Verve Therapeutics Inc.

Company Overview

Verve Therapeutics is a clinical-stage genetic medicine company pioneering a novel approach to cardiovascular disease (CVD) treatment, shifting from chronic management to single-course gene editing therapies. Its strategy integrates several 21st-century biomedical advances: human genetic analysis, gene editing, mRNA-based therapies, and lipid nanoparticle (LNP) delivery. The company targets genes predominantly expressed in the liver to disrupt the production of lipoproteins responsible for ASCVD.

The company’s pipeline includes single-course in vivo gene editing programs designed to replicate natural disease resistance mutations, turning off specific genes to lower blood lipids and reduce ASCVD risk. The company initially focuses on treating familial hypercholesterolemia (FH), a genetic condition causing persistently high LDL-C levels and an increased risk of early-onset ASCVD.

Verve’s lead product candidate, VERVE-101, aims to disable the PCSK9 gene in the liver permanently. They are conducting the Heart-1 clinical trial, a global Phase 1b open-label study to assess the safety and tolerability of VERVE-101 in patients with heterozygous familial hypercholesterolemia (HeFH), established ASCVD, and uncontrolled hypercholesterolemia. This trial also includes pharmacokinetic analyses and monitoring changes in bloodPCSK9 protein and LDL-C levels.

The trial consists of three parts: (A) a single ascending dose phase, (B) an expansion single-dose cohort where more participants receive the potentially therapeutic dose, and (C) an optional second-dose cohort for eligible participants from the lower dose cohorts in Part A to receive a second treatment. Country-specific protocols have been developed through interactions with regulators in New Zealand, the U.K., and the U.S. FDA to address various eligibility, design, and conduct modifications.

The company’s second product candidate, VERVE-102, also targets PCSK9 and utilizes our proprietary GalNAc-LNP delivery technology.

Financial Highlights (FY 2024)

Company's Business Segments

• Gene Editing Medicines : The company develops single-course gene editing treatments for cardiovascular diseases.