In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia - PubMed
3 hours ago
- #Base Editing
- #Hypercholesterolemia
- #Clinical Trial
- VERVE-102 is an adenine base-editing therapy targeting PCSK9 to lower LDL cholesterol in hypercholesterolemia patients.
- A phase 1, open-label, single-ascending-dose study with 35 participants showed dose-dependent reductions: up to 88% in PCSK9 and 62% in LDL cholesterol at the highest dose (1.0 mg/kg).
- Treatment was generally safe with mild-to-moderate infusion-related reactions and transient liver enzyme elevations, with durable effects observed for at least one year in 15 participants.