A first-in-human Cleveland Clinic trial found that one infusion of an experimental CRISPR-Cas9 treatment continued to lower cholesterol and triglycerides for a year in people whose conditions were difficult to control with medicine.
The Phase 1 study included 15 patients. At the highest dose, LDL cholesterol was 52.5 percent lower than at the start of the study after 12 months, while triglycerides were 47.8 percent lower.
The treatment, called CTX310, works in the liver by switching off ANGPTL3, a gene involved in both measures. Cleveland Clinic reported that no serious safety events related to the therapy occurred during the yearlong follow-up.
This is still early research, not an approved treatment. The results were presented at the European Society of Cardiology meeting and published in the New England Journal of Medicine, and a larger study is planned.
