VERVE-102
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Indications
- hypecholesterolemia, elevated plasma LDL cholesterol
Dosage
- one time infusion of 1.0 mg/kg
Mechanism of action
- VERVE-102 consists of a messenger RNA encoding an adenine base-editor protein & a guide RNA targeting PCSK9, encapsulated in a lipid nanoparticle incorporating N-acetylgalactosamine[1]
- editing therapy designed to durably inactivate PCSK9 in the liver
- reduction in plasma LDL cholesterol of 62%[1]
More general terms
References
- ↑ 1.0 1.1 1.2 Vafai SB, Taubel J, Ashdown T, et al In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia. N Engl J Med. 2026. May 25. Not yet indexed in PubMed https://www.nejm.org/doi/full/10.1056/NEJMoa2601283