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zodasiran
- Development stage
- Phase 3
- Lead developer
- Arrowhead Pharmaceuticals
- 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
Zodasiran (formerly ARO-ANG3) is an investigational RNA interference (RNAi) therapeutic developed by Arrowhead Pharmaceuticals for the treatment of lipid disorders, specifically homozygous familial hypercholesterolemia (HoFH). It is designed to reduce the production of angiopoietin-like protein 3 (ANGPTL3), a hepatocyte-expressed regulator of lipid and lipoprotein metabolism. By utilizing the company's Targeted RNAi Molecule (TRiM) platform, zodasiran silences the ANGPTL3 gene, leading to decreased levels of triglycerides, low-density lipoprotein cholesterol (LDL-C), and other atherogenic lipoproteins. The drug is administered via subcutaneous injection and is currently being evaluated in Phase 3 clinical trials, including the YOSEMITE and SPRUCE studies, for patients with HoFH who require additional lipid-lowering therapy.
- Other names
- zodasiranVSA003VSA-003VSA 003ARO-ANG3ARO-ANG-3ARO-ANG 3ARO-ANG33ARO-ANG-33ARO-ANG 33
02
Targets
ANGPTL3 (Angiopoietin-like protein 3)
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