A single infusion of CTX310, a CRISPR-Cas9 gene-editing therapy targeting ANGPTL3, lowered LDL cholesterol by 49% and triglycerides by 55% at 60 days in patients with treatment-resistant dyslipidemia, according to a phase 1 trial in the New England Journal of Medicine. Among 15 participants, ANGPTL3 levels fell 73% at 30 days with the highest 0.8 mg/kg dose. No serious treatment-related adverse events occurred; mild infusion reactions were reported in 20% of patients. Responses varied widely (LDL reduction 19.5–86.6%). The therapy, developed by CRISPR Therapeutics, may offer durable lipid lowering pending long-term safety and efficacy follow-up.