A single infusion of an experimental CRISPR therapy lowered triglycerides by an average of 63% after 30 days in a group of ten adults with severe hypertriglyceridemia. The phase 1b results for CTX310 were presented at the American Heart Association’s scientific meeting in Chicago.
CTX310 targets ANGPTL3, a gene involved in regulating blood fats. Before treatment, the group’s median triglyceride level exceeded 1,000 mg/dL. High triglyceride levels are associated with acute pancreatitis and cardiovascular disease, making the group relevant for further research.
Seven participants had reached at least 90 days of follow-up. The study reported one moderate infusion reaction that resolved and no treatment-related serious adverse events at the data cutoff. The small, open-label study measured changes in blood markers. Larger and longer studies are needed to establish durability, safety and whether the approach prevents pancreatitis or cardiovascular events.
