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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.

Penjelasan sederhana

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.

Pelajari lebih dalam

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

Written by The CRISPR Atlas editorial team Last updated Aug 25, 2026

Connected in the Atlas

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Technologies

Base Editing

Diseases

High Cholesterol and Familial Hypercholesterolemia

Genes

PCSK9

Companies

Eli Lilly and CompanyVerve Therapeutics
Hanya informasi edukatif Halaman ini adalah referensi, bukan nasihat medis. Status riset dan regulasi dapat berubah; periksa tanggal pembaruan terakhir di atas dan konfirmasi hal-hal penting dengan sumber primer yang tercantum.