"In a step toward the wider use of gene editing, a treatment that uses Crispr successfully slashed high cholesterol levels in a small number of people. In a trial conducted by Swiss biotech company Crispr Therapeutics, 15 participants received a one-time infusion meant to switch off a gene in the liver called ANGPTL3. Though rare, some people are born with a mutation in this gene that protects against heart disease with no apparent adverse consequences."
"The Nobel Prize-winning Crispr technology has mostly been used to address rare diseases, but these latest findings, while early, add to the evidence that the DNA-editing tool could be used to treat common conditions as well. "This will probably be one of the biggest moments in the arc of Crispr's development in medicine," Samarth Kulkarni, CEO of Crispr Therapeutics, tells WIRED. The company is behind the only approved gene-editing treatment on the market, Casgevy, which treats sickle cell disease and beta thalassemia."
A Phase I trial by Crispr Therapeutics enrolled 15 participants aged 31 to 68 with uncontrolled LDL cholesterol and triglycerides. The intervention was a one-time infusion designed to switch off the liver gene ANGPTL3. The highest dose reduced LDL cholesterol and triglycerides by an average of about 50 percent within two weeks. Effects persisted for at least 60 days, the trial's duration. The study was conducted in the UK, Australia, and New Zealand between June 2024 and August 2025. Results were presented at the American Heart Association meeting and published in The New England Journal of Medicine.
Read at WIRED
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