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 Jun 27, 2026 · Updated 1 time

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Our safety recommendation!

By submitting, you agree to our Terms of use

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

Show contact details

Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Research Site 1
RECRUITING

Adelaide, 5000, Australia
Research Site 10
NOT_YET_RECRUITING

Jacksonville, Florida, 32216, United States
Research Site 11
RECRUITING

Port Orange, Florida, 32127, United States
Research Site 12
NOT_YET_RECRUITING

Portland, Oregon, 97239, United States
Research Site 13
RECRUITING

High Point, North Carolina, 27260, United States
Research Site 14
NOT_YET_RECRUITING

Durham, North Carolina, 27710, United States
Research Site 15
NOT_YET_RECRUITING

Brisbane, 4064, Australia
Research Site 16
NOT_YET_RECRUITING

Brisbane, Australia
Research Site 17
RECRUITING

Orlando, Florida, 32789, United States
Research Site 18
NOT_YET_RECRUITING

Cambridge, United Kingdom
Research Site 2
RECRUITING

Melbourne, 3168, Australia
Research Site 3
RECRUITING

Heidelberg, 3084, Australia
Research Site 4
RECRUITING

Christchurch, 8011, New Zealand
Research Site 5
RECRUITING

Auckland, 2025, New Zealand
Research Site 6
RECRUITING

London, SE1 1YR, United Kingdom
Research Site 7
NOT_YET_RECRUITING

Camperdown, 2050, Australia
Research Site 8
NOT_YET_RECRUITING

London, E1 1BB, United Kingdom
Research Site 9
NOT_YET_RECRUITING

Cleveland, Ohio, 44195, United States