For people with very high LDL cholesterol, treatment may involve taking medications regularly for years. Researchers are now exploring whether gene editing could offer a different approach.
Verve 102 is an experimental treatment being developed to lower LDL cholesterol by targeting the PCSK9 gene in liver cells. It is designed as a one-time intravenous infusion and uses a technology called in vivo base editing, which allows a targeted change to DNA inside the body.
The treatment is being studied particularly in people with conditions such as heterozygous familial hypercholesterolaemia and premature coronary artery disease, who may need additional LDL cholesterol lowering.
VERVE 102 is still an investigational treatment. It is not approved for routine use and cannot currently be considered a replacement for statins or other established cholesterol medicines.
To understand Verve 102, it helps to understand PCSK9. The liver contains LDL receptors that help remove LDL cholesterol, often called bad cholesterol, from the bloodstream. PCSK9 is a protein that promotes the breakdown of some of these receptors.
When PCSK9 activity is reduced, more LDL receptors remain available on liver cells. This allows the liver to remove more LDL cholesterol from the blood.
Verve 102 is designed to target the PCSK9 gene and make a specific DNA change that reduces the production of functional PCSK9 protein.
This is different from simply taking a medicine that temporarily blocks the protein. Gene editing aims to create a change that could potentially have a long-lasting effect.
WHAT HAPPENED IN THE EARLY HUMAN TRIAL?
The early results from the Phase 1b Heart 2 trial have generated interest because researchers observed substantial reductions in both PCSK9 and LDL cholesterol after a single infusion.
Across the doses studied, PCSK9 levels fell by approximately 51% to 88%, while LDL cholesterol fell by approximately 9% to 62%.
At the highest tested dose of 1 mg per kg, LDL cholesterol fell by approximately 62%, equivalent to an average reduction of around 78 mg per dL.
The reduction was reported over 18 months of follow-up, suggesting that the effect can persist well beyond the initial treatment.
However, these are early clinical findings. The Phase 1b study was primarily designed to assess safety, tolerability and biological effects. It was not large enough to establish whether VERVE 102 prevents heart attacks or strokes.
DOES VERVE 102 PERMANENTLY LOWER CHOLESTEROL?
This is where the findings need to be explained carefully.
Verve 102 is designed to produce a durable genetic change, so researchers hope that one treatment could provide long-lasting LDL reduction.
But it has not yet been proven that the effect will last for a patient’s entire lifetime.
The human data currently provide follow-up of up to 18 months. That is encouraging, but it cannot establish what will happen over decades.
Therefore, Verve 102 is better described as a potentially durable one-time gene editing treatment, rather than a treatment proven to permanently lower cholesterol.
No.
Statins remain an established treatment for lowering LDL cholesterol and reducing cardiovascular risk. Other medicines, including ezetimibe and PCSK9 inhibitors, are also available for patients who need additional LDL lowering.
Verve 102 is still being tested and has not been approved as an alternative to these treatments.
Researchers will need to establish its long-term safety, durability and ability to reduce cardiovascular events before it could potentially become part of routine cholesterol care.
The biggest difference is that Verve 102 is attempting to address the genetic pathway involved in cholesterol regulation with a single treatment.
If future studies show that the approach is safe and its effects remain durable, it could potentially change how some people with inherited or difficult-to-treat high cholesterol are managed.
For now, however, the treatment remains experimental.
In simple terms, Verve 102 is a one-time gene editing treatment designed to switch down the PCSK9 pathway in the liver and lower LDL cholesterol. Early results are promising, but much more research is needed before it can be considered an approved treatment.
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