SAN FRANCISCO — The promise of genome editing has given rise to potentially curative medicines for rare, life-threatening diseases that generally affect small numbers of patients. But since Verve Therapeutics was founded in 2018, it’s had a wider aperture: using the molecular tools of genome editing to go after the most common cause of death in the world.
The plan, according to Verve CEO Sekar Kathiresan, is to edit out heart attacks for the millions of people at risk for severe cardiovascular disease. It’s a goal that’s more than a little audacious. There’s just one approved genome editing medicine, a treatment for sickle cell disease, and it only passed Food and Drug Administration review last month. Base editing, the CRISPR successor Verve uses to conduct its molecular surgery, is only a few years old.
“When we started, there were so many things that we were kind of going against the grain on,” Kathiresan told STAT at the J.P. Morgan Healthcare Conference in San Francisco this week. “In 2018, it was all about platforms, not about specific diseases and products, or taking this kind of idea for a large indication as opposed to cancer or rare disease. But we’ve always been motivated by a deep conviction in one simple research insight, which is that if your blood cholesterol is really low, lifelong, it is very hard to get a heart attack.”