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Drug intelligence / Profile preview
Apolipoprotein CIII (apoC-III) antisense oligonucleotides are a class of RNA-targeted therapeutics designed to inhibit the synthesis of apolipoprotein C-III, a key regulator of triglyceride metabolism. ApoC-III is primarily produced in the liver and acts as an inhibitor of lipoprotein lipase (LPL) and hepatic uptake of triglyceride-rich lipoproteins. By binding to the complementary sequence of APOC3 mRNA, these antisense oligonucleotides trigger RNase H-mediated degradation of the mRNA, thereby reducing apoC-III protein levels and facilitating the clearance of plasma triglycerides. Key clinical-stage and approved assets in this class include volanesorsen (Waylivra) and the next-generation GalNAc-conjugated olezarsen (Tryngolza), which are developed or approved for the treatment of severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS).
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