Editing cholesterol genes could stop the biggest killer on earth

According to the company, that 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 with time.

The patient in New Zealand had an inherited risk for extra-high cholesterol and was already suffering from heart disease. However, the company believes the same technique could eventually be used on millions of people in order to prevent cardiovascular disease.

“If this works and is safe, this is the answer to heart attack—this is the cure,” says Sekar Kathiresan, a gene researcher who started Verve three years ago and is the company’s CEO.

It’s been 10 years since scientists developed CRISPR, a technology for making targeted changes to the DNA in cells, but until now the method has been tried only on people suffering from rare diseases like sickle-cell anemia, and only as part of exploratory trials.

If Verve’s experiment works, it could signal far wider use of gene editing to prevent common conditions. Large swaths of the world’s population have LDL that is too high, but many people can’t get it under control. Worldwide, more people die of atherosclerotic cardiovascular disease than from anything else.

 “Of all the different genome editing ongoing on the clinic, this one could have the most profound impact because of the number of people who could benefit,” says Eric Topol, a cardiologist and researcher at Scripps Research.