Crispr Gene Therapy Shows Promise in Cholesterol Reduction

A recent study presented at the American Heart Association meeting reveals that Crispr gene therapy has successfully reduced levels of harmful LDL cholesterol and triglycerides by half in a small group of participants. According to The Straits Times, a single infusion of Crispr Therapeutics' experimental gene therapy, known as CTX310, was administered to fifteen patients aged 31 to 68, all of whom had high triglycerides, high LDL cholesterol, or both, and had previously not responded to other treatments. The lead researcher, Dr. Steven Nissen from the Cleveland Clinic, reported that among the four patients who received the highest dose, triglyceride levels decreased by an average of fifty-five percent, and LDL cholesterol levels dropped by fifty percent just two weeks after treatment, with these levels remaining low for at least two months following the infusion.

CTX310 works by switching off a gene called ANGPTL3, which has been linked to lower lifetime risks of heart disease without adverse effects. The therapy is particularly notable as it could replace the need for daily pills or monthly injections, offering a one-time treatment that is both safe and effective. Dr. Luke Laffin, co-leader of the study, expressed that if future trials confirm the therapy's safety and efficacy, it could significantly change treatment practices for patients with high cholesterol.

The trial was conducted in Australia, New Zealand, and Britain, testing five different doses of CTX310. While three participants experienced temporary side effects, including nausea and elevated liver enzymes, these issues resolved quickly. Participants will be monitored for one year following the trial, with an option for an additional fifteen years of follow-up.

Crispr Therapeutics chief Sam Kulkarni noted the potential of gene editing technologies in treating common diseases, as most gene therapies have primarily focused on rare diseases. The company aims to submit its findings to U.S. regulators and hopes to begin phase two studies by 2026, with plans to potentially bring the therapy to market in four to five years. Initially targeting genetics-related high cholesterol, the therapy could become an option for millions of Americans if approved. Unlike other gene therapies, such as the nearly two million dollar treatment for sickle cell disease, this cholesterol treatment is expected to be a straightforward infusion procedure, potentially costing less than $100,000, as indicated by Kulkarni.