Volanesorsen (ISIS 304801) is a chemically stabilized antisense oligonucleotide that selectively inhibits apolipoprotein CIII messenger RNA, resulting in reduced triglyceride levels and improved insulin sensitivity, Volanesorsen is actively studied as a targeted nucleic acid therapeutic strategy for hypertriglyceridemia, familial chylomicronemia syndrome, and type 2 diabetes.

In human apo-CIII transgenic mice, Volanesorsen treatment (1.5-50 mg/kg, i.p., weekly) for 2 weeks demonstrated a dose-dependent reduction in hepatic human apo-CIII mRNA, plasma apo-CIII protein, and plasma triglycerides (TG). In primate models, efficacy was also observed. specifically, in Cynomolgus monkeys, subcutaneous administration (4-40 mg/kg, weekly) for 13 weeks reduced liver apoC-III mRNA levels in a dose-responsive manner. Furthermore, in hypertriglyceridemic Rhesus monkeys, treatment with 10 mg/kg (s.c., weekly) for 10 weeks lowered plasma triglyceride levels [1][2]

H2O: 40 mg/mL (5.58 mM), Sonication is recommended.