A one-off infusion instead of daily tablets: using the CRISPR-Cas9 gene scissors, researchers have significantly lowered blood lipids for a whole year in patients with difficult-to-treat lipid metabolism disorders. The results of the first trial in humans were presented at the European Society of Cardiology (ESC) Congress 2026 and simultaneously published in the New England Journal of Medicine.
What was studied?
The therapy is called CTX310 and comes from the Swiss company CRISPR Therapeutics. It targets the ANGPTL3 gene in the liver. The protein produced by this gene slows down the breakdown of blood lipids. People who naturally carry an inactive variant have strikingly low cholesterol and triglyceride levels – with no recognisable health disadvantages. The gene scissors are intended to mimic exactly this “natural protection”.
The phase 1 trial included 15 people with lipid metabolism disorders that did not respond sufficiently to conventional medicines. They received a single infusion at doses between 0.1 and 0.8 mg/kg body weight, after pre-treatment with corticosteroids and antihistamines.
Results after twelve months
- LDL cholesterol: minus 52.5 % compared with baseline (highest dose)
- Triglycerides: minus 47.8 % (highest dose)
- Safety: no serious adverse events attributed to the therapy
The durability is particularly noteworthy: initial two-month data had already been published in November 2025. It now appears that the effect remains stable over a year. Since the liver cells are permanently altered, a lifelong effect is the stated goal.
Assessment
First author Dr Luke Laffin of the Cleveland Clinic and his team see the method as a possible route for people in whom statins and other lipid-lowering drugs are not sufficient. At the same time, this is a small, early trial. The US Food and Drug Administration (FDA) recommends a follow-up of 15 years for gene-editing therapies – only then can long-term safety really be assessed. Whether lowering blood lipids also prevents heart attacks and strokes must be shown in larger trials.
Important for the assessment: the trial was funded by CRISPR Therapeutics.
What happens next?
CTX310 is now being tested in a phase 1b trial in severe hypertriglyceridaemia and treatment-resistant hypercholesterolaemia – including in the USA. New data are to be presented as “Late-Breaking Science” at the American Heart Association’s Scientific Sessions on 9 November 2026. In parallel, the company is developing further liver edits, for example against lipoprotein(a) and against treatment-resistant high blood pressure (CTX340).
Note: This article is for general information only and does not replace medical advice. CTX310 is an experimental therapy and is not approved.
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