Abstract

Gene Silencing in HSPCs with an Apolipoprotein A1-based Nanodelivery Platform

Roy van der Meel, Assistant Professor, Department of Biomedical Engineering, , Eindhoven University of Technology

Nucleic acid therapeutics are revolutionizing healthcare via gene inhibition, addition, replacement or editing. However, nucleic acid-based drugs require chemical modifications and sophisticated nanotechnology to prevent their degradation, reduce immunostimulatory effects, and ensure intracellular delivery. Lipid nanoparticle (LNP) technology is the current gold standard platform that enabled the clinical translation of the first siRNA drug Onpattro and the COVID-19 mRNA vaccines. Nevertheless, current LNP systems are mostly suited for vaccine purposes following local administration or hepatic delivery following intravenous administration. To unleash RNA’s full therapeutic potential, we introduce modular nanoplatform technology for systemic nucleic acid delivery to immune cells in hematopoietic organs using apolipoproteins.