Bad cholesterol slashed 62% by single dose of gene-editing drug in small trial
Summary
A new gene-editing drug called VERVE-102 has been tested in a small early trial to lower bad cholesterol (LDL) with a single treatment. The drug was safe and reduced bad cholesterol by up to 62% in the highest dose group, potentially lowering heart disease risk if effects last long-term.Key Facts
- VERVE-102 uses gene-editing technology delivered by mRNA in nanoparticles targeting liver cells.
- The drug edits the PCSK9 gene to stop production of a protein that raises bad cholesterol levels.
- In the trial, 35 patients received different doses to test safety and effectiveness.
- The highest dose group (1 mg/kg) saw an average 62% drop in LDL cholesterol.
- The lowest dose group (0.3 mg/kg) saw a 9% drop in LDL cholesterol.
- No serious side effects were reported, only mild and temporary liver enzyme increases.
- The drug’s positive effects were sustained during the follow-up period of up to 18 months for some patients.
- Lowering LDL by this amount may reduce heart disease risk by about 50% if sustained over 20 years.
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