VERVE-102 is an investigational gene-editing therapy designed to permanently turn off the PCSK9 gene in the liver to reduce LDL cholesterol.In a phase Ib study, a single infusion of the base-editing therapy reduced PCSK9 and LDL cholesterol levels in patients with heterozygous familial hypercholesterolemia or premature coronary artery disease.The lipid nanoparticle delivery system is engineered to reduce the risk of acute adverse events in light of prior issues with a predecessor therapy.
An investigational gene therapy passed muster for reducing PCSK9 and LDL cholesterol levels, with reassuring safety, among patients with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD), an interim analysis of a phase Ib trial suggested.
Depending on the dose of VERVE-102, mean reductions in PCSK9 levels ranged from 51% to 88%, while mean reductions in LDL cholesterol ranged from 9% to 62%, in the Heart-2 study, reported Sekar Kathiresan, MD, co-founder of Verve Therapeutics, a subsidiary of Eli Lilly, and colleagues.
LDL cholesterol fell by an absolute 78 mg/dL at the highest VERVE-102 dose of 1.0 mg/kg. The reduction looked durable, with the study reaching at least 1 year of follow-up for 15 participants.
