abstract |
The present disclosure provides RNAi agents capable of inhibiting apoprotein C-III (also called APOC3, apoC-III, APOC-III, and APO C-III) gene expression, such as double-stranded RNAi agents, and It relates to a composition comprising an APOC3 RNAi agent. APOC3 RNAi agents disclosed herein can be conjugated to targeting ligands, including ligands comprising N-acetyl-galactosamine, to facilitate delivery to cells, including hepatocytes. Pharmaceutical compositions comprising one or more APOC3 RNAi agents, optionally with one or more additional therapeutic agents, are also described. In vivo delivery of the APOC3 RNAi agent provides inhibition of APOC3 gene expression, and may result in lower triglyceride and / or cholesterol levels in the subject. APOC3 RNAi agents can be used in the treatment of APOC3-related diseases and disorders, including hypertriglyceridemia, cardiovascular disease, and other metabolic-related disorders and diseases. |