Abstract

Nucleic Acid Delivery with Biological and Synthetic Lipid Nanoparticles

Raymond Schiffelers, Professor of Nanomedicine, University Medical Center Utrecht

Nucleic acid nanomedicines gain momentum. Starting with Onpattro, delivering siRNA to the liver and followed by the local injection of COVID mRNA vaccines, we currently witness an avalanche of new therapeutic applications. Following the same lipid nanoparticle recipe as Onpattro and the vaccines, enzyme replacement therapy for rare metabolic disorders in the liver and CRISPR mRNA/sgRNA combinations for hepatic gene editing are being explored clinically, amongst many others. For other applications in new tissues, this recipe needs to be tweaked. In this lecture, applications to spleen targeting, heart delivery and immune cell targeting will be discussed, and biological and synthetic systems are compared.