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Gene-Editing shot could slash cholesterol for good

NCT ID NCT06164730

Disease control Sponsor: Verve Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Sep 03, 2026 · Updated 9 times

Summary

This early-stage trial tests a single IV dose of VERVE-102, a gene-editing drug designed to permanently lower LDL (bad) cholesterol by turning off a specific gene in the liver. It involves 85 adults with inherited high cholesterol or early heart disease who still need lower cholesterol despite current treatments. The main goal is to check safety, but researchers will also measure how much cholesterol drops.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
VERVE-102 (a gene-editing drug given by IV infusion)
What this could lead to
If successful, this could lead to a one-time treatment that dramatically lowers bad cholesterol, reducing heart attack risk without daily pills.
What could go wrong
This is an early, small safety trial (Phase 1b) with only 85 people. It may not work as hoped, and there are unknown risks from gene editing, including possible liver effects or immune reactions.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 85 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2024

Expected to finish

Oct 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 70 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

coronary artery disorder familial hypercholesterolemia Hyperlipoproteinemia Type II

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Heterozygous familial hypercholesterolemia Premature coronary heart disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    27 sites in 6 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Clinical Study Center

    RECRUITING

    Dothan, Alabama, 36305, United States

  • Clinical Study Center

    RECRUITING

    Pomona, California, 91768, United States

  • Clinical Study Center

    RECRUITING

    Boca Raton, Florida, 33434, United States

  • Clinical Study Center

    RECRUITING

    Jacksonville, Florida, 32216, United States

  • Clinical Study Center

    RECRUITING

    Winter Park, Florida, 32789, United States

  • Clinical Study Center

    RECRUITING

    Indianapolis, Indiana, 46237, United States

  • Clinical Study Center

    RECRUITING

    High Point, North Carolina, 27260, United States

  • Clinical Study Center

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Clinical Study Center

    RECRUITING

    DeSoto, Texas, 75115, United States

  • Clinical Study Center

    RECRUITING

    Renton, Washington, 98057, United States

  • Clinical Study Center

    RECRUITING

    Adelaide, Australia

  • Clinical Study Center

    RECRUITING

    Melbourne, Australia

  • Clinical Study Center

    RECRUITING

    Sydney, Australia

  • Clinical Study Center

    RECRUITING

    Chicoutimi, Canada

  • Clinical Study Center

    RECRUITING

    Hamilton, Canada

  • Clinical Study Center

    RECRUITING

    Montreal, Canada

  • Clinical Study Center

    RECRUITING

    Québec, Canada

  • Clinical Study Center

    RECRUITING

    Toronto, Canada

  • Clinical Study Center

    RECRUITING

    Vancouver, Canada

  • Clinical Study Center

    RECRUITING

    Rehovot, Israel

  • Clinical Study Center

    RECRUITING

    Christchurch, New Zealand

  • Clinical Study Center

    RECRUITING

    Birmingham, United Kingdom

  • Clinical Study Center

    RECRUITING

    Cambridge, United Kingdom

  • Clinical Study Center

    RECRUITING

    Edinburgh, United Kingdom

  • Clinical Study Center

    RECRUITING

    London, United Kingdom

  • Clinical Study Center

    RECRUITING

    Manchester, United Kingdom

  • Clinical Study Center

    RECRUITING

    Nottingham, United Kingdom

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