A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease

Summary

VT-10201 is an Open-label, Phase 1b, Single-ascending Dose Study That Will Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia (HeFH) or Premature Coronary Artery Disease (CAD) Who Require Additional Lowering of LDL-C. VERVE-102 Uses Base-editing Technology Designed to Disrupt the Expression of the PCSK9 Gene in the Liver and Lower Circulating PCSK9 and LDL-C. This Study is Designed to Determine the Safety and Pharmacodynamic Profile of VERVE-102 in This Patient Population.

Timeline

Start

2024-04-30

Primary completion

2026-08

Completion

2027-08

Publications

• Vafai SB, Taubel J, Ashdown T, Patel RS, Diamondali S, Cegla J, Soran H, Bashir B, Abitbol A, Gaudet D, Lauziere A, Brunham LR, Newby DE, Nicholls SJ, Scott RS, Kerr J, Tardif JC, Lunken C, Humphries SE, Karsten V, Tyler PD, Zhang X, Huniti N, Flight PA, Jensen CL, Falzone R, Biedenkapp JC, Lister T, Stolz LE, Khera AV, Kathiresan S. In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia. N Engl J Med. 2026 May 25. doi: 10.1056/NEJMoa2601283. Online ahead of print.

Drugs

Indications