A single injection of an experimental gene-editing therapy has slashed ‘bad’ cholesterol levels by half for a full year, a new study has found.
After 15 participants received an injection of a drug known as CTX310, they still had reduced levels of low-density lipoprotein (LDL) cholesterol one year later.
Those receiving the highest dose saw a 53 per cent decrease in their LDL cholesterol after one dose of CTX310. This is similar to the 50 per cent reduction in cholesterol that can be achieved by taking daily statin pills, say experts.
Chronically high levels of LDL cholesterol, sometimes labelled ‘bad’ cholesterol, lead to furring up of blood vessel walls which in turn increases the risk of heart attacks and strokes.
Currently, the most commonly prescribed medication for high cholesterol is oral statins.
“The issue with a statin is you have to take a pill every day of your life,” says Prof Steve Humphries, a cardiovascular genetics expert at University College London.
“So the big advantage of a one-off treatment is: that’s it. You don’t have to worry about any of that stuff.”
CTX310 works by permanently switching off a gene in the liver called ANGPTL3. This reduces levels of fatty molecules in the blood including cholesterol and triglycerides.
It’s still early days for the drug, which has to be tested in long-term safety trials before it can be approved.
But once we know they’re safe, could gene-editing therapies like CTX310 replace statins – one of the most widely prescribed drugs in the world?
The problem of cost
Don’t expect statins to disappear just yet. Experts say a cheap, proven pill is hard to beat.
“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,” says Prof 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.”
Crucially, adds Humphries, statins are “as cheap as anything”, with each tablet “costing almost nothing”.
Despite gene-editing therapy needing to be given only once, the process of doing so is resource-intensive for a health service.
You have to bring the patient in for an infusion, not only of the drug, but of additional medications that are needed to prevent an immune reaction to the treatment, explains Ray.
All of this requires outpatient clinics and staff with the necessary equipment and training to deliver the treatment. He is doubtful that creating this infrastructure would be feasible at the moment, at least in the UK where he works.
“You can tell me if you think we can do that for three million people in this country, even if it’s once in a lifetime,” says Ray.
Even though statins have to be taken daily for life, he estimates that they are still “probably cheaper”. And the benefit of a long-term treatment is that the cost is spread over time, rather than being paid upfront.
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Access only for those who really need it
The relatively high cost of gene-editing therapies like CTX310 may mean they are given more sparingly – to the people who most need them.
In the CTX310 trial, all 15 participants had previously been diagnosed with disorders that meant they had exceptionally high levels of either cholesterol or triglycerides, and were therefore at greater risk of having a heart attack or stroke compared with the general population.
Before enrolling in the trial, the participants had already tried standard therapies at maximum intensity. So they were likely to be taking combinations of high-dose statins and other lipid-lowering drugs when they received the CTX310 injection.
For such patients, a gene-editing therapy alone may not be enough to normalise their lipid levels, but its long-lasting effect could still improve their quality of life.
“For patients who need lifelong lipid-lowering, these ‘one-and-done’ treatments may offer a future with fewer pills,” says Prof Riyaz Patel, a cardiologist at University College London.
Those with a genetic condition known as homozygous familial hypercholesterolaemia (FH), which affects 1 in 300,000 people, would be prime candidates for a one-off treatment, adds Ray.
People born with homozygous FH have extremely high cholesterol levels from childhood. Their blood is “like treacle”, says Ray, and their cardiovascular risk is so high that they can die of a heart attack before reaching their teenage years.
In a 2026 trial for another gene-editing therapy called VERVE-102, which Humphries and Patel both co-authored, all 35 participants were diagnosed with heterozygous FH, a milder form of the condition, which affects around 1 in 300 people.
A single dose reduced LDL cholesterol by 62 per cent in the highest-dose group.
In people with FH, there is a window of opportunity at a young age when cholesterol levels are high but the consequences are not yet apparent.
If gene-editing therapies like CTX310 and VERVE-102 turn out to be safe long-term, says Ray, they could change the risk trajectory of people affected by FH, if given early in life.
Why timing of treatment matters
Starting treatment early for high cholesterol is important for everyone, not just those affected by FH.
“The prime window is before disease has occurred,” says Ray.
In the future, he says, doctors may be able to predict an individual’s lifetime risk of cardiovascular disease very early on in their lives, by looking at their polygenic risk score – a calculation of overall risk of developing a disease according to a person’s genetic makeup.
But for now, such predictions are not routinely available for the vast majority of the population. Meanwhile, many people will develop atherosclerosis as a result of chronically elevated cholesterol.
Even a one-and-done injection that reduces cholesterol levels by 50 per cent may not be enough in more advanced disease.
“There’s no single drug on the planet that can lower your cholesterol by 80 or 90 per cent because there are multiple pathways that regulate cholesterol,” says Ray.
“For anyone with more advanced disease, you have to combine more than one treatment.”
It looks, then, as though no cholesterol-lowering treatment is likely to monopolise the market any time soon.
Testing of CTX310 continues and it will go through another clinical trial in which around 90 participants across the US, UK, Australia and New Zealand will receive one dose of the drug.
Treatment efficacy and safety will be evaluated over a one-year period, and perhaps then we’ll be one step closer to cutting down cholesterol for good.
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