First human ANGPTL3 gene editing study shows TG and cholesterol reduction

A one-time infusion of an investigational ANGPTL3-targeting CRISPR-Cas9 therapy, CTX310, reduced TG by approximately 55% and LDL-C by up to approximately 50%. These were the key findings from a Phase 1 first-in-human, single ascending dose study of CTX310 i.v. administered to 15 adults with uncontrolled hypercholesterolaemia, hypertriglyceridaemia or mixed dyslipidaemia while on maximally tolerated medical therapy.