According to the company, this tiny edit should be enough to permanently lower a person’s levels of “bad” LDL cholesterol, the fatty molecule that causes arteries to clog and harden over time.
Patients in New Zealand were at an inherited risk of very high cholesterol and already had heart disease. However, the company believes that the same technology could eventually be used in millions of people to prevent cardiovascular disease.
“If this works and is safe, this is the answer to a heart attack — that’s the cure,” said Sekar Kathiresan, CEO of Verve, a genetic researcher who founded the company three years ago.
It’s been a decade since scientists developed CRISPR technology, which makes targeted changes to DNA in cells, but until now it’s only been used in people with rare diseases like sickle cell anemia It was tried on the body, and only as part of an exploratory experiment.
If Verve’s experiment is successful, it could herald wider use of gene editing to prevent common diseases. Most of the world’s population has high levels of LDL, but many cannot control it. Worldwide, more people die from atherosclerotic cardiovascular disease than from other diseases.
“Of all the different genome editing going on in the clinic, this one may have the most profound impact because of the large number of people who could benefit,” said Eric Topol, a cardiologist and researcher at Scripps Research.