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LY-M003
- Development stage
- Unknown
- Lead developer
- Lingyi Biotech
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
LY-M003 is an investigational adeno-associated virus (AAV)-based gene therapy developed by Lingyi Biotech for the treatment of Wilson's disease (hepatolenticular degeneration). The therapy uses a recombinant AAV vector to deliver a functional copy of the ATP7B gene into liver cells via intravenous infusion. This enables long-term expression of ATP7B protein in hepatocytes, aiming to restore copper metabolism, reduce copper accumulation in organs, and improve systemic symptoms associated with Wilson's disease. The drug has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA. Clinical trials are ongoing to evaluate its safety, tolerability, and efficacy[1][2][3][4].
- Other names
- LY-M003LY-M-003LY-M 003LY M003
02
Targets
ATP7B (Copper ion transporter ATPase 7B)
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