NederlandsOne-time gene edit sharply lowers dangerous blood fat in small study
In ten adults with very high triglycerides, this blood fat fell by an average of 63 per cent 30 days after a single infusion of the CRISPR treatment CTX310. Next, researchers will study how long the effect lasts.
A one-time gene edit has sharply lowered the level of fat in the blood of ten adults who had far too much of it. Thirty days after a single infusion of the experimental treatment CTX310, their triglycerides were on average 63 per cent lower, and in some participants as much as 85 per cent lower. The US hospital Cleveland Clinic reports this.
Triglycerides are the most common fat in human blood. In severe hypertriglyceridaemia, a strongly raised level, the risk of heart and vascular disease and of acute inflammation of the pancreas (pancreatitis) goes up. People have to keep taking existing medicines for the rest of their lives, often as a monthly or three-monthly injection.
Cardiologist Ashish Sarraju of Cleveland Clinic presented the results on 9 November 2026 at the annual meeting of the American Heart Association in Chicago. He calls them encouraging, precisely because these patients are at extra risk of both pancreatitis and heart and vascular disease.
Switching off one gene in the liver
The liver makes the protein ANGPTL3. It acts as a brake on an enzyme that clears fat from the blood. People who naturally have a non-working version of this gene have lower blood fats and, according to the news site Tech Times, also a lower chance of narrowed coronary arteries.
CTX310 copies this. Tiny fat droplets carry the parts of the CRISPR-Cas9 gene scissors through the blood to the liver. There, the gene scissors cut the ANGPTL3 gene so that it no longer works. This change in the liver cells is permanent.
In the participants, the level of ANGPTL3 fell by more than 65 per cent. Other blood values linked to heart and vascular disease also went down, such as non-HDL cholesterol (on average 51 per cent lower) and apolipoprotein B (23 per cent lower), Tech Times reports.
A severely affected group
All ten participants had diabetes, and earlier fat-lowering treatments had not helped them enough. Three of them had already had pancreatitis. At the start, their triglycerides were on average (the median) above 11 millimoles per litre.
According to Cleveland Clinic, the participants generally tolerated the treatment well, without serious side effects caused by the treatment. In none of the 25 people who have received CTX310 so far did liver values rise in a worrying way, Tech Times reports.
Those 25 also include an earlier study in 15 people with a less severe disorder of fat metabolism. There, after a year, triglycerides were still 48 per cent lower on average and LDL cholesterol 53 per cent lower. This suggests that the effect can last a long time.
The new results concern ten people, one month after treatment. The next step is research in larger groups and over a longer period: does the effect hold for years, does the gene edit stay safe, and does the treatment really prevent pancreatitis and heart problems? CTX310 was developed by the biotech company CRISPR Therapeutics, based in Zug, Switzerland, which funds the research.
What does this mean for the road to the singularity?
This study shows that a single treatment can strongly reduce a lifelong metabolic disorder for which people now need medicines for years. If larger studies confirm how well it works and how safe it is, a one-time procedure in the liver could take its place next to pills and injections. This fits a broader movement: more and more diseases are not just kept under control but tackled at the source, and that brings a longer healthy life a good deal closer.
What is the technological singularity? Read the explanation →
