So Far, So Good for PCSK9-Targeting Gene Therapy in Hypercholesterolemia

A single dose of Lilly's PCSK9 base editor, VERVE-102, reduced PCSK9 by up to 88% and LDL-C by up to 62%,…

A single dose of Lilly's VERVE-102 base editor reduced PCSK9 by up to 88% and LDL-C by up to 62% in Phase 1b, with effects sustained for 18 months across 35 patients with familial hypercholesterolemia or premature CAD. For health tech and biopharma investors, this signals a credible shift from chronic lipid-lowering therapy (statins, PCSK9 inhibitors) to a one-time genomic intervention, with Phase 2 enrollment starting before end of 2026.