One-Time Gene Edit Cuts Cholesterol in Half: CRISPR Therapy Shows Promise in Landmark Human Trial

A New Frontier in Heart Health

A one-time gene-editing therapy may offer a lifelong solution to high cholesterol, according to early human trial results presented at the American Heart Association’s annual meeting in New Orleans.

The Phase 1 study, involving 15 participants, showed that a single infusion of an experimental CRISPR-based medicine — CTX310, developed by CRISPR Therapeutics — safely and effectively reduced low-density lipoprotein (LDL) cholesterol and triglycerides by nearly 50% within two months.

If proven safe in larger studies, the therapy could transform how doctors treat heart disease, the leading cause of death in the United States.

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Permanent Genetic Fix

Unlike daily cholesterol-lowering drugs such as statins, which need lifelong adherence, CTX310 works by editing a gene in the liver that regulates cholesterol production.

The treatment uses CRISPR, a Nobel Prize–winning tool that allows scientists to “cut and correct” DNA. In this case, it disables the ANGPTL3 gene, which normally produces a protein that limits the liver’s ability to break down fats. Turning off this gene enables the body to clear more cholesterol naturally.

Some people are born with a less active version of ANGPTL3 and have lifelong protection against heart disease — a phenomenon researchers are now trying to replicate through gene editing.

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Dramatic Results, But Questions Remain

Dr. Steven Nissen, chief academic officer at the Cleveland Clinic’s Heart, Vascular & Thoracic Institute and a lead investigator in the study, called the findings “spectacular.”

“If you’d asked me 15 years ago whether we could safely alter genes in humans to prevent heart disease, I would have said it was impossible,” Nissen said.

Participants received a single infusion lasting about 4.5 hours. Among those given the highest dose, LDL cholesterol dropped by 48.9% and triglycerides fell by 55.2% within two months.

Side effects were generally mild, including temporary nausea and back pain. One participant experienced a short-term rise in liver enzymes, which returned to normal. Another volunteer died months later from an unrelated cause, researchers noted.