatment of Gaucher disease type I and III. It is a recombinant adeno-associated virus serotype 8 (rAAV8) vector-based therapy that delivers a functional copy of the human GBA1 gene to liver cells via intravenous infusion. The expressed glucocerebrosidase enzyme aims to restore deficient enzyme activity in patients with Gaucher disease, leading to reduced accumulation of harmful glycolipid metabolites and sustained therapeutic benefit. Developed by Lingyi Biotech in China, LY-M001 has received IND approval from both the US FDA and China's NMPA and is currently being evaluated in early-phase clinical trials[1][2][4][7].
- Other names
- Recombinant adeno-associated virus vector serotype 8 carrying the coding sequence of human GBA1 gene
02
Targets
GBA1 (Glucosylceramidase)
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