Simple explanation
Statins and cholesterol injections work, but only while you keep taking them. VERVE-102 is designed to be given once: it switches off a liver gene called PCSK9, and lower LDL cholesterol should follow for life. Some people are naturally born with that gene switched off, and they have low cholesterol and no apparent ill effects — which is the whole reason this target was chosen.
Go deeper
VERVE-102 is a lipid-nanoparticle base editor installing a loss-of-function change in hepatic PCSK9, intended as a single-dose treatment for heterozygous familial hypercholesterolemia and premature atherosclerotic cardiovascular disease. It has FDA Fast Track designation and reported durable LDL cholesterol reduction in Phase 1b. Eli Lilly acquired Verve Therapeutics in 2025 for approximately $1 billion plus contingent value rights, one of which is triggered by dosing the first patient in a US Phase 3 trial.
Sources
- Eli Lilly and Company · 2025
Lilly to acquire Verve Therapeutics to advance one-time treatments for people with high cardiovascular risk ↗