What if, for long-term cholesterol reduction, one day a single procedure that alters the function of a specific gene directly inside the body would suffice, instead of daily pills?
Initial clinical results show that such a scenario is no longer science fiction.
On August 28, 2026, in New England Journal of Medicine the one-year results of a trial of the experimental therapy CTX310 were published. It uses the CRISPR-Cas9 system to turn off the ANGPTL3 gene directly in liver cells. After a single intravenous infusion, reductions in ANGPTL3, LDL cholesterol, and triglycerides were maintained for at least a year.
Why Turn Off ANGPTL3
ANGPTL3 is a protein produced mainly in the liver that regulates lipid metabolism, in particular by suppressing the activity of lipoprotein lipase and endothelial lipase.
Nature has already suggested a potential therapeutic strategy to researchers. In people with inherited variants leading to loss of ANGPTL3 function, levels of certain atherogenic lipids are significantly lower. This made ANGPTL3 an attractive target for the prevention of cardiovascular diseases.
CTX310 attempts to replicate such an effect artificially.
CRISPR components are placed in lipid nanoparticles and administered intravenously. The nanoparticles predominantly reach the liver, where the CRISPR-Cas9 system introduces a change into ANGPTL3 and reduces the production of the corresponding protein.
That is, it is no longer about a drug that temporarily blocks the protein and requires regular administration, but about an intervention in the very genetic mechanism of its production.
What Happened After a Year
The first phase of the study involved 15 people with severe lipid metabolism disorders. By the time of the new analysis, all of them had been followed for at least 12 months.
The most pronounced effect was observed at the maximum studied dose — 0,8 mg/kg.
After a year, the average ANGPTL3 level remained approximately 79% below baseline, with a maximum reduction reaching 89%.
At the same time, LDL cholesterol decreased by an average of 53%, and triglycerides by 48%.
In some participants, the reduction reached 84% for LDL and 78% for triglycerides.
What is particularly interesting is not only the reduction in the indicators itself, but its duration: the effect persisted a year after a single infusion.
And What About Safety?
So far, the results look promising. In Phase 1a, no dose-limiting toxic effects related to treatment were identified, and during extended follow-up, researchers found no new safety signals related to the therapy.
But caution is needed here.
Fifteen people is a very small group, and one year is a short period for irreversible genome editing. Therefore, participants will require long-term monitoring, and the safety of the method must be confirmed in significantly larger studies.
Moreover, it is not yet known whether such a reduction in lipids will directly lead to a decrease in the number of heart attacks, strokes, and other cardiovascular events. Separate large clinical trials are needed to answer this question.
Will pills no longer be needed?
Not yet.
CTX310 remains an experimental therapy, and the conducted study does not prove that a patient can stop taking statins and other medications after the procedure.
But the concept itself is extremely interesting.
Most modern drugs work as long as a person continues to take them. CRISPR therapy offers a completely different model: to change once the biological mechanism that sustains the pathological process for years.
The study has already moved into Phase 1b. Now a fixed dose of CTX310 is being studied, corresponding to the most effective dose from the first phase, primarily in patients with severe hypertriglyceridemia and refractory lipid metabolism disorders.
It is too early to talk about a "cure forever." We only know that after one procedure, the effect lasts for at least a year.
But this is precisely where an important frontier of modern medicine lies. CRISPR is gradually moving beyond the treatment of rare genetic diseases and approaching conditions that affect millions of people.
Perhaps the medicine of the future will not only control the consequences of metabolic disorders on a daily basis. In some cases, it will be able to change once the molecular mechanism that sustains the disorder.
CTX310 is so far just one of the first steps in this direction.
