Novel treatment could slash cholesterol, triglyceride levels

Early results have indicated the potential benefit of a new therapy in patients with high cholesterol.

In a Phase 1 trial published in The New England Journal of Medicine, researchers assigned 15 adult patients with hypercholesterolemia, hypertriglyceridemia or mixed dyslipidemia who were undergoing treatment with lipid-lowering therapy to receive intravenous CTX310 to target the ANGPTL3 gene. The treatment comprised lipid-nanoparticle-encapsulated CRISPR-Cas9 messenger RNA and guide RNA to target hepatic ANGPTL3 to induce a loss-of-function mutation.

After a follow-up of 60 days, the researchers found that CTX310 was effective at substantially reducing low-density lipoprotein cholesterol, triglyceride and ANGPTL3 levels. However, there were wide variations in reductions among the study participants.

They reported no dose-limiting adverse effects, three Grade 2 infusion-related reactions that subsided following the cessation of treatment administration, and two severe adverse events, including one sudden death 179 days posttreatment.

The findings demonstrated that CTX310 could help mitigate the lifetime risk of atherosclerotic cardiovascular disease.

Read more: The New England Journal of Medicine

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