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CRISPR Therapeutics to Present Phase 1b Data on In Vivo Gene-Editing Therapy for Heart Disease

CRISPR Therapeutics

CRISPR Therapeutics announced that late-breaking clinical data from its Phase 1b trial of CTX310 will be presented at the American Heart Association Scientific Sessions 2026. CTX310 is an in vivo CRISPR/Cas9 therapy targeting ANGPTL3, a gene involved in cholesterol and triglyceride metabolism. The Phase 1b trial focuses on severe hypertriglyceridemia, and the data will be presented in an oral session on November 9, 2026, at 8:00 a.m. CST.

  • CTX310 uses CRISPR gene editing delivered directly into the body to silence ANGPTL3, a gene that drives high triglycerides and cardiovascular disease risk.
  • The oral presentation, the highest-priority format for new trial data, is titled 'CRISPR-Cas9 Gene Editing Therapy Targeting ANGPTL3 for Severe Hypertriglyceridemia: Results From Phase 1b Trial.'
  • CRISPR is advancing CTX310 for both severe hypertriglyceridemia and mixed dyslipidemia, indications with large patient populations.
  • The Phase 1b trial advances the program into patients with more severe disease, a step beyond Phase 1 safety data.
  • In vivo CRISPR therapies remain early-stage; human data presented at major cardiology meetings signals progress toward clinical validation.
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