When 35 Londoners stepped into a clinic to receive a single infusion of VERVE-102, they became part of a medical milestone: the first patients to test a gene-editing therapy that could fundamentally reshape how we treat high cholesterol. The results, published in the New England Journal of Medicine, show the treatment reduced "bad" cholesterol by up to 62% in a dose-dependent manner, with effects lasting up to 18 months—all from one infusion.
This matters urgently because heart attacks and strokes remain among the UK's biggest killers, and they're often driven by dangerously high levels of LDL cholesterol. More than seven million Britons currently take daily medications like statins to manage their cholesterol, but the reality of long-term compliance is brutal: up to half of patients stop taking their pills within a year. Some struggle with daily dosing discipline; others experience side effects. For those living with inherited high cholesterol or premature heart disease, the burden of lifelong medication can feel relentless.
VERVE-102 works by copying something that nature already does in rare individuals. The therapy uses gene editing to switch off the PCSK9 gene, which normally instructs the liver to produce a protein that prevents the body from clearing LDL cholesterol from the blood. People born with a naturally inactive version of this gene enjoy remarkably low cholesterol throughout their lives and dramatically lower heart disease risk. The new therapy aims to deliver that same protection through a single, permanent edit.
The Phase 1b trial enrolled 35 adults with either heterozygous familial hypercholesterolemia (an inherited form of very high cholesterol) or premature coronary artery disease—patients referred through UCLH who carried serious cardiovascular risk. At the highest dose tested, the reduction in LDL cholesterol reached 62%, with some participants tracked for up to 18 months showing sustained benefit. Safety appeared strong at the top dose: no serious side effects emerged, though some participants experienced mild infusion reactions and temporary, minor changes in liver tests.
Professor Riyaz Patel, a consultant cardiologist at Barts Health and UCLH and one of the trial's local leads, called it "an extremely exciting milestone." His observation captures the shift this represents: "The therapy has the potential to provide a 'one and done' approach to a very common condition, which would be transformative in preventing heart attacks and strokes over the long term." For patients weary of daily pills or monthly injections, the vision of a permanent solution is profound.
The research was supported by clinicians from UCL, UCLH, and Barts Health NHS Trust, with findings presented at the European Atherosclerosis Society Congress in Athens in May. The trial is sponsored by Verve Therapeutics, owned by Eli Lilly, and larger studies are already planned. Though it remains early days, the signal is unmistakable: a treatment that once seemed like science fiction—using gene editing to rewrite our biology and prevent disease—is now showing real promise in real patients. For millions managing cholesterol today, that possibility feels like genuine hope.