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Scientists have edited a gene that may reduce high cholesterol permanently
A one-time gene-editing infusion targeting ANGPTL3 reduced LDL cholesterol and triglycerides by 50% in 15 participants, with effects lasting at least 60 days.
- 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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CRISPR gene therapy slashes 'bad' cholesterol, triglycerides by half in small study
A single infusion of CRISPR Therapeutics' experimental gene therapy was safe and reduced levels of harmful LDL cholesterol and triglycerides by half in four people taking the highest dose, raising hope for a one-time treatment.
·United Kingdom
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Anti-Aging, Acupuncture and Health NewsGene Therapy Shows Promise for Cutting LDL in Half in Tough-to-Treat Dyslipidemia
(MedPage Today) -- NEW ORLEANS – A single injection of a gene-edited protein was deemed productive and safe in patients with dyslipidemia that was unresponsive to lipid-lowering therapy, according to a phase I trial. IV CTX310 is an in vivo...
·New York, United States
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Leaning Left6Leaning Right0Center14Last UpdatedBias Distribution70% Center
Bias Distribution
- 70% of the sources are Center
70% Center
L 30%
C 70%
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