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Gene Delivery and Gene Editing (Focus Group - GDGE)

(197) Development of ionizable lipids for gene delivery to the lung using an Ugi four component reaction

Introduction:

Lipid nanoparticles (LNPs) have significantly advanced nucleic acid delivery, especially due to their pivotal role in the development of the COVID-19 vaccine. However, extrahepatic delivery after intravenous delivery remains a challenge. We developed a library of ionizable lipids named Thor lipids, utilizing the Ugi four-component reaction (U4CR), which exhibited high efficiency and specificity in delivering mRNA payloads to the lungs upon intravenous administration.

Learning Objectives:

  • Learn lipid structures' influence on overcoming liver tropism and transfection efficiency.
  • Understand combinatorial chemistry for the rapid development of novel lipids for pulmonary delivery
  • Analyze CRISPR-Cas9 gene editing outcomes in lung endothelial cells