Journal of Biomedical Science (Sep 2024)

Current landscape of mRNA technologies and delivery systems for new modality therapeutics

  • Ruei-Min Lu,
  • Hsiang-En Hsu,
  • Ser John Lynon P. Perez,
  • Monika Kumari,
  • Guan-Hong Chen,
  • Ming-Hsiang Hong,
  • Yin-Shiou Lin,
  • Ching-Hang Liu,
  • Shih-Han Ko,
  • Christian Angelo P. Concio,
  • Yi-Jen Su,
  • Yi-Han Chang,
  • Wen-Shan Li,
  • Han-Chung Wu

DOI
https://doi.org/10.1186/s12929-024-01080-z
Journal volume & issue
Vol. 31, no. 1
pp. 1 – 36

Abstract

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Abstract Realizing the immense clinical potential of mRNA-based drugs will require continued development of methods to safely deliver the bioactive agents with high efficiency and without triggering side effects. In this regard, lipid nanoparticles have been successfully utilized to improve mRNA delivery and protect the cargo from extracellular degradation. Encapsulation in lipid nanoparticles was an essential factor in the successful clinical application of mRNA vaccines, which conclusively demonstrated the technology's potential to yield approved medicines. In this review, we begin by describing current advances in mRNA modifications, design of novel lipids and development of lipid nanoparticle components for mRNA-based drugs. Then, we summarize key points pertaining to preclinical and clinical development of mRNA therapeutics. Finally, we cover topics related to targeted delivery systems, including endosomal escape and targeting of immune cells, tumors and organs for use with mRNA vaccines and new treatment modalities for human diseases.

Keywords