11/11/2025
In a Phase 1, first-in-human study, the experimental CRISPR-Cas9 treatment CTX310 safely reduced LDL cholesterol and triglycerides after a single infusion. By turning off the ANGPTL3 gene in the liver, researchers achieved lasting lipid reductions in patients with hard-to-treat disorders. The findings—presented at the American Heart Association’s Scientific Sessions 2025 and published in the New England Journal of Medicine—highlight a major step forward in precision cardiovascular medicine.
Source: Medical Xpress - Medical and Health News
https://bit.ly/4qX1g7W
In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, according to a preliminary late-breaking science presentation Saturday at the American Heart Association's S...