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CRISPR therapy takes aim at stubborn high cholesterol in first human trial

NCT ID NCT07491172

Disease control Sponsor: CRISPR Therapeutics AG 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 18, 2026 · Updated 10 times

Summary

This early-stage trial tests a new CRISPR-based treatment called CTX310 for people with severe cholesterol and triglyceride problems that don't improve with standard therapies. The treatment uses a tiny fat bubble to deliver gene-editing tools that turn off a gene linked to high lipid levels. About 90 adults aged 18 to 75 will receive a single dose to see if it is safe and can lower harmful blood fats.

What this could mean

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

Active substance
CTX310 (a CRISPR gene-editing therapy delivered via lipid nanoparticles)
What this could lead to
If successful, this could provide a one-time treatment to lower dangerous cholesterol and triglycerides in people who don't respond to standard medications.
What could go wrong
This is a very early (Phase 1) and small trial focused on safety, so it may not work as hoped. There are also unknown risks from using CRISPR to edit genes inside the body.

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 90 people

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

Started

Jun 2024

Expected to finish

Jun 2028

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 75 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.

Key Inclusion Criteria: 1. Age of ≥18 and ≤75 years at the time of signing the informed consent. 2. Able to provide written informed consent. 3. Participants diagnosed with persistent dyslipidemias defined by TG ≥150 mg/dL - and LDL-C ≥70 mg/dL in participants with ASCVD, or LDL-C ≥70 or 100mg/dL in participants with or without ASCVD respectively, or TG ≥500 mg/dL. 4. Refractory to the maximal intensity or MTD of standard of care lines of lipid-lowering therapies available through routine clinical care, for at least 12 weeks prior to screening 5. Female participants must be postmenopausal or surgically sterile. 6. All male participants and their female partners must agree to the use of an acceptable method of effective contraception for the duration of the study. Exclusion Criteria: 1. Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply. 2. Evidence of liver disease, defined as but not limited to: LFTS \>2 × upper limit of normal (ULN), or total bilirubin \>2 × ULN, or INR \>1.5 × ULN, or liver stiffness measured by liver elastography 3. Abnormal or compromised function of kidney, heart, blood or liver. 4. Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1. 5. Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran). 6. Positive serology for HIV, hepatitis B or hepatitis C (antibody, surface antigen orNAT). Serology consistent with prior immunization will be eligible for the trial. 7. Any prior malignancy within the past 5 years, or current malignancy (exceptions for resected or removed basal cell carcinoma, squamous cell carcinoma in situ and carcinoma in situ of the cervix or breast). 8. Women of childbearing potential. Note: Other protocol defined Inclusion/Exclusion criteria may apply. Note: The inclusion and exclusion criteria listed represent the global protocol. Additional or modified eligibility criteria may apply in certain countries in accordance with local regulatory and ethics committee requirements and the approved country-specific protocol.

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Conditions

The condition(s) this trial relates to.

cardiovascular disorder Dyslipidemias familial hypercholesterolemia homozygous familial hypercholesterolemia Hypercholesterolemia hypercholesterolemia, familial, 1 hyperlipidemia hyperlipoproteinemia type V hypertriglyceridemia inherited lipid metabolism disorder Lipid Metabolism Disorders metabolic disease

As listed by the trial registrant

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

Cardiovascular Dyslipidemias Heterozygous familial hypercholesterolemia (HEFH) Homozygous familial hypercholesterolemia (HOFH) Hypercholesterolaemia Hypertriglyceridemia Lipid disorder Metabolic disease Mixed hyperlipemia Severe hypertriglyceridemia (SHTG)

How to take part

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

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    17 sites in 4 countries. The list below names each one and where it is.

  3. 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 ↗

  4. 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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Aotearoa

    RECRUITING

    Auckland, 2025, New Zealand

  • Austin Health

    RECRUITING

    Heidelberg, 3084, Australia

  • CORE Research

    RECRUITING

    Brisbane, 4064, Australia

  • Duke Early Phase Clinical Research

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Flourish Orlando

    RECRUITING

    Orlando, Florida, 32789, United States

  • Integrity Clinical Research

    RECRUITING

    Hialeah, Florida, 33015, United States

  • Jacksonville Center for Clinical Research

    RECRUITING

    Jacksonville, Florida, 32216, United States

  • Monash Medical Center

    RECRUITING

    Melbourne, 3168, Australia

  • NZCR

    RECRUITING

    Christchurch, 8011, New Zealand

  • Oregon Health & Science University (OHSU)

    RECRUITING

    Portland, Oregon, 97239, United States

  • Peters Medical Research

    RECRUITING

    High Point, North Carolina, 27260, United States

  • Richmond Pharmacology

    RECRUITING

    London, SE1 1YR, United Kingdom

  • Royal Adelaide

    RECRUITING

    Adelaide, 5000, Australia

  • Royal Papworth Hospital

    RECRUITING

    Cambridge, United Kingdom

  • Royal Prince Alfred Hospital

    NOT_YET_RECRUITING

    Camperdown, 2050, Australia

  • United Medical Research

    RECRUITING

    Port Orange, Florida, 32127, United States

  • University of Rochester

    RECRUITING

    Rochester, New York, 14642, United States

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