MIT.nano Seminar Series

Kathryn Whitehead
Associate Professor and Dean’s Career Fellow
Chemical Engineering and Biomedical Engineering
Carnegie Mellon University

Monday, December 6, 2021
3:00 PM – 4:00 PM EST
Zoom webinar

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 Whitehead's, have created lipid nanoparticles that spontaneously package RNA and deliver the RNA to key cellular targets in vivo. Here, Whitehead will describe biodegradable, ionizable lipid-like materials called "lipidoids" that her 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, the lungs, and immune cells.

This talk will describe an especially potent lipid nanoparticle, its chemical characteristics that confer efficacy, and potential applications. Together, this data advances our understanding of lipid nanoparticle chemistry and are expected to contribute to the successful formulation of future generations of mRNA therapies