The patients came in with high cholesterol. And it was the kind that clings stubbornly to arteries and shadows families for generations. They walked into a clinical trial and left, perhaps, with a glimpse of the future.
In early results from a small study, a single infusion of an experimental medicine called VERVE-102 cut levels of LDL cholesterol — the so-called “bad cholesterol” — by as much as 69%. And it did so not with daily pills or monthly injections, but with a one-time genetic edit.
“This is reality; it’s not science fiction. We’re actually doing it,” said Dr. Riyaz Patel, a cardiologist at University College London, for the BBC.
Flipping a genetic switch
Globally, almost half the adult population suffers from high cholesterol. While we have drugs like statins that can lower cholesterol, you need to take them daily. But VERVE-102 is no ordinary drug. It’s a form of in vivo base editing — a precise method of rewriting DNA without breaking the genetic code. This particular edit targets PCSK9, a gene that helps regulate how efficiently the liver clears out LDL cholesterol from the blood. Fewer PCSK9 proteins mean more LDL receptors on liver cells, and less cholesterol clogging the bloodstream.
The early trial, known as Heart-2, enrolled 14 patients with heterozygous familial hypercholesterolemia (HeFH), a genetic disorder that leads to dangerously high cholesterol levels from an early age. These weren’t your average patients with a bad diet, they often require lifelong therapy and still face an elevated risk of heart disease.
Each participant received a single intravenous infusion of VERVE-102, which packages the gene editor inside a lipid nanoparticle (LNP) designed to deliver it directly to the liver. There were no reported side effects and no signs of liver damage in any participant.
Researchers gave participants three different doses.
- At 0.3 mg/kg, patients saw a 21% reduction in LDL cholesterol.
- At 0.45 mg/kg, the reduction averaged 41%.
- At 0.6 mg/kg, LDL dropped 53% on average, with one patient achieving a 69% reduction.
These are extremely promising results, despite the study being in its early stages.
Why this could be huge
Cholesterol, a waxy substance essential for the body, becomes a killer when it accumulates in arteries, forming plaques that can rupture and cause heart attacks or strokes. Lowering LDL is one of the most effective ways to prevent these events.
VERVE-102 was designed initially for people with inherited high cholesterol, but its underlying mechanism — permanently switching off the PCSK9 gene — could be relevant to anyone struggling with high LDL cholesterol. PCSK9 plays a central role in regulating how much LDL the liver can remove from the bloodstream. Inhibiting this gene helps lower cholesterol in almost anyone, regardless of their genetic background.
In fact, many existing cholesterol-lowering drugs, like PCSK9 inhibitors, already target the same protein. The difference is that VERVE-102 aims to make that change permanent, with a single treatment.
More study is still needed
The initial results of the clinical trial have not yet been peer-reviewed.
The next step is to test higher doses. Two patients in the 0.7 mg/kg cohort have already received VERVE-102. So far, no red flags have emerged. Verve expects to release full results from this dose escalation phase later this year and begin a Phase 2 trial — a larger test that will include U.S. patients for the first time.
Meanwhile, pharmaceutical giant Eli Lilly is watching closely. It has the right to co-develop and co-commercialize the drug if it chooses to “opt in.” That decision is expected in the second half of 2025, when we should also have more data on the effectiveness of this treatment.
This treatment is not a cure for everyone with high cholesterol. But it could be a lifeline for those struggling with it the most.
