Rewiring endogenous genes in CAR T cells for tumour-restricted payload delivery

Nature. 2025 Aug;644(8075):241-251. doi: 10.1038/s41586-025-09212-7. Epub 2025 Jul 2.

Abstract

The efficacy of chimeric antigen receptor (CAR) T cell therapy in solid tumours is limited by immunosuppression and antigen heterogeneity1-3. To overcome these barriers, 'armoured' CAR T cells, which secrete proinflammatory cytokines, have been developed4. However, their clinical application has been limited because of toxicity related to peripheral expression of the armouring transgene5. Here, we have developed a CRISPR knock-in strategy that leverages the regulatory mechanisms of endogenous genes to drive transgene expression in a tumour-localized manner. By screening endogenous genes with tumour-restricted expression, we have identified the NR4A2 and RGS16 promoters as promising candidates to support the delivery of cytokines such as IL-12 and IL-2 directly to the tumour site, leading to enhanced antitumour efficacy and long-term survival of mice in both syngeneic and xenogeneic models. This effect was concomitant with improved CAR T cell polyfunctionality, activation of endogenous antitumour immunity and a favourable safety profile, and was applicable in CAR T cells from patients.

MeSH terms

  • Animals
  • CRISPR-Cas Systems / genetics
  • Cell Line, Tumor
  • Female
  • Gene Knock-In Techniques
  • Humans
  • Immunotherapy, Adoptive* / methods
  • Interleukin-12 / genetics
  • Interleukin-12 / immunology
  • Interleukin-12 / metabolism
  • Interleukin-2 / administration & dosage
  • Interleukin-2 / genetics
  • Interleukin-2 / immunology
  • Interleukin-2 / metabolism
  • Male
  • Mice
  • Neoplasms* / genetics
  • Neoplasms* / immunology
  • Neoplasms* / pathology
  • Neoplasms* / therapy
  • Promoter Regions, Genetic / genetics
  • Receptors, Chimeric Antigen* / genetics
  • Receptors, Chimeric Antigen* / immunology
  • Receptors, Chimeric Antigen* / metabolism
  • T-Lymphocytes* / immunology
  • T-Lymphocytes* / metabolism
  • Transgenes / genetics

Substances

  • Receptors, Chimeric Antigen
  • Interleukin-2
  • Interleukin-12