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New One-Shot Gene Editing Therapy May Reduce Bad Cholesterol By 62%

A single infusion cut PCSK9 by up to 88% and LDL cholesterol by up to 62%, with no treatment-related serious adverse events reported.

  • A landmark study published in the New England Journal of Medicine revealed that an experimental gene-editing therapy successfully lowered harmful LDL cholesterol by up to 62% in high-risk patients.
  • The one-time intravenous treatment, known as VERVE-102 and developed by Eli Lilly and Verve Therapeutics, uses precise base-editing technology to permanently deactivate the PCSK9 gene in the liver.
  • Data from the Phase 1b clinical trial involving 35 adults showed that the treatment was generally well-tolerated and that its cholesterol-lowering effects remained durable for up to 18 months in some participants.
  • While cardiologists view this "one-and-done" approach as a historic breakthrough for people with inherited high cholesterol, experts caution that larger, long-term trials are still required to fully verify its permanent safety profile.
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PR Newswire broke the news in United States on Monday, May 25, 2026.
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