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Evaluating VERVE-102 for lowering cholesterol in specific heart disease patients

Open-label, Phase 1b, Single Ascending Dose Study to Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia or Premature Coronary Artery Disease Who Require Additional Lowering of Low-density Lipoprotein Cholesterol

Phase 1 Interventional Verve Therapeutics, Inc. · NCT06164730

This study is testing a new gene-editing treatment called VERVE-102 to see if it can lower cholesterol levels in people with certain heart conditions.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment85 (estimated)
Ages18 Years to 70 Years
SexAll
SponsorVerve Therapeutics, Inc. Industry-sponsored
Locations22 sites (Dothan, Alabama and 21 other locations)
Trial IDNCT06164730 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and pharmacodynamic profile of VERVE-102, a gene-editing therapy designed to lower LDL cholesterol levels by disrupting the PCSK9 gene in the liver. It targets patients with heterozygous familial hypercholesterolemia or premature coronary artery disease who require additional LDL-C lowering. The study is open-label and employs a single-ascending dose approach to assess the effects of VERVE-102. Participants will be monitored for safety and efficacy throughout the trial.

Who should consider this trial

Good fit: Ideal candidates include individuals diagnosed with heterozygous familial hypercholesterolemia or premature coronary artery disease who need further LDL-C reduction.

Not a fit: Patients with homozygous familial hypercholesterolemia or those with active liver disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly lower cholesterol levels and reduce the risk of heart disease in patients with specific genetic predispositions.

How similar studies have performed: Other studies utilizing gene editing technologies for cholesterol management have shown promise, indicating potential for success in this novel approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Diagnosis of HeFH or premature CAD

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

Where this trial is running

Dothan, Alabama and 21 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Heterozygous Familial HypercholesterolemiaPremature Coronary Heart DiseaseVERVE-102Familial HypercholesterolemiaCoronary Artery DiseaseDose EscalationGene EditingBase Editing
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.