Verve Therapeutics has launched with $58.5 million in funding to pursue a lofty goal: ridding the world of the leading cause of death, coronary heart disease.
The Cambridge, Massachusetts–based biotech firm plans to use gene-editing tools to develop drugs that permanently address the underlying causes of heart disease.
Currently, people with heart disease take daily cholesterol-lowering pills. But they don’t help everyone: in the US alone, someone has a heart attack every 40 seconds, according to the American Heart Association.
Verve wants to change that, says founder and CEO Sekar Kathiresan, a renowned cardiologist who will leave his academic posts at Massachusetts General Hospital, the Broad Institute, and Harvard Medical School to lead the firm. “Imagine an injection administered once a life that safely lowers cholesterol—lifelong—and confers enduring protection against heart attack in adults.”
That theoretical once-and-done injection will be a CRISPR-based therapeutic that can turn off specific genes that cause abnormally high lipid levels in the blood.
CRISPR and related tools have galvanized the biotech industry, but turning them into therapies will mean overcoming several challenges. One major issue is sneaking the drug inside cells so it can snip out stretches of DNA. Verve has already teamed with Alphabet’s life sciences subsidiary Verily to develop lipid nanoparticles that can serve as delivery vehicles for Verve’s drugs.
And because CRISPR therapies have yet to be studied in humans, researchers do not fully understand their off-target effects. To that end, Verve will first treat a rare form of high cholesterol called homozygous familial hypercholesterolemia. If safe in that population, the drug will be tested more broadly. “It’s a progressive phase development strategy from rare orphan disease all the way to protecting the world,” Kathiresan says.