CRISPR lipid treatment shows three-year durability in primates

A single dose of a CRISPR treatment reduced circulating ANGPTL3 protein by a mean of more than 90% in nonhuman primates, with durability confirmed through 3 years, researchers reported in Nature. The findings supported initiation of an ongoing first-in-human trial in patients with refractory hyperlipidemia syndromes. The treatment delivers RNA inside nanoparticles to liver cells, where a base editor changes DNA to inactivate ANGPTL3, a driver of elevated cholesterol and triglycerides. In mouse and nonhuman primate models of hyperlipidemia, inactivation was associated with reductions of approximately 40 to 50% in LDL cholesterol and 50 to 70% in triglycerides. Those lipid reductions are animal results; the study reports no human treatment outcomes.