VERVE-102

VERVE-102 is a novel, investigational in vivo base editing medicine developed as a single-course gene editing therapy to permanently turn off the PCSK9 gene in the liver. By using an adenine base editor and a guide RNA (gRNA) targeting PCSK9, both encapsulated in a lipid nanoparticle (LNP) and administered via intravenous infusion, it aims to durably reduce disease-driving LDL cholesterol (LDL-C). The therapy uses Verve Therapeutics' proprietary GalNAc-LNP delivery technology for targeted delivery to liver cells. Its primary indication is for patients with heterozygous familial hypercholesterolemia (HeFH), premature coronary artery disease (CAD), or hyperlipidemia who require additional LDL-C lowering. The mechanism of action involves permanent disruption of the PCSK9 gene, leading to reduced production of PCSK9 protein and increased LDL receptor activity, thereby lowering blood LDL-C levels[1][2][4][5][6].

Other names

base editing therapy targeting cardiovascular diseasein vivo base-editing cardiovascular medicine