Gene Editing Treatment Targets Cholesterol-Linked Protein in New Trial Data

The *New England Journal of Medicine* posted findings on CTX310, a treatment that uses CRISPR-Cas9 gene editing — a technique that makes precise changes to DNA — to target a protein called ANGPTL3. That protein plays a role in regulating fats (lipids) in the bloodstream, and higher levels are associated with raised cardiovascular risk.

The research article, shared by the journal’s official account and tagged to cardiology and the ESC Congress, focuses on the durability of the gene editing — in other words, how long the effect lasts after a single treatment. CTX310 works by editing the gene that produces ANGPTL3 to durably reduce its activity, rather than requiring ongoing medication.

Gene editing therapies of this kind are still in early clinical stages. The journal published this as a peer-reviewed article reporting completed one-year follow-up data from a small phase 1a trial.