Description

Verve Therapeutics, Inc., a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, announced the lifting of the clinical hold and clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) to conduct a clinical trial in the United States evaluating VERVE-101 for the treatment of heterozygous familial hypercholesterolemia (HeFH). The clearance enables clinical development of an in vivo base editing product candidate in the United States. VERVE-101 is designed to be a single-course treatment that permanently turns off the PCSK9 gene in the liver to reduce disease-driving low-density lipoprotein cholesterol (LDL-C). HeFH is a prevalent and potentially life-threatening inherited disease characterized by lifelong elevations in blood LDL-C and accelerated atherosclerotic cardiovascular disease (ASCVD). The heart-1 trial is evaluating the safety, tolerability, pharmacokinetic and pharmacodynamic profile of VERVE-101 in patients with HeFH, and is currently being conducted at sites in the United Kingdom and New Zealand. Interim clinical data from the heart-1 trial including safety parameters and changes in blood PCSK9 protein and blood LDL-C levels are expected to be presented at the American Heart Association's Scientific Sessions 2023