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CRISPR Gene-Edited T Cells Show Early Promise in Advanced Colorectal Cancer Trial

University of Minnesota researchers report safety, scalability, and a complete remission in a first-in-human trial targeting the intracellular checkpoint gene CISH.

Overview

  • The phase 1 trial enrolled 12 patients with end-stage metastatic colorectal cancer, utilizing CRISPR/Cas9 to edit tumor-infiltrating lymphocytes (TILs).
  • Knocking out the intracellular checkpoint gene CISH enhanced T cell recognition and attack on tumors, a novel approach inaccessible to traditional therapies.
  • The gene-edited TIL infusions were well tolerated, with no serious adverse events linked to the gene-editing procedure.
  • Several patients experienced disease stabilization, and one achieved a complete response, with metastatic tumors disappearing and not recurring for over two years.
  • Efforts are now focused on reducing production costs, simplifying manufacturing processes, and expanding clinical evaluations to validate and optimize the therapy.