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Health

Single CRISPR dose halves cholesterol over full year in small trial

An experimental gene-editing therapy sustained lower lipid levels in 15 patients with treatment-resistant disorders, according to ScienceDaily.

Updated Sep 27, 2026 · 14:17 UTC1 reports

The short version

  • A Phase 1 trial evaluated the single-dose CRISPR therapy CTX310 in 15 patients with lipid disorders resistant to standard treatments.[ScienceDaily]
  • At the highest dose tested, participants experienced a 52.5% reduction in LDL cholesterol and a 47.8% drop in triglycerides after one year.[ScienceDaily]
  • No serious treatment-related safety events were reported during the 12-month follow-up period.[ScienceDaily]
  • Researchers plan to track participants for 15 years to evaluate the long-term safety of the experimental treatment.[ScienceDaily]

Key facts

  • The Phase 1 trial tested CTX310 in 15 patients with lipid disorders that did not respond adequately to standard medications.[ScienceDaily]
  • At 12 months, patients receiving the highest dose had a 52.5% drop in LDL cholesterol and a 47.8% decrease in triglycerides.[ScienceDaily]
  • CTX310 works by targeting and disabling the ANGPTL3 gene in the liver to lower circulating blood fats.[ScienceDaily]
  • The trial was funded by CRISPR Therapeutics AG and published in the New England Journal of Medicine.[ScienceDaily]

What remains uncertain

  • The long-term safety and efficacy profile of CTX310 remains unknown, requiring a planned 15-year monitoring period across a small sample size of 15 people.[ScienceDaily]

Sources

Outlet counts describe coverage, not independent confirmation. Reports may share a wire service or original source.