A Safety and Tolerability Study Evaluating CTX310 in Subjects With Refractory Dyslipidemias

Some people have dangerously high levels of fats (lipids) in the blood — such as LDL cholesterol or triglycerides — that do not respond adequately to standard medications. This is called refractory dyslipidemia, and it significantly increases the risk of heart attack, stroke, and other serious cardiovascular events. CTX310 is an experimental gene-editing therapy that aims to permanently lower lipid levels by modifying a specific gene involved in fat metabolism. This Phase 1 study is the first time CTX310 is being tested in humans. The primary goals are to confirm that a single infusion of CTX310 is safe and tolerable, and to identify the right dose to carry forward into future trials. Participants will receive one intravenous infusion and then be closely monitored over time, with an invitation to join a long-term follow-up study after completion. You may be eligible if you have persistent dyslipidemia that has not responded to the maximum tolerated doses of standard treatments, have adequate kidney, liver, and heart function, and are a postmenopausal woman or a man willing to use contraception for at least 12 months after treatment. People with a history of cancer, active viral infections, significant coagulation disorders, or who have previously received gene therapy are not eligible.