FAP-Synergistic Organ-Targeted mRNA-LNP for Overcoming Delivery Barriers in Hepatic and Pulmonary Fibrosis

J Am Chem Soc. 2026 Mar 11;148(9):9309-9327. doi: 10.1021/jacs.5c16886. Epub 2026 Feb 23.

Abstract

The inefficient delivery of lipid nanoparticles (LNPs)-encapsulated mRNA to activated fibroblasts in fibrotic tissues remains a major challenge for antifibrotic therapy. To overcome this, we develop a fibrosis organ and cell unified-targeting system (FOCUS) for mRNA delivery with LNPs. FOCUS LNPs combines an ionizable lipid library (synthesized via Ugi multicomponent reactions) with fibroblast activation protein-α (FAP)-targeting lipid-like ligands. These LNPs achieve ∼3-fold and ∼12-fold greater mRNA expression in fibrotic liver and lung, along with ∼2-5-fold higher fibroblast distribution─unattainable by organ- or ligand-only targeting. As proof of concept, Pentraxin-2 mRNA (mPTX-2)-loaded FOCUS LNPs drive localized PTX-2 production in murine models of pulmonary and hepatic fibrosis, reducing collagen deposition by 60-80% while avoiding systemic side effects like impaired wound healing. Mechanistically, mPTX-2 FOCUS LNPs suppress monocyte-to-fibrocyte differentiation, M2 macrophage polarization, and fibroblast activation. This work establishes a precision mRNA delivery platform for fibrotic diseases, offering a safer and more effective therapeutic strategy.

MeSH terms

  • Animals
  • Humans
  • Lipids* / chemistry
  • Liver Cirrhosis* / drug therapy
  • Liver Cirrhosis* / metabolism
  • Liver Cirrhosis* / pathology
  • Membrane Proteins* / metabolism
  • Mice
  • Mice, Inbred C57BL
  • Nanoparticles* / chemistry
  • Pulmonary Fibrosis* / drug therapy
  • Pulmonary Fibrosis* / metabolism
  • Pulmonary Fibrosis* / pathology
  • RNA, Messenger* / administration & dosage
  • RNA, Messenger* / chemistry
  • RNA, Messenger* / genetics
  • RNA, Messenger* / metabolism

Substances

  • RNA, Messenger
  • Membrane Proteins
  • Lipids