A single injection of an experimental gene-editing therapy cut bad cholesterol levels by half after one year, a new [url=https://www.nejm.org/doi/10.1056/NEJMc2609825?referrer=https%3A%2F%2Fwww.google.com%2F]study[/url] found.
Fifteen participants who received an injection of the drug CTX310 maintained reduced levels of low-density lipoprotein cholesterol twelve months later, according to findings presented at the European Society of Cardiology annual meeting and published in the New England Journal of Medicine, ScienceDaily reported.
Those receiving the highest dose saw a 52.5 per cent decrease in their LDL cholesterol and a 47.8 per cent drop in triglycerides. This reduction matches the fifty per cent drop typically achieved by taking daily statin pills.
Chronically high levels of LDL cholesterol lead to [url=https://academic.oup.com/eurheartj/article/38/32/2459/3745109]furring up of blood vessel walls[/url], which increases the risk of heart attacks and strokes. Oral statins currently serve as the standard treatment, but patients must take a pill every day.
“The issue with a statin is you have to take a pill every day of your life,” said Steve Humphries, a cardiovascular genetics expert at University College London, according to BBC Science Focus Magazine.
“So the big advantage of a one-off treatment is: that’s it. You don’t have to worry about any of that stuff,” Humphries said.
Liver Gene Disabling
CTX310 works by permanently switching off a gene in the liver called ANGPTL3. This process reduces levels of fatty molecules circulating in the blood.
The treatment uses CRISPR-Cas9 gene-editing technology to make targeted changes to DNA in the liver. Patients in the trial received doses ranging from 0.1 to 0.8 mg/kg after first receiving corticosteroids and antihistamines to prevent an immune reaction.
“Building upon the initial data presented in November 2025, the durability of the lipid-lowering effect was impressive,” said Luke Laffin, a Cleveland Clinic cardiologist and first author of the study, according to ScienceDaily.
“It is encouraging that there were no serious safety events related to CTX310 in the trial and in the year following treatment,” Laffin said.
Cost and Infrastructure Hurdles
Despite the durability of a single infusion, experts say cheap daily pills remain difficult to beat on cost. Statins cost almost nothing per tablet, whereas gene-editing therapies require outpatient clinic infrastructure, specialized training, and additional medications.
“When you have a global population of eight billion people and the number one cause of death is cardiovascular disease, you’re trying to find a cost-effective way of reducing that,” said Kosh Ray, a cardiologist and public health expert at Imperial College London.
“Given that statins are pretty safe and well-tolerated by the majority, it’s going to be statins,” Ray said.
Ray questioned whether health services could build the required infrastructure to deliver infusions to millions of people. He estimates that daily statins spread the cost over time rather than requiring a large upfront expense.
Targeting Severe Lipid Disorders
High costs mean therapies like CTX310 may be reserved for patients who need them most. Trial participants had exceptionally high cholesterol or triglyceride levels and had already tried maximum-intensity standard therapies.
“For patients who need lifelong lipid-lowering, these ‘one-and-done’ treatments may offer a future with fewer pills,” said Riyaz Patel, a cardiologist at University College London.
Patients with homozygous familial hypercholesterolaemia, a genetic condition affecting 1 in 300,000 people, represent prime candidates. These individuals have extremely high cholesterol from childhood, giving their blood a consistency resembling treacle.
A separate [url=https://www.nejm.org/doi/full/10.1056/NEJMoa2601283]trial[/url] evaluated a gene-editing therapy called VERVE-102 in 35 participants with heterozygous familial hypercholesterolaemia. A single dose reduced LDL cholesterol by 62 per cent in the highest-dose group.
Trial Continuation Plans
Researchers plan to continue long-term safety follow-up for participants for another 15 years, in line with Food and Drug Administration recommendations for gene-editing therapies. Meanwhile, testing of CTX310 [url=https://clinicaltrials.gov/study/NCT07491172]continues in a [clinical trial][/url] involving approximately 90 participants across the United States, United Kingdom, Australia, and New Zealand.
“We look forward to continuing to investigate this therapy in a larger number of patients,” Laffin said.
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