CRISPR Therapeutics is advancing CTX310, a one-time intravenous CRISPR-Cas9 therapy designed to disable the ANGPTL3 gene, which regulates blood-fat levels. In a 15-person Phase 1 study, the highest tested dose—0.8 mg/kg—produced substantial reductions in LDL (“bad”) cholesterol and triglycerides. Reported averages were roughly 49% lower LDL and 55% lower triglycerides after about 60 days. Follow-up data indicated that the effect persisted for one year: LDL fell by about 53% and triglycerides by about 48% among the highest-dose participants. ANGPTL3 levels declined by roughly 79%. The treatment was generally well tolerated, with no treatment-related serious adverse events reported. Some infusion-related reactions, including fever, nausea and back pain, occurred at higher doses. Because the trial was small and early-stage, the results do not establish long-term safety or cardiovascular benefit. Gene-editing patients require extended monitoring, reportedly up to 15 years. CRIS...
- Get link
- X
- Other Apps