For Healthcare Professionals

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


About the study

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.

Who can take part

You may be eligible to participate in the study if you meet the following criteria:


Inclusion Criteria:

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


Exclusion Criteria:

  1. Active or history of chronic liver disease
  2. Current treatment with PCSK9 monoclonal antibody therapy
  3. Current or past treatment with inclisiran
  4. Clinically significant or abnormal laboratory values as defined by the protocol
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Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply

Contact the study center to learn if this study is a good match for you.

Study’s details


Heterozygous Familial Hypercholesterolemia,Atherosclerotic Cardiovascular Disease,Hypercholesterolemia

Age (in years)

18 - 75


Phase 1

Participants needed


Est. Completion Date

Dec 31, 2024

Treatment type



Verve Therapeutics, Inc. identifier


Study number


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