In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia.

A single dose of VERVE-102 achieved up to 88% reduction in PCSK9 and 62% decrease in LDL cholesterol in adults with hypercholesterolemia.

• Why it matters: Lowering LDL cholesterol is crucial for reducing cardiovascular risk, but current therapies often require lifelong treatment; a durable genetic approach could transform management.
• What they did: The phase 1 trial involved 35 adults receiving one intravenous infusion of VERVE-102 at six dose levels, measuring safety, PCSK9, and LDL cholesterol changes over at least 28 days.
• The result: The therapy caused dose-dependent, sustained reductions in PCSK9 and LDL cholesterol with minimal adverse effects, supporting further development of in vivo base editing for cardiovascular disease.