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

Brief summary (registry, in English)

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.

What you commit to

• Study type: Interventional · Phase 1
• Planned participants: 85
• From 30 Apr 2024 to the primary completion date, Oct 2026
• 27 sites in 6 countries
• Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study design.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Who the study is looking for

Age in the registry: 18 years to 70 years · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (27)

Sites in registry order. Save your city in your profile to sort them by distance.

Related publications

• In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia.N Engl J Med · 2026 · PMID 42187087