|
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. |
| No information available. |
Summary of Clinical Use  |
| A phase 2 study in participants with mixed dyslipidemia has been completed [2]. |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT04832971 | Study of ARO-ANG3 in Adults With Mixed Dyslipidemia | Phase 2 Interventional | Arrowhead Pharmaceuticals | In patients with mixed hyperlipidemia zodasiran treatment was associated with significant decreases in triglyceride levels at 24 weeks. Whether this translates to clinical benefits (reduction in cardiovascular disease risk) in the longer term remains to be determined. | 2 |
| NCT05217667 | Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH) | Phase 2 Interventional | Arrowhead Pharmaceuticals | ||
