Download #1 peptide tracker for GLP-1, weight loss & muscle gain with 50% OFF! #1 peptide tracker50% OFF! Download now

One-time gene therapy significantly lowers "bad" cholesterol

Amy Peck - Expert Contributor
Contributed by Amy Peck Expert Contributor
Teddy Gordon - Editor-in-Chief
Written by Teddy Gordon Editor-in-Chief
Fact checked
Therapy 2 min read
Updated: Nov 23, 2025
Fact checked

Key Findings

  • CTX310 treatment dramatically decreases LDL and triglycerides by using CRISPR to block the ANGPTL3 gene;
  • For at least 60 days, a single treatment decreased triglycerides by 55% and LDL by nearly 49%;
  • Pilot Phase 1 trial's side effects were minimal, but researchers suggest to start larger studies.

A single infusion of a CRISPR-based (gene-editing technology that uses components from a bacterial defense system to cut and modify DNA) treatment can safely and effectively lower LDL (low-density lipoprotein) cholesterol and triglyceride levels in patients with blood lipid problems, based to a new Phase 1 gene therapy trial.

Researchers tested a treatment called CTX310, which blocks the liver gene ANGPTL3 by using CRISPR-Cas9 integrated in lipid nanoparticles. By blocking the enzymes that break down fats, ANGPTL3 often increases blood fat levels. Triglycerides and "bad" cholesterol drastically decreased once this gene is turned off.

A single infusion was given to 15 adults who took part in the trial at multiple locations in Australia, New Zealand, and the UK. Within two weeks, LDL cholesterol and triglycerides decreased by approximately 49% and 55%, each at the highest dose tested (0.8 mg/kg). [1]

These reductions remained for at least 60 days.

Metric Value Notes
Number of participants 15 Adults with uncontrolled lipid disorders
Median age of participants 53 years Age range: 31–68
Percentage male 87% Majority male cohort
ANGPTL3 reduction (highest dose, 0.7 mg/kg) ~79.7% Mean decline in ANGPTL3 levels
ANGPTL3 reduction (0.8 mg/kg) ~73.2% Slightly lower than 0.7 mg/kg dose
LDL-C reduction (0.8 mg/kg) 48.9% Substantial cholesterol lowering
Triglyceride reduction (0.8 mg/kg) 55.2% Substantial triglyceride lowering
Duration of follow-up 60 days Short-term data after infusion
Study factsheet.

A few people complained of mild side effects, such as nausea, back pain, or temporary increases in liver enzymes.

Why It Matters

Age plays an important role in dyslipidemia, as older adults typically have higher LDL-C levels. [2] This therapy might reduce the need for daily or monthly cholesterol medicine, scientists say this could be a real game-changer. A big clinical breakthrough could be the potential for only one therapy with lasting effects.

Many patients stop taking their cholesterol medications within the first year.

What to Keep in Mind

Worth noting, not every patient responded in the same way, and this was a small, early study. 

Individual genetics, inflammation, or liver fat may have led to the variations in efficiency that the researchers found. Broader research will be required, as a large number of participants were men. Larger trials and longer-term safety will be necessary before this treatment is made widely accessible.

Scientific References

  • 1. Laffin, L. J., Nicholls, S. J., Scott, R. S., Clifton, P. M., Baker, J., Sarraju, A., Nissen, S. E. (2025). Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3. New England Journal of Medicine.
  • 2. Liu, H. H., & Li, J. J. (2015). Aging and dyslipidemia: a review of potential mechanisms. Ageing research reviews, 19, 43-52.

Why trust Longeviters.com

We have strict editorial guidelines. Every review, guide, or a new hack we share goes through a clear set of standards to ensure accuracy & fairness.
Our biohacking experts have analyzed & tested 150+ longevity tools, trends, and wellness products.
Longeviters recommendations & verdicts are not influenced by third-parties and are based on our own experience, findings and expertise.

About the authors

Amy Peck - Expert Contributor

Amy Peck

Expert Contributor

Teddy Gordon - Editor-in-Chief

Teddy Gordon

Editor-in-Chief

TOP 3 Most Popular Coupon Codes

Verified
Nurecover Independence Day Sale
Rating 5.0
Verified
Nordic Wave Memorial Day Sale
Rating 5.0
HigherDose Memorial Day Sale
Rating 5.0
×
50% OFF
Nurecover Independence Day Sale
0 DAYS
00 HOURS
00 MINUTES
00 SECONDS
GET DEAL