The field of nanomedicine is moving from an age of renaissance towards industrial revolution, in part, due to the ability of lipid nanoparticles (LNP) to package and deliver antigenic mRNA against SARS-CoV2, leading to the development of a powerful vaccine against COVID. The next horizon for our field remains tissue/cell-specific mRNA delivery that can lead to treatment of several diseases. Our lab has worked extensively in LNP design, synthesis, structure, and its impact on intracellular delivery of mRNA. I will talk about our labs pursuit onto understanding the journey of an LNP-mRNA within the cellular interior. These fundamental insights led us to design nanoparticles that can deliver mRNA to different tissues for the treatment of cystic fibrosis, retinal degeneration, and as COVID-19 therapeutics. We have developed novel LNPs that target the lungs after inhalation or IV injection for gene delivery and editing. We have shown that novel peptide guided LNPs can deliver mRNA selectively in the retina of non-human primates. Evolution of a cell-selective carrier for mRNA delivery through approaches will be discussed. Such platform technologies with capability to precisely deliver cargo to manipulate cells, correct diseases, with minimal off-target effects, will transform modern medicine
