HEART DISEASE

VERVE THERAPEUTICS, Boston IN THE FUTURE, a one-time infusion of a gene-altering medicine could permanently lower cholesterol and dramatically reduce the risk of having a heart attack or stroke. That may sound like something straight out of science fiction, but it’s actually the goal of Dr. Sekar Kathiresan, an esteemed former cardiologist from Massachusetts General Hospital who launched the Boston biotech firm Verve Therapeutics in 2019 to make his vision a reality. Verve is developing a therapy that uses a form of CRISPR gene editing to inactivate a gene called PCSK9 in the liver. In studies with monkeys, the genetic tweak significantly reduced levels of LDL cholesterol, sometimes called “bad” cholesterol. Although there are already approved drugs that target PCSK9 to lower cholesterol, they are not as widely used as some doctors would like. A one-and-done approach might change that. “Gene editing could be a definitive approach that would really make a difference,” says Dr. Jorge Plutzky, director of preventive cardiology at Brigham and Women’s Hospital. “There’s a lot of basis for thinking that it should work.” The company will first test the therapy in people with familial hypercholesterolemia, an inherited condition that affects about 1.3 million Americans and increases the risk of earlyonset heart disease. If the treatment proves safe and effective, Verve may expand to broader groups of people at risk of heart disease. Verve began the first tests of its therapy in humans in New Zealand last summer. That study is focused on gauging the safety of the therapy and finding the right dose to use. Verve is still waiting for permission from the FDA to start a similar trial in the United States.