ALN-ANGsc

ALN-ANGsc is an investigational, subcutaneously administered small interfering RNA (siRNA) therapeutic designed to inhibit the synthesis of Angiopoietin-like 3 (ANGPTL3) in the liver. Developed by Alnylam Pharmaceuticals in collaboration with Regeneron Pharmaceuticals, the drug utilizes Alnylam's GalNAc-conjugate delivery platform (Enhanced Stabilization Chemistry) to specifically target hepatocytes. By silencing the ANGPTL3 gene, ALN-ANGsc aims to lower circulating levels of triglycerides and LDL cholesterol, addressing unmet needs in patients with mixed hyperlipidemia and other forms of dyslipidemia. Preclinical studies in rodent and non-human primate models have demonstrated significant and durable reductions in ANGPTL3 protein and lipid levels following a single dose, supporting its potential for infrequent dosing in clinical settings.