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VSA003
- Development stage
- Unknown
- Lead developer
- Visirna Therapeutics
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
VSA003 is an innovative siRNA therapeutic developed by Visirna Biotechnology, a joint venture between Arrowhead Pharmaceuticals and Vivo Capital, specifically for the Greater China market. It is the China-specific version of Arrowhead's zodasiran (ARO-ANG3). VSA003 utilizes RNA interference (RNAi) to target and degrade the mRNA of angiopoietin-like protein 3 (ANGPTL3) in hepatocytes, facilitated by a GalNAc-conjugation delivery system. ANGPTL3 is a key regulator of lipid metabolism that inhibits lipoprotein lipase and endothelial lipase; thus, its reduction leads to decreased levels of low-density lipoprotein cholesterol (LDL-C) and triglycerides through both LDLR-dependent and LDLR-independent pathways. The drug is currently in Phase III development for homozygous familial hypercholesterolemia (HoFH) and Phase I for other dyslipidemias and metabolic conditions like NAFLD/NASH. It received Breakthrough Therapy Designation from China's Center for Drug Evaluation (CDE) in early 2024.
- Other names
- zodasiran
02
Targets
ANGPTL3 (Angiopoietin-like protein 3)
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