VERVE-101

VERVE-101 is an investigational, in vivo CRISPR base-editing medicine designed as a single-course treatment to permanently inactivate the PCSK9 gene in the liver. By introducing a precise A-to-G DNA base change at a splice donor site of PCSK9, VERVE-101 disrupts production of hepatic PCSK9 protein, leading to durable reductions in low-density lipoprotein cholesterol (LDL-C). The therapy consists of messenger RNA encoding an adenine base editor and an optimized guide RNA targeting PCSK9, both delivered via engineered lipid nanoparticles. Developed by Verve Therapeutics, VERVE-101 is being evaluated primarily for heterozygous familial hypercholesterolemia (HeFH) and established atherosclerotic cardiovascular disease (ASCVD) with uncontrolled LDL-C levels. Early clinical data show dose-dependent and sustained reductions in both blood PCSK9 protein and LDL-C following a single infusion[1][2][3][4][5][6][7][8].

Other names

PCSK9 base editorPCSK-9 base editorPCSK 9 base editor