VERVE-102
VERVE-102 is Verve Therapeutics' in vivo base-editing therapy designed to provide a one-time, permanent reduction in LDL cholesterol in patients with heterozygous familial hypercholesterolemia (HeFH) — a dominant genetic disorder affecting 1 in 250 people that causes severely elevated LDL from birth. A single IV infusion delivers an adenine base editor via lipid nanoparticles to liver cells, converting a single DNA letter in the PCSK9 gene to a permanent loss-of-function variant that prevents the liver from degrading LDL receptors. The goal is lasting LDL-C reduction from one infusion, as an alternative to lifelong statin therapy and bi-weekly PCSK9 inhibitor injections.
Upcoming catalysts
Programs
Familial Hypercholesterolemia
HeFH affects 1 in 250 people and causes 3–10× elevated lifetime cardiovascular risk from birth due to impaired LDL receptor function; many patients cannot reach LDL-C targets despite maximal statins plus bi-weekly PCSK9 inhibitor injections. VERVE-102 aims to permanently knock out hepatic PCSK9 with a single IV infusion; Phase 1b Cohort 3 dose-escalation data measuring PCSK9 knockdown %, LDL-C reduction, and hepatic safety in HeFH patients are expected in 2026.