APOC3 antisense oligonucleotide

APOC3 antisense oligonucleotides (ASOs) are a class of RNA-targeted therapeutics designed to inhibit the production of apolipoprotein C-III (ApoC-III), a protein that plays a central role in triglyceride metabolism. ApoC-III inhibits lipoprotein lipase (LPL) and hepatic lipase, and interferes with the hepatic uptake of triglyceride-rich lipoproteins (TRLs), thereby increasing plasma triglyceride levels. By binding to APOC3 mRNA and inducing its degradation (typically via RNase H), these ASOs reduce ApoC-III synthesis, leading to significant reductions in triglycerides and TRLs. This class includes assets such as volanesorsen, which is approved for familial chylomicronemia syndrome (FCS), and olezarsen, a GalNAc-conjugated ASO in late-stage development for severe hypertriglyceridemia. Recent research also suggests potential benefits in preserving pancreatic beta-cell function and improving glycemic control in type 2 diabetes.

Other names

APOC3 ASOAPOC-3 ASOAPOC 3 ASOApoC-III antisense oligonucleotide