CTX310 gene editing therapy reduced LDL cholesterol by up to 48.9% and triglycerides by 55.2% after a single infusion in adults with refractory hypercholesterolemia or hypertriglyceridemia.
RCT (n=15)
Yes
Does a single intravenous infusion of the CRISPR-Cas9 therapy CTX310 reduce atherogenic lipoproteins in adults with refractory hypercholesterolemia or hypertriglyceridemia?
CTX310, an in vivo CRISPR-Cas9 therapy targeting ANGPTL3, provides proof-of-concept for a one-time, durable gene-editing strategy to substantially reduce atherogenic lipoproteins in patients with refractory dyslipidemia.
Effect estimate: 48.9% reduction in LDL cholesterol
Mondal et al. (Wed,) conducted a rct in Adults with refractory hypercholesterolemia or hypertriglyceridemia despite maximal therapy, including 40% with established atherosclerotic cardiovascular disease and 40% with familial hypercholesterolemia (n=15). CTX310 (in vivo CRISPR-Cas9 gene editing therapy targeting ANGPTL3) vs. None (Phase 1 single-arm dose-escalation trial) was evaluated on Reduction in atherogenic lipoproteins (LDL cholesterol, triglycerides, non-HDL lipoproteins) at 60 days post-infusion (48.9% reduction in LDL cholesterol). CTX310 gene editing therapy reduced LDL cholesterol by up to 48.9% and triglycerides by 55.2% after a single infusion in adults with refractory hypercholesterolemia or hypertriglyceridemia.