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