Arrowhead Pharmaceuticals, Inc.
Methods For The Treatment Of APOC3-Related Diseases And Disorders

Abstract:

Described are methods for treating diseases and disorders that can be mediated in part by a reduction in APOC3 gene expression in a human subject in need of treatment, using pharmaceutical compositions that include APOC3 RNAi agents. The disclosed pharmaceutical compositions that include APOC3 RNAi agents, when administered to a human subject in need thereof according to the methods disclosed herein, treat diseases and disorders associated with elevated triglyceride (TG) levels, such as familial chylomicronemia syndrome (FCS), hypertriglyceridemia, obesity, dyslipidemia, non-alcoholic steatohepatitis, non-alcoholic fatty liver disease, hyperlipidemia, abnormal lipid and/or cholesterol metabolism, atherosclerosis, cardiovascular disease, coronary artery disease, hypertriglyceridemia induced pancreatitis, metabolic syndrome, type II diabetes mellitus, chylomicronemia, multifactorial chylomicronemia, or lipodystrophy syndromes including familial partial lipodystrophy.