CBE Seminar (Zoom): Lipid Nanoparticles for RNA Delivery - SARS-CoV-2 Vaccines, Chemistry and Beyond

Non-UCI people, please use this registration link: https://forms.gle/ZJDQ46zDzH3Y6qnz8 

Abstract: Messenger RNA (mRNA) therapeutics have been thrust into the limelight, thanks to the early, positive clinical trial news on a SARS-CoV2 vaccine from Pfizer/BioNTech and Moderna. These vaccines were made possible by a herculean effort to overcome the most significant barriers that have hindered translational efforts. Arguably, the largest challenge has been that RNA molecules do not readily enter their cellular targets within the body. This is because they are large (104-106 g/mol) and negatively charged; they do not have favorable biodistribution properties nor an ability to cross the cellular membrane of target cells. In response to these issues, industrial and academic laboratories, including my own, have created lipid nanoparticles that spontaneously package RNA and deliver the RNA to key cellular targets in vivo. Here, I will describe biodegradable, ionizable lipid-like materials called "lipidoids" that my lab has used to create RNA-loaded lipid nanoparticles that induce protein expression in mice. Lipidoids efficiently manipulate gene expression in a variety of biological systems, including the liver, lungs and immune cells. This talk will describe an especially potent lipid nanoparticle, its chemical characteristics that confer efficacy and potential applications. Together, these data advance our understanding of lipid nanoparticle chemistry and are expected to contribute to the successful formulation of future generations of mRNA therapies. 

Bio: Kathryn A. Whitehead is an associate professor and Dean’s Career Fellow in the Departments of Chemical Engineering and Biomedical Engineering (courtesy) at Carnegie Mellon University. Her lab develops drug delivery systems for RNA, proteins, and applications in maternal and infant health. She obtained bachelor and doctoral degrees in chemical engineering (University of Delaware, UC Santa Barbara) before an NIH Ruth L. Kirschstein Postdoctoral Fellowship at MIT. Professor Whitehead is the recipient of numerous awards, including the NIH Director’s New Innovator Award, the DARPA Director’s Fellowship and the ASEE Curtis W. McGraw Research Award. She has also received the Controlled Release Society’s Young Investigator Award and served on its Board of Directors. Professor Whitehead is an elected Fellow of the American Institute for Medical and Biological Engineering, was named a Pioneer on the MIT Technology Review’s Innovators Under 35 list and one of the Brilliant Ten by Popular Science. Most recently, she gave a TED Talk on the lipid nanoparticles (i.e., “fat balls”) used in the COVID-19 mRNA vaccines. Her publications have been cited over 8,000 times, and several of her patents have been licensed and sublicensed for reagent and therapeutic use. 

Host: Professor Herdeline Ardoña