CRISPR gene editing of angiopoietin-like 3: toward one-time precision therapy for dyslipidaemia

In a landmark study published in The New England Journal of Medicine , Laffin et al. 1 present the first-in-human clinical evaluation of CTX310, an investigational in vivo CRISPR–Cas9 therapy engineered to induce permanent loss-of-function mutations in the hepatic ANGPTL3 gene, resulting in sustained lowering of atherogenic lipoproteins. This marks a major step toward durable, potentially lifelong lipid lowering through a single therapeutic intervention, 1 potentially replacing years of ongoing lipid-lowering treatment for atherosclerotic cardiovascular disease (ASCVD) patients.