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ZS805
- Development stage
- Phase 2
- Lead developer
- Real & Best Biotech
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
ZS805 is a recombinant adeno-associated virus (rAAV)-based gene therapy developed for the treatment of Fabry disease. It utilizes a gene expression cassette equipped with a proprietary liver-specific promoter and an optimized human GLA (α-galactosidase A) gene, enabling targeted and efficient expression of α-galactosidase A in hepatocytes. This approach aims to provide long-term or potentially life-long therapeutic benefit from a single administration, addressing the underlying enzyme deficiency in Fabry disease. The drug is classified as an innovative biologic (class 1) and represents one of the first rAAV-based gene therapies for this indication in China[1][2][5].
02
Targets
GLA (Alpha-galactosidase A)
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