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

NCT ID NCT07491172

Recruiting now Disease control Sponsor: CRISPR Therapeutics AG Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jul 24, 2026 · Updated 5 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.

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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)

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

Aotearoa
RECRUITING

Auckland, 2025, New Zealand
Austin Health
RECRUITING

Heidelberg, 3084, Australia
Barts
RECRUITING

London, E1 1BB, United Kingdom
CORE Research
NOT_YET_RECRUITING

Brisbane, 4064, Australia
Cleveland Clinic
NOT_YET_RECRUITING

Cleveland, Ohio, 44195, United States
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)
NOT_YET_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
NOT_YET_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
NOT_YET_RECRUITING

Rochester, New York, 14642, United States

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