In a new article published in the New England Journal of Medicine, researchers report that a gene-editing therapy helped people with increased levels of cholesterol and triglycerides maintain lower levels of these lipids for at least a year. The findings build on earlier results of the therapy, reported last November.
In the small study, 15 people with severe forms of high cholesterol and triglycerides showed drops in the two lipids of up to 50%, which the latest results show lasted for at least a year.
The fact that the people in the study were able to maintain lower levels of LDL and triglycerides for up to a year means that CRISPR was able to edit enough cells in the liver, where much of the body’s cholesterol and triglycerides are produced, to give patients the benefit of having lower levels of the lipids. It also shows that these edited cells continue to produce new generations of cells that carry the CRISPR edit in AGNPTL3. People who received the highest dose of the CRISPR therapy saw levels of the ANGPTL3 enzyme drop by nearly 80%, which contributed to a drop in LDL and triglycerides of about 50%. “This is a really big step for CRISPR to show the durability of the result,” says Dr. Luke Laffin, co-director of the Center for Blood Pressure Disorders at the Cleveland Clinic and lead author of the study.
Kulkarni says the company has already begun the next phase of studies and anticipates additional results by the end of this year. That study will continue to focus on people with severe cases of high cholesterol and triglycerides, which affects about two to three million people in the U.S. If that study and further late-stage studies of the CRISPR therapy continue to show positive results, the company might consider conducting studies in people with more average elevations in their lipids.
“The biggest promise of gene-editing in cardiovascular medicine is the notion of a one-time treatment that would be all you need,” he says. “Once you reduce levels of ANGPTL3, it remains durably reduced—presumably for life, but at least for one year. It’s very exciting.”
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