One CRISPR Infusion Kept Cholesterol and Triglycerides Down for a Full Year
On August 28, 2026, CRISPR Therapeutics presented one-year results for CTX310, a single-infusion gene-editing treatment that switches off the ANGPTL3 gene in the liver. The data, shown at the European Society of Cardiology (ESC) Congress 2026 and published in the New England Journal of Medicine, found that one dose kept both LDL cholesterol and triglycerides down for a full year.
It is one of the clearest signs yet that a one-time gene edit could manage blood fats that today require lifelong medication.
Why ANGPTL3?
ANGPTL3 is a protein made by the liver. It blocks two enzymes, lipoprotein lipase and endothelial lipase, that break down fat-carrying particles in the blood. When ANGPTL3 is active, those particles are cleared more slowly, and levels of triglycerides and LDL cholesterol rise.
Human genetics made ANGPTL3 an attractive target. Some people are born with natural loss-of-function variants in the gene. They have unusually low LDL cholesterol and triglycerides and a lower risk of coronary artery disease, with no known harm from lacking the protein. Natural protective mutations like these, and how scientists turn them into medicines, are the focus of my book The Cheat Codes of Our Genes.
The target is already validated by medicine. Evinacumab (Evkeeza), an antibody that blocks ANGPTL3, is approved for homozygous familial hypercholesterolemia (HoFH), a severe inherited form of high cholesterol. It must be given by infusion every four weeks.
How CTX310 works
CTX310 is given as a single intravenous infusion. It uses a lipid nanoparticle that travels to the liver and delivers two components into liver cells:
Cas9 messenger RNA, which the cell uses to make the Cas9 enzyme.
A guide RNA that directs Cas9 to the ANGPTL3 gene.
Cas9 cuts the gene, and the cell's repair process introduces small errors that disable it. The edited liver cells make far less ANGPTL3. Because the change is in the DNA, the effect is designed to be permanent, while the Cas9 RNA itself is broken down within days.
What the trial showed
The Phase 1a study enrolled 15 adults with hard-to-treat lipid disorders, across four groups: HoFH, heterozygous familial hypercholesterolemia (HeFH), severe hypertriglyceridemia, and mixed dyslipidemia. Doses ranged from 0.1 to 0.8 mg/kg of lean body weight.
At the highest dose, 0.8 mg/kg, the reductions held steady through one year:
ANGPTL3 protein: down 79% on average, and up to 89%.
Triglycerides: down 48% on average, and up to 78%.
LDL cholesterol: down 53% on average, and up to 84%.
Lower doses produced smaller changes, showing a clear dose response. "The durability of the lipid-lowering effect was impressive," said Dr. Luke Laffin, a preventive cardiologist at the Cleveland Clinic.
Safety
No treatment-related serious side effects were reported.
Three participants had Grade 2 infusion reactions, and one had an allergic reaction that resolved.
There were no Grade 3 or higher liver enzyme increases, an important finding for a therapy that edits liver cells.
Because the edit is permanent, every participant will be followed for 15 years to watch for late effects.
How it compares with PCSK9 editing
CTX310 is part of a growing field of one-time gene edits for heart disease. Eli Lilly's VERVE-102 uses base editing to switch off PCSK9, a different liver gene, and has shown LDL reductions of up to 62%.
The two targets do different things. Turning off PCSK9 mainly lowers LDL cholesterol. Turning off ANGPTL3 lowers both LDL and triglycerides, which could help patients with mixed lipid problems. It also lowers LDL through a route that does not depend on the LDL receptor, which is why it can work in HoFH patients whose receptors barely function.
What happens next
CRISPR Therapeutics is moving into a Phase 1b study using a fixed dose equivalent to 0.8 mg/kg. The company expects to share an update on the severe hypertriglyceridemia group in the second half of 2026. Early data from this program were first reported in November 2025, and the new results show the effect lasting well beyond that point.
The bottom line
One infusion cut LDL cholesterol by about half and triglycerides by nearly half, and the drop was still there a year later. The study is small and early, and long-term safety will take years to establish. But CTX310 shows that a single CRISPR edit in the liver can do what patients now need pills or monthly infusions to achieve.
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