Researchers in Australia have begun the first human test of a new gene‑editing therapy that could slash bad cholesterol and triglycerides in people who struggle to control their blood fats.
The trial uses CTX310, a one‑time CRISPR‑Cas9 treatment delivered by tiny fat‑based particles to the liver. The therapy turns off the ANGPTL3 gene, which normally raises LDL (“bad”) cholesterol and triglycerides—two key risk factors for heart disease.
Three patients at the Victorian Heart Hospital (a partnership of Monash Health and Monash University) received the high‑dose version of CTX310. After just two weeks, their LDL dropped an average of 50 % and triglycerides fell by 55 %, staying low for at least two months. All 15 study participants saw near‑60 % reductions, with only mild, short‑term side effects.
“CTX310 is the first treatment to dramatically lower both LDL and triglycerides at the same time,” says Dr. Stephen Nicholls, director of the hospital and lead investigator. “A single course that works for months could change how we prevent heart disease, cut costs, and improve lives.”
The study, published in the New England Journal of Medicine, is part of a global Phase 1 trial running across Australia, New Zealand and the UK. Future research will test CTX310 in larger, more diverse groups to confirm its safety and effectiveness.
Source: ianslive
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