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Scientists have edited a gene that may reduce high cholesterol permanently

  • On Saturday, CRISPR Therapeutics reported one infusion reduced LDL cholesterol and triglycerides by about half in 15 volunteers in a Phase I trial presented at the American Heart Association meeting in New Orleans and published in The New England Journal of Medicine.
  • The drug targets the ANGPTL3 gene to switch off its function in the liver, mimicking a protective genetic mutation found in about 1 in 250 people in the U.S.
  • Participants received varying doses via an infusion lasting about two and a half hours in the Phase I trial across the U.K., Australia, and New Zealand, with ages 31 and 68 and baseline median LDL 155 mg/dL and triglycerides 192 mg/dL.
  • One participant died months after the infusion, and researchers plan Phase 2 clinical trials soon to test if a one-time gene edit can safely prevent heart attacks and strokes.
  • CRISPR Therapeutics, maker of Casgevy, cautioned that long‑term safety and costs remain unknown, despite potential to give people a cure, said Dr. Luke Laffin.
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With gene modification, doctors could one day permanently reduce dangerously high cholesterol levels, possibly eliminating the need for medication, according to a new pilot study published this Saturday in the New England Journal of Medicine.

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Merck, the company that introduced statins in the world nearly 40 years ago, has a new, intensely powerful pill that can reduce dangerous LDL cholesterol levels to almost never seen in adults. The new pill, enlicitide, blocks a liver protein, PCSK9, which slows the body’s ability to eliminate cholesterol. With most blocked PCSK9 levels, LDL levels plummet and rates of heart attacks and strokes in high-risk patients fall by up to 20 percent in th…

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npr broke the news in Washington, United States on Saturday, November 8, 2025.
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