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Treatment · Familial hypercholesterolemia

VERVE-102

A one-time base-editing infusion intended to lower LDL cholesterol permanently by switching off PCSK9 in the liver.

Phase II base editingin vivoPCSK9
Clinical research Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.

Eenvoudige uitleg

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.

Dieper ingaan

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.

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