Single Gene-Editing Infusion Cuts Bad Cholesterol for a Year, Early Trial Shows

A small Phase 1 trial found that a single infusion of CTX310, a CRISPR-Cas9 therapy targeting ANGPTL3 in the liver, reduced LDL cholesterol by 52.5% and triglycerides by 47.8% at 12 months across doses, with no serious safety events; durability suggests potential year-long lipid control, though larger studies and 15-year safety follow-up are planned.