Prodrug-tethered lipid nanoparticles for synergistic messenger RNA cancer immunotherapy

Nat Nanotechnol. 2026 Mar;21(3):430-442. doi: 10.1038/s41565-025-02102-z. Epub 2026 Mar 18.

Abstract

Regulating T cell phenotypes between activation and exhaustion remains a significant challenge for messenger RNA-based cancer immunotherapy. A potential approach to improve anti-cancer T cell activity is to co-deliver interleukin-12 (IL-12), to stimulate effector T cells, and indoleamine 2,3-dioxygenase (IDO) inhibitor, to suppress T cell exhaustion. Here we design prodrug ionizable lipid nanoparticles (pLNPs), via a library of prodrug ionizable lipids (pILs), incorporating an intracellularly cleavable IDO inhibitor within the pIL structure and encapsulating IL-12 messenger RNA. The lead pIL shows enhanced mRNA transfection over a clinically utilized ionizable lipid, as well as strong immunomodulatory effects via release of the IDO inhibitor. In a subcutaneous colon cancer mouse model, pLNP drives complete regression of primary tumours by eliciting effector T cell infiltration while reducing exhaustion, induces a memory T cell response and stimulates a systemic immune response that allows for regression of distal tumours in this study. These results highlight the promise of pLNPs for small-molecule drug and mRNA combination cancer immunotherapy.

MeSH terms

  • Animals
  • Cell Line, Tumor
  • Colonic Neoplasms* / immunology
  • Colonic Neoplasms* / therapy
  • Female
  • Humans
  • Immunotherapy* / methods
  • Indoleamine-Pyrrole 2,3,-Dioxygenase / antagonists & inhibitors
  • Interleukin-12 / genetics
  • Lipids* / chemistry
  • Liposomes
  • Mice
  • Mice, Inbred C57BL
  • Nanoparticles* / chemistry
  • Neoplasms* / immunology
  • Neoplasms* / therapy
  • Prodrugs* / chemistry
  • Prodrugs* / pharmacology
  • RNA, Messenger* / genetics

Substances

  • Prodrugs
  • RNA, Messenger
  • Lipids
  • Interleukin-12
  • Lipid Nanoparticles
  • Indoleamine-Pyrrole 2,3,-Dioxygenase
  • Liposomes