VERVE-201

VERVE-201 is a novel, investigational in vivo base editing gene therapy designed to permanently inactivate the ANGPTL3 gene in the liver. By turning off this gene, VERVE-201 aims to reduce disease-driving low-density lipoprotein cholesterol (LDL-C) and remnant cholesterol levels. The drug utilizes Verve Therapeutics' proprietary GalNAc-LNP delivery technology for targeted delivery to liver cells. It is being developed as a single-course treatment for patients with refractory hypercholesterolemia—those who cannot achieve adequate LDL-C reduction despite maximally tolerated standard of care therapies, including PCSK9 inhibitors—and for individuals with homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder characterized by extremely high LDL-C and premature atherosclerotic cardiovascular disease. The goal of VERVE-201 is to provide durable LDL-C lowering and reduce the lifelong treatment burden associated with current therapies[1][2][5][6][7].

Other names

ANGPTL3 targeted gene editing therapyANGPTL-3 targeted gene editing therapyANGPTL 3 targeted gene editing therapy