Single-dose ION775, a GalNAc-conjugated siRNA targeting APOC3, lowers triglycerides for up to 12 months in moderate hypertriglyceridemia.
BACKGROUND Apolipoprotein C-III (apoC-III) regulates triglyceride-rich lipoprotein metabolism. ION775 is a GalNAc-conjugated small interfering RNA designed to silence APOC3 mRNA and enable infrequent dosing. OBJECTIVE To evaluate the safety, tolerability, and lipid effects of single-dose ION775 in adults with moderat...