Even after decades of drug breakthroughs aimed at preventing heart attacks, they remain the world’s leading cause of death. The pills and injections on the market do the job of lowering the cholesterol that clogs blood vessels and puts people at risk of a heart attack. But not everyone has access to them, and some won’t stick to treatment plans that can last the rest of their lives. Verve Therapeutics Inc. is proposing a radical solution: altering a person’s genome—the body’s instruction manual—to stop the buildup of bad cholesterol. “We’re on the cusp of potentially transforming that model to a one-and-done treatment,” says Sekar Kathiresan, chief executive officer of the Cambridge, Mass.-based company.
Verve plans to initially target those who’ve already had a heart attack because of extremely high cholesterol caused by a hereditary condition known as familial hypercholesterolemia, which affects 31 million people globally. If it works to reduce low-density lipoprotein (LDL), or “bad,” cholesterol in that group, the company would look to widen the treatment pool, eventually aiming to give it to young people as a preventive measure, though it’s too early to say when that could happen.