Therapeutic mRNA delivery with targeted lipid nanoparticles: next-generation transformative medicines

Cell & Gene Therapy Insights 2022; 8(4), 219–230

DOI: 10.18609/cgti.2022.278

Published: 27 March 2022
Umar Iqbal, Jagdeep K Sandhu

Messenger RNA (mRNA) has recently emerged as a new class of genetic drug for the prevention and treatment of various diseases. The rapid development and clinical deployment of COVID-19 vaccines worldwide has highlighted the potential of mRNA-based technologies as useful tools for the treatment of emerging infections. The clinical translation of mRNA therapeutics has been enabled due to the recent advances in drug delivery systems, including encapsulation of mRNA in lipid nanoparticles (LNPs) and improved intracellular delivery strategies. Therapeutic mRNA can also be leveraged for the treatment of genetic disorders, rare diseases and even cancer. However, broad application of therapeutic mRNA is limited due to its preferential accumulation in the liver. In this article we discuss strategies that can be employed to direct LNPs away from the liver and precisely deliver therapeutic mRNA to target cells of interest. The goal of delivering therapeutic mRNA in vivo represents a significant opportunity and a future of many new possibilities.