In Vivo CRISPR Base Editing of <i>ANGPTL3</i> for Durable Lipid Lowering

What happened: A Nature paper describes a single-course infusion of guide RNA and mRNA encoding a CRISPR adenine base editor, delivered in a GalNAc lipid nanoparticle, that inactivates ANGPTL3 in the liver. Key numbers: More than 90% mean reduction in circulating ANGPTL3 protein in nonhuman primates, durable to 3 years after one dose. Why it matters: The results provided the scientific foundation for an ongoing first-in-human trial in patients with refractory hyperlipidemia syndromes, pointing to a possible single-course alternative to chronic lipid-lowering treatment.

A Nature paper describes a single-course infusion of guide RNA and mRNA encoding a CRISPR adenine base editor, delivered in a GalNAc lipid nanoparticle, that inactivates ANGPTL3 in the liver.

The results provided the scientific foundation for an ongoing first-in-human trial in patients with refractory hyperlipidemia syndromes, pointing to a possible single-course alternative to chronic lipid-lowering treatment.