Photo: Cards84664 / Wikimedia Commons / CC BY 4.0
CHICAGO, Ill. – A one-time CRISPR-Cas9 gene-editing infusion lowered triglycerides by an average of 63% after 30 days in 10 adults with severe hypertriglyceridemia, according to results from a Cleveland Clinic Phase 1b trial presented Monday at the American Heart Association’s annual meeting. Reductions reached as high as 85% in individual participants, and no serious side effects related to the treatment were observed.
The photograph shows the Cleveland Clinic’s Sydell and Arnold Miller Family Pavilion in Cleveland in April 2021. It is an archival photo of the institution, not a scene from the trial.
The findings were presented during a late-breaking science session, according to the Cleveland Clinic’s news release dated November 9. The investigational treatment, called CTX310, is designed to turn off the ANGPTL3 gene, which helps regulate lipid metabolism and contributes to elevated triglyceride levels. Participants received a single intravenous infusion.
Thirty days later, triglyceride levels had fallen by an average of 63%, and ANGPTL3 levels had dropped by more than 65%. The clinic said non-HDL cholesterol, apolipoprotein B and triglyceride-rich lipoprotein cholesterol also fell. The release described the treatment as generally well tolerated.
“The results are encouraging because they suggest it may be possible to achieve substantial triglyceride reductions with a single treatment,” said Dr. Ashish Sarraju, a Cleveland Clinic cardiologist who presented the study. He said the result is particularly important for this group because severe hypertriglyceridemia raises the risk of pancreatitis as well as cardiovascular disease.
Researchers hope the approach could give long-lasting benefits without ongoing medication, but the clinic said more research is needed to learn how durable the effects are. The study builds on earlier Phase 1 research that covered a range of lipid disorders and doses. It was funded by CRISPR Therapeutics AG of Zug, Switzerland, which means the sponsor of the therapy paid for the work.
The results come with clear limits. The trial was small, with 10 participants, and the reported outcome is a change in blood measures at 30 days. The release did not say the therapy reduces heart attacks, strokes or pancreatitis, and CTX310 is an investigational treatment, not an approved one. It also did not report the doses used in this trial, a full list of adverse events or a comparison with a control group.
Still to be answered are how long the reductions last, whether safety holds over longer follow-up, and whether larger controlled trials confirm the early readout. The clinic said the aim is long-lasting benefit without ongoing medication, a goal the data so far cannot confirm.
JBizNews Desk | Chicago, Ill.
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