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Synonyms: ARO-ANG3
Compound class:
Nucleic acid
Comment: Zodasiran (ARO-ANG3) is a RNA interference (RNAi, or siRNA) class novel therapeutic. The molecule includes bases that are modified to improve stability, and is congugated (at R1 position) with N-acetylgalactosamine (GalNAc), which binds to an asialoglycoprotein receptor expressed on hepatocyte membranes to promote delivery of the drug to the liver [1]. Once bound, the siRNA is internalised in lysosomes and is subsequently released into the cytosol. Zodasiran is designed to reduce synthesis of angiopoietin-like 3 (ANGPTL3), an hepatic peptide that regulates lipid and lipoprotein metabolism, including triglyceride-rich lipoproteins and cholesterol that are associated with cardiovascular disease risk.
We have been unable to locate a full chemical SMILES for the ligand, or its HELM notation. |
| References |
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1. Holm A, Løvendorf MB, Kauppinen S. (2021)
Development of siRNA Therapeutics for the Treatment of Liver Diseases. Methods Mol Biol, 2282: 57-75. [PMID:33928570] |
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2. Rosenson RS, Gaudet D, Hegele RA, Ballantyne CM, Nicholls SJ, Lucas KJ, San Martin J, Zhou R, Muhsin M, Chang T et al.. (2024)
Zodasiran, an RNAi Therapeutic Targeting ANGPTL3, for Mixed Hyperlipidemia. N Engl J Med, 391 (10): 913-925. [PMID:38809174] |
