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Verve Begins Dosing Patients With Base Editing Cardiovascular Drug in Heart-2 Trial

NEW YORK – Verve Therapeutics has treated the first patient with its investigational in vivo base editing cardiovascular medicine VERVE-102 within a Phase Ib trial, the company said Tuesday.

In the Heart-2 trial, Boston-based Verve is evaluating the activity of VERVE-102 in adult patients with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD). The single ascending dose study has an adaptive design in which researchers are enrolling three to nine patients with these two conditions within four dose cohorts. The trial will provide insights into VERVE-102's impact on blood PCSK9 and low-density lipoprotein cholesterol (LDL-C) levels, as well as its safety, tolerability, and pharmacokinetics.

HeFH is an inherited disorder characterized by elevated LDL-C, and in premature CAD, high cholesterol leads to coronary artery blockages early in life. Patients with these conditions have high LDL-C and are at risk for heart attacks and sudden death.

With VERVE-102, the company is aiming to reduce LDL-C in HeFH and premature CAD patients by inactivating the PCSK9 gene in the liver. The therapy comprises messenger RNA expressing an adenine base editor and a guide RNA targeting the PCSK9 gene. Verve is using its GalNAc-LNP delivery technology, which enables lipid nanoparticles to shuttle the gene editing medicine to liver cells using the asialoglycoprotein receptor or the LDL receptor.

Verve is currently enrolling patients at clinical trial sites in Canada and the UK and is expecting a readout from within its PCSK9 program next year. 

"Dosing the first patient in the Heart-2 Phase 1b clinical trial for VERVE-102 is an important step in the continued progress of our pipeline," Verve Cofounder and CEO Sekar Kathiresan said in a statement. "We are focused on transforming the care of atherosclerotic cardiovascular disease through gene editing medicines that can lead to sustained reductions in blood cholesterol after a single course of treatment."