Drug intelligence / Profile preview

apolipoprotein CIII antisense oligonucleotide

Development stage
Unknown
Lead developer
Ionis Pharmaceuticals
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous
01

Overview

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).

Other names
APOC3 ASOAPOC-3 ASOAPOC 3 ASOAPOC3 antisense oligonucleotideAPOC-3 antisense oligonucleotideAPOC 3 antisense oligonucleotideapoC-III ASOapoC-III antisense oligonucleotideapolipoprotein C-III antisense oligonucleotide
02

Targets

APOC3 (Apolipoprotein C-III)

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