An experimental therapy called VERVE-102 lowered LDL cholesterol by up to 62 percent after a single dose in an early safety study. The results come from a Phase I trial involving 35 patients with high cholesterol or early cardiovascular disease. Data were published this week in the New England Journal of Medicine.
Researchers reported that the treatment appeared safe, with no serious side effects even at the highest dose. Participants experienced only a temporary mild rise in liver enzymes. The drug uses mRNA packaged in nanoparticles to edit the PCSK9 gene in liver cells, permanently reducing production of a protein that raises LDL levels.