Bad cholesterol slashed 62% by single dose of gene-editing drug in small trial
Ars Technica • • business
Key Points:
- An experimental gene-editing therapy, VERVE-102, designed to lower bad cholesterol (LDL) after a single infusion, has shown promising safety and efficacy results in a Phase I trial involving 35 patients.
- The therapy uses mRNA-based gene editing to permanently disrupt the PCSK9 gene in liver cells, reducing the enzyme that destroys LDL receptors, thereby lowering LDL cholesterol levels.
- Patients receiving the highest dose experienced an average 62% reduction in LDL cholesterol, which could potentially cut cardiovascular disease risk by 50% if sustained long-term.
- No serious adverse events were reported, though a mild, temporary increase in a liver enzyme suggested minor liver injury; LDL reductions have been sustained up to 18 months in some groups.
- Developed by Verve Therapeutics (acquired by Eli Lilly), VERVE-102 has received FDA Fast Track designation, but further larger and longer trials are needed to confirm safety and effectiveness.