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Gene-Editing shot aims to slash cholesterol that drugs Can't touch

NCT ID NCT06451770

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jul 17, 2026 · Updated 1 time

Summary

This early-stage study tests a single dose of VERVE-201, a gene-editing therapy, in 36 adults with dangerously high cholesterol and triglycerides that don't respond to standard treatments. The treatment aims to permanently turn off a liver gene (ANGPTL3) to lower blood fats. The main goal is to check safety, but researchers will also measure how well it works.

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 36 people

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

Started

Oct 2024

Expected to finish

Dec 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: * Refractory hypercholesterolemia * Refractory hypertriglyceridemia Exclusion Criteria: * Active or history of chronic liver disease * Current treatment with monoclonal antibody targeting ANGPTL3 or prior treatment within specified timeframe * Clinically significant or abnormal laboratory values as defined by the protocol

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Get notified about this study

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Conditions

The condition(s) this trial relates to.

Hypercholesterolemia hyperlipidemia Hyperlipoproteinemia Type II hypertriglyceridemia

As listed by the trial registrant

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

Hypercholesterolemia Hypertriglyceridemia Refractory hyperlipidemia

How to take part

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

  1. The places running it

    16 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

    Clearwater, Florida, 33756, United States

  • Clinical Study Center

    RECRUITING

    Miami Lakes, Florida, 33016, United States

  • Clinical Study Center

    RECRUITING

    Port Orange, Florida, 32127, United States

  • Clinical Study Center

    RECRUITING

    New York, New York, 10029, United States

  • Clinical Study Center

    RECRUITING

    Adelaide, Australia

  • Clinical Study Center

    RECRUITING

    Melbourne, Australia

  • Clinical Study Center

    RECRUITING

    Chicoutimi, Canada

  • Clinical Study Center

    RECRUITING

    Montreal, Canada

  • Clinical Study Center

    RECRUITING

    Toronto, Canada

  • Clinical Study Center

    RECRUITING

    Amsterdam, Netherlands

  • Clinical Study Center

    RECRUITING

    Cape Town, South Africa

  • Clinical Study Center

    RECRUITING

    Johannesburg, South Africa

  • Clinical Study Center

    RECRUITING

    Liverpool, United Kingdom

  • Clinical Study Center

    RECRUITING

    London, United Kingdom

  • Clinical Study Center

    RECRUITING

    Manchester, United Kingdom

  • Clinical Study Center 2

    RECRUITING

    Manchester, United Kingdom

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