A Study of VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular Disease

Open-label, Phase 1b, Single-ascending Dose and Optional re Dosing Study to Evaluate the Safety of VERVE-101 Administered to Patients With Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, and Uncontrolled Hypercholesterolemia

VT-1001 is an open-label, phase 1b, single-ascending dose study that will evaluate the safety of VERVE-101 administered to patients with heterozygous familial hypercholesterolemia (HeFH), atherosclerotic cardiovascular disease (ASCVD), and uncontrolled hypercholesterolemia. VERVE-101 uses base-editing technology designed to disrupt the expression of the PCSK9 gene in the liver and lower circulating PCSK9 and LDL-C in patients with established ASCVD due to HeFH. This study is designed to determine the safety and pharmacodynamic profile of VERVE-101 in this patient population.

Inclusion Criteria:

• Male and/or female participants 18 up to 75 years at time of signing of informed consent
• Female participants not of child-bearing potential
• Diagnosis of HeFH
• Established ASCVD

Exclusion Criteria:

• Active or history of chronic liver disease
• Current treatment with PCSK9 monoclonal antibody therapy
• Current or past treatment with inclisiran
• Clinically significant or abnormal laboratory values as defined by the protocol