Member Spotlight: Q&A with Verve Therapeutics

Oct 01, 2021

Every month, MassBio spotlights a member company and the great work they’re doing to advance the life sciences industry and support the patients we serve. In October, we spoke with Sekar Kathiresan, M.D., Co-Founder and CEO, Verve Therapeutics, a biotechnology company pioneering a new approach to the care of cardiovascular disease, transforming treatment from chronic management to single-course gene editing medicines. Dr. Kathiresan is a cardiologist and scientist who has focused his career on understanding the inherited basis for heart attack and leveraging those insights to improve the care of cardiovascular disease. Before joining Verve, Dr. Kathiresan’s roles included director of the Massachusetts General Hospital Center for Genomic Medicine, director of the Cardiovascular Disease Initiative at the Broad Institute, and professor of medicine at Harvard Medical School.

Tell us about your organization, its mission, and current initiatives.

Verve is pioneering gene-editing medicines to treat patients with cardiovascular disease. Our goal is to disrupt today’s chronic care model for treating cardiovascular disease by providing a new therapeutic approach with single-course gene-editing treatments.

Our team is bringing together human genetic analysis, gene editing, messenger RNA (mRNA) based therapies, and lipid nanoparticle (LNP) delivery to realize a new future of longevity and vitality for tens of millions of people around the globe with or at risk for cardiovascular disease.

Our lead product candidate, VERVE-101, is designed to be a single-course in vivo liver gene editing treatment. VERVE-101 utilizes LNP-mediated delivery to target the liver and base editing technology to make a single A-to-G base change at a specific site in the PCSK9 gene to disrupt PCSK9 protein production, which subsequently lowers LDL-C levels in the blood.

How does your organization’s activities help patients now and into the future?

Despite advances in treatment over the last 50 years, cardiovascular disease (CVD) remains a global epidemic. The current paradigm of chronic care is fragile – requiring rigorous patient adherence, varying side effects for a subset of the population from existing therapeutics, extensive healthcare infrastructure, and regular healthcare access – and leaves many patients without adequate care. CVD is a leading contributor to reductions in life expectancy and is one of the most expensive health conditions in the U.S., costing the U.S. healthcare system more than $320 billion per year in annual costs and lost productivity. Verve is aiming to disrupt that chronic care model.  

What do you see as the biggest challenge facing the life sciences industry today?

The life sciences industry is making strides to elevate diversity as a key opportunity for improvement, both in clinical trial conduct and in the workforce. And while specific initiatives aimed at increasing diversity within the life sciences and biotech industry have grown, women, people of color, and other previously marginalized populations still lack sufficient representation. With this reality top of mind, Verve remains steadfast in its commitment to promoting equity, diversity, and inclusion across all levels of the organization. We appreciate the benefit that multiple perspectives can provide when building the preeminent gene-editing company focused on protecting the world from cardiovascular disease.

What’s next for your organization / what are you focused on in the coming year?

We’ve seen encouraging preclinical results for VERVE-101. In an in vivo proof-of-concept study, a single intravenous infusion of a base editor targeting PCSK9 achieved 67% whole liver DNA editing and resulted in an 89% reduction in blood levels of PCSK9 protein and a 59% reduction in blood levels of LDL-C in non-human primates (NHP) at two weeks as compared to baseline. These results were durable out to 10 months, suggesting that our vision of a one-time treatment, permanent lowering of LDL cholesterol to treat heart attack is likely to become a reality for patients. We look forward to submitting an IND application and initiate Phase 1 clinical trial for VERVE-101.

We are also looking toward our second gene-editing program, designed to target the ANGPTL3 gene, a regulator of cholesterol and triglyceride metabolism. We aim to select a clinical development candidate and begin IND-enabling studies in 2022.

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