08/05/2026
A new experimental cholesterol treatment called VERVE-102 is being studied as a possible one-time therapy for lowering LDL cholesterol, often called “bad cholesterol.” Instead of relying on daily pills or repeated injections, the treatment is designed to make a precise genetic change in the liver.
VERVE-102 targets a gene called PCSK9, which plays an important role in controlling LDL cholesterol levels in the blood. When PCSK9 activity is reduced, the body can remove LDL cholesterol more effectively.
The treatment uses base editing, a refined form of gene editing that changes a single DNA letter without cutting both strands of DNA. It is delivered through a single IV infusion and is intended to switch off PCSK9 in liver cells.
Early results came from the Heart-2 Phase 1b clinical trial, which initially reported data from 14 participants. These participants had heterozygous familial hypercholesterolemia, premature coronary artery disease, or both, meaning they were people at high risk who needed strong and lasting LDL reduction.
In the early Heart-2 update, VERVE-102 produced dose-dependent reductions in both PCSK9 and LDL cholesterol. At the higher dose level, the average LDL cholesterol reduction was reported at around 53%, while the largest individual reduction reached 69% after one infusion.
The early safety report stated that there were no treatment-related serious adverse events among the first 14 participants and no clinically significant laboratory abnormalities observed. However, the study is still in an early stage, so larger trials and longer follow-up are needed.
If future studies confirm that the treatment is safe, durable, and effective, VERVE-102 could represent a major shift in cholesterol care. It may be especially important for people with inherited high cholesterol or very high cardiovascular risk who struggle to keep LDL levels controlled over many years.