Overview
With mRNA-based vaccines, new therapies using nucleic acids as the payload have now cemented their place as a useful platform to treat disease. This has spurred the development of novel excipients that can deliver these nucleic acids to cells. However, the lipid nanoparticle (LNP) delivery system, as used in some of the COVID-19 vaccines, has some limitations, for example its liver tropism.
Together with Stanford University, Evonik is developing and commercializing a new concept for gene delivery based on a class of dynamic oligomeric cationic materials. These materials are designed to self-assemble with polyanionic nucleotide genes, to deliver these genes to cells, and to release the gene once inside the cell. This webinar will provide an overview of these novel materials – known as Charge-Altering Releasable Transporters (CARTs).
CARTs were developed at Stanford University by Professors Robert Waymouth, Paul Wender and Ronald Levy. These amphipathic oligomers operate through an unprecedented mechanism, serving initially as oligo (α-amino ester) cations that complex, protect and deliver mRNA, and then change physical properties through a degradative, charge-neutralizing intramolecular rearrangement, leading to intracellular release of functional mRNA and highly efficient protein expression, both in cell culture and in live mice.
As a participant in this webinar, you will learn about:
- Background and challenges in developing mRNA and nucleic acid therapeutics
- New catalysts and synthetic methods for the design of functional materials for gene delivery
- CART-mRNA delivery including cell and organ selectivity
- Vaccination strategies using CART-RNA for cancer and SARS-CoV-2
- Commercialization possibilities for CARTs
