A landmark Phase 1b clinical trial led by researchers at the Cleveland Clinic has demonstrated that a one-time CRISPR-Cas9 gene-editing therapy can dramatically reduce dangerous blood fat levels in patients with severe hypertriglyceridemia. Presented at the American Heart Association’s annual meeting, the investigational treatment—known as CTX310—targets the ANGPTL3 gene responsible for regulating lipid metabolism. Thirty days after receiving a single intravenous infusion, participants experienced an average 63% drop in triglyceride levels (with reductions reaching up to 85%) alongside substantial decreases in non-HDL cholesterol, offering a potential long-term safeguard against cardiovascular disease and acute pancreatitis.
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