Cleveland Clinic Trial Shows Gene-Editing Therapy Dramatically Lowers Dangerous Blood Fat Levels
A Phase 1b clinical trial conducted by Cleveland Clinic has demonstrated that a single-dose CRISPR-Cas9 gene-editing therapy, CTX310, significantly reduced triglyceride levels in patients suffering from severe hypertriglyceridemia. The findings were presented at the American Heart Association’s annual meeting in Chicago. The trial involved 10 adults with severe hypertriglyceridemia, a condition that elevates the risk of heart disease. Thirty days after receiving a single intravenous infusion of CTX310, participants experienced an average reduction of 63% in triglyceride levels, with some reductions reaching up to 85%. The therapy targets the ANGPTL3 gene, which is a key regulator of lipid metabolism. Beyond triglycerides, the treatment also lowered non-HDL cholesterol, apolipoprotein B, and triglyceride-rich lipoprotein cholesterol levels. The treatment was generally well tolerated, with no serious side effects reported.
