Overview
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.
Eligibility
Inclusion Criteria:
- Diagnosis of HeFH or premature CAD
- Females of non-childbearing potential or males
Exclusion Criteria:
- Homozygous familial hypercholesterolemia
- Active or history of chronic liver disease
- Current treatment with PCSK9 inhibitor or prior treatment within specified timeframe
- Clinically significant or abnormal laboratory values as defined by the protocol