APOC3 dyslipidemia program

The APOC3 dyslipidemia program, developed by Arrakis Therapeutics, is a preclinical drug candidate utilizing a proprietary RNA-targeted small molecule (rSM) platform. The candidate is designed to bind directly to the mRNA encoding apolipoprotein C-III (APOC3), a protein that plays a critical role in lipid metabolism by inhibiting lipoprotein lipase and the hepatic uptake of triglyceride-rich lipoproteins. By targeting the APOC3 RNA structure, the small molecule aims to inhibit protein translation, thereby reducing APOC3 levels and promoting the clearance of triglycerides. This approach is intended for the treatment of dyslipidemia and the reduction of atherosclerotic cardiovascular disease risk. Unlike other APOC3-targeted therapies such as antisense oligonucleotides or siRNAs, this program focuses on an orally bioavailable small-molecule modality.

Other names

apolipoprotein C3APOC3 RNA-targeted small moleculeAPOC-3 RNA-targeted small moleculeAPOC 3 RNA-targeted small moleculeAPOC3 rSMAPOC-3 rSMAPOC 3 rSM