CRISPR Gene Therapy Shows Promise in Reducing Cholesterol Levels

A recent study presented at the American Heart Association meeting reveals promising results from a CRISPR gene therapy aimed at reducing harmful cholesterol levels. According to The Straits Times, a single infusion of Crispr Therapeutics' experimental gene therapy, identified as CTX310, was administered to participants, significantly lowering low-density lipoprotein, or LDL cholesterol, and triglycerides by approximately fifty percent in a small group.

Dr. Steven Nissen from the Cleveland Clinic, who led the study, emphasized the significance of these results, noting that they have never had a treatment capable of achieving such reductions in both LDL and triglycerides simultaneously.

The therapy functions by silencing a gene known as ANGPTL3, with previous studies indicating that individuals with an inactive version of this gene exhibit lower lifetime risks of heart disease without adverse effects.

The trial included fifteen patients, aged thirty-one to sixty-eight, with high triglycerides, high LDL cholesterol, or both, who had previously failed other treatments. Among four patients receiving the highest dose, the average triglyceride level dropped by fifty-five percent, and LDL levels decreased by fifty percent just two weeks post-treatment, with levels remaining low for at least two months.

Temporary reactions, including nausea and elevated liver enzymes, were observed in three participants but resolved quickly. Participants will be monitored for one year post-trial, with an option for an extended fifteen-year follow-up.

Crispr Therapeutics' chief, Sam Kulkarni, noted the potential of gene editing for treating common diseases, contrasting it with traditional gene therapies that often target rare diseases. The company plans to submit data to US regulators and aims to initiate phase two studies by two thousand twenty-six, with hopes of having a market-ready product in four to five years.

Initially targeting genetically related high cholesterol, this therapy could eventually benefit millions of Americans if approved. Kulkarni anticipates that the cost will likely be less than one hundred thousand dollars, a stark contrast to existing therapies that can exceed two million dollars.

This study marks a pivotal moment in gene therapy, potentially transforming patient care for cardiovascular diseases.