Gene Therapy Cuts Bad Cholesterol 62% With Single Dose

Imagine never having to take cholesterol pills again after getting a single treatment that works for life.

That future just got closer. Researchers testing an experimental gene-editing drug called VERVE-102 just published promising results from 35 patients in the New England Journal of Medicine.

The patients who received the highest dose saw their bad cholesterol drop by 62%, bringing levels down to an average of 78 mg per deciliter. For people with high cholesterol, this kind of reduction could cut their risk of heart disease by half if it holds for 20 years.

Here's the exciting part: the effect has lasted up to 18 months so far across all patient groups, with no signs of wearing off.

VERVE-102 works by permanently turning off a gene that produces an enzyme called PCSK9, which normally destroys receptors that clear bad cholesterol from the blood. Without PCSK9, the liver keeps more of these helpful receptors, naturally lowering cholesterol levels.

The treatment uses a single infusion of nanoparticles containing gene-editing instructions. These particles target liver cells and make a precise change to one DNA letter in the PCSK9 gene, essentially breaking it permanently.

The trial tested six different doses, starting low to ensure safety. No serious side effects occurred, though some patients had a temporary, mild increase in a liver enzyme.

Participants were people who developed heart disease early in life or had inherited high cholesterol conditions. They represent patients at the highest risk who struggle most with current treatments.

The Ripple Effect

Coronary artery disease remains one of the world's leading causes of death. Millions of people take daily cholesterol medications like statins, but many still can't get their levels under control.

Dr. Riyaz Patel, a cardiologist at University College London who worked on the trial, called the results encouraging. He noted that many patients struggle to maintain healthy cholesterol despite ongoing efforts with available medicines.

Eli Lilly, which bought the drug's developer Verve Therapeutics for $1.3 billion last year, hopes this could eventually offer an option beyond lifetime medication management. The FDA has already granted VERVE-102 a Fast Track designation for faster review.

The company's senior vice president Sekar Kathiresan sees potential for the treatment to expand beyond just the highest-risk patients, offering more people a one-time solution to high cholesterol.

Of course, this is still early days. The trial was small, and researchers need larger studies with longer follow-up periods to confirm the drug truly works and stays safe over time.

But for the millions managing high cholesterol with daily pills, a single treatment that could protect their hearts for decades offers genuine hope.