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apoCIII-siRNA
- Development stage
- Unknown
- Lead developer
- Alnylam Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous, Subcutaneous
01
Overview
apoCIII-siRNA (apolipoprotein C-III small interfering RNA) refers to a class of gene-silencing therapeutics designed to target and degrade the messenger RNA (mRNA) encoding apolipoprotein C-III (APOC3). APOC3 is a key glycoprotein synthesized primarily in the liver that plays a critical role in lipid metabolism by inhibiting lipoprotein lipase (LPL) and delaying the clearance of triglyceride-rich lipoproteins (TRLs). By utilizing the RNA interference (RNAi) pathway, apoCIII-siRNA molecules selectively reduce hepatic APOC3 expression, thereby restoring LPL activity and promoting the clearance of triglycerides, VLDL, and chylomicrons. This therapeutic approach is being investigated for the treatment of severe hypertriglyceridemia, mixed dyslipidemia, familial chylomicronemia syndrome (FCS), metabolic syndrome, and type 1 diabetes. Clinical-stage candidates in this class include plozasiran (ARO-APOC3) and ALN-APOC3, while preclinical research has also been conducted by institutions such as the Karolinska Institutet.
- Other names
- APOC3 siRNAAPOC-3 siRNAAPOC 3 siRNAapolipoprotein C-III siRNAapolipoprotein CIII siRNA
02
Targets
AGO2/RISC (Protein Argonaute-2 (AGO2) and RNA-induced silencing complex (RISC))LPL (Lipoprotein lipase)APOC3 (Apolipoprotein C-III)
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