Cholesterol levels cut in half with one-time gene editing drug in trial
15 days ago
- #Cholesterol
- #Gene Therapy
- #CRISPR
- A Phase 1 clinical trial involving 15 people showed that a CRISPR-based gene-editing therapy safely and effectively reduced LDL cholesterol and triglycerides by about 50%.
- The therapy targets the ANGPTL3 gene in the liver, which normally boosts cholesterol levels, mimicking the effect of naturally low-functioning versions of the gene.
- Unlike daily statins, this therapy is designed to work permanently after a single infusion, potentially offering a lifelong solution for high cholesterol.
- The trial participants, aged 50s and 60s, had uncontrolled high LDL or triglycerides, with median levels far above healthy thresholds before treatment.
- Side effects were minimal, including temporary spikes in liver enzymes and minor issues like nausea or back pain during infusion.
- Experts express both excitement and caution, noting the need for long-term safety studies before widespread use, despite the promising early results.
- CRISPR technology, first used in 2012, is gaining traction, with the FDA approving the first CRISPR-based drug for sickle cell disease in 2023.
- Future phases of the CTX310 treatment trials will include more participants, including those in the U.S., to further assess efficacy and safety.