A single CRISPR infusion lowers blood fats in monkeys for three years

by Andrew Zinin, Medical Xpress

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High levels of LDL cholesterol and triglycerides, which are types of blood fats, are major contributors to atherosclerotic cardiovascular disease. Gene-editing technologies offer a potential alternative to lipid-lowering medications. In a new CRISPR experiment, a single infusion of a gene-editing treatment kept triglyceride levels low in monkeys for three years. The findings support the possibility of controlling blood fats with a lasting DNA change, potentially replacing daily doses of medication.

Researchers at Verve Therapeutics and collaborating institutions report the results in Nature. Verve, an Eli Lilly subsidiary, funded the work. Most authors are company employees, and several hold Lilly shares or are named on related patent applications.

A brief treatment, a lasting edit

The treatment disables ANGPTL3, a gene encoding a liver-secreted protein that slows the clearance of fats from the blood. Reducing that protein can lower both triglycerides and LDL cholesterol.

The gene has already been identified as a treatment target: evinacumab, an antibody drug for a severe inherited cholesterol disorder, blocks ANGPTL3 but administration requires infusions every four weeks. Gene editing aims instead to stop the liver from producing the protein.

To build their single-course treatment, the researchers packaged RNA instructions for a CRISPR base editor and an RNA guide inside tiny fat-based particles. The guide directs the editor to ANGPTL3, where changing one DNA letter disrupts the instructions for making the protein. The delivery components and RNA are largely cleared after treatment, and the DNA change is designed to remain.

In the durability study, monkeys with normal blood lipid levels received either one infusion of the treatment or a control formulation. Ten animals in the active group were followed for three years.

In follow-up testing, ANGPTL3 protein levels in the group averaged about 94% below pretreatment levels. Triglycerides averaged 43% lower, while LDL cholesterol fell by a more modest 13%. Liver samples also showed similar levels at seven weeks and three years.

Another entrance into liver cells

The researchers also tested whether the approach could work when LDL receptors, which help clear cholesterol from the blood, were in short supply. Severe deficiency of these receptors underlies homozygous familial hypercholesterolemia, a rare inherited disorder that causes dangerously high cholesterol.

Receptor deficiency creates a delivery problem as well: conventional fat-based particles rely heavily on LDL receptors to enter liver cells. The team's particles carry a sugar-based targeting molecule called GalNAc, which provides access through a different receptor, an approach established in earlier work by members of the research team.

In four monkeys whose LDL-receptor gene had been disrupted, treatment targeting ANGPTL3 reduced average LDL cholesterol from 458 to 247 milligrams per deciliter after 35 days—a 46% drop. This shorter experiment did not establish years of cholesterol lowering in animals with receptor deficiency.

From monkeys to patients

Liver enzymes temporarily rose in the CRISPR treated monkeys, returning to baseline within two weeks. Follow-up tests and tissue examinations found no evidence of lasting liver toxicity.

In cultured human liver cells, the human version of the treatment produced no detectable unintended editing at roughly 3,000 candidate DNA sites. This targeted search cannot exclude every possible unwanted change however.

That human version, called VERVE-201, is being evaluated in a phase 1b trial for people whose blood lipids remain inadequately controlled. The hope is to "alleviate the burdens of chronic lipid-lowering treatment," the researchers write. Whether a lasting edit can safely deliver that benefit is now a question for the human trials.

Written for you by our author Andrew Zinin, edited by Lisa Lock, and fact-checked and reviewed by Robert Egan—this article is the result of careful human work. We rely on readers like you to keep independent science journalism alive. If this reporting matters to you, please consider a donation (especially monthly). You'll get an ad-free account as a thank-you.

Publication details

Taiji Mizoguchi et al., In Vivo CRISPR Base Editing of ANGPTL3 for Durable Lipid Lowering, Nature (2026). DOI: 10.1038/s41586-026-11233-9

Journal information: Nature

Key medical concepts

ANGIOPOIETIN-LIKE 3Triglycerides

Clinical categories

CardiologyClinical pharmacology Who's behind this story?

Andrew Zinin

Master's in physics with research experience. Long-time science news enthusiast. Plays key role in Science X's editorial success. Full profile →

Lisa Lock

BA art history, MA material culture. Former museum editor, paramedic, and transplant coordinator. Editing for Science X since 2021. Full profile →

Robert Egan

Bachelor's in mathematical biology, Master's in creative writing. Well-traveled with unique perspectives on science and language. Full profile →

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