Efficacy and Safety of an Investigational Single-course CRISPR Base Editing Therapy Targeting PCSK9 in Non-human Primate and Mouse Models.

VERVE-101 was well-tolerated in in non-human primates and led to 83% lower blood PCSK9 protein and 69% lower LDL-C with durable effects up to 476 days following dosing. These results have supported initiation of a first-in-human clinical trial in patients with heterozygous familial hypercholesterolemia and atherosclerotic cardiovascular disease.