mRNA as a therapeutic platform technology ideally suited for a truly individualized patient treatment
BioNTech RNA Pharmaceuticals GmbH, Mainz, firstname.lastname@example.org
Messenger (m)RNA is increasingly investigated as a platform technology for multiple therapeutic applications. We have developed an innovative mRNA-based cancer immunotherapy platform harboring proprietary chemical modifications and sequence elements that potentiate the bioavailability, immunogenicity and immunomodulatory properties of the mRNA.
With respect to manufacturing, mRNA has several advantages compared to other biopharmaceuticals. The manufacturing process is completely cell-free. This leads not only to lower cost-of-goods, but also impacts manufacturing time and safety of the product with respect to microbial or animal-derived impurities. One process can be essentially used to manufacture any RNA sequence, significantly shortening development time for a new project.
Neo-epitopes derived from tumor mutations are ideal targets for immunotherapy against cancer. As the vast majority of cancer mutations are unique to the individual patient, personalized approaches are needed. By combining sequencing of tumor samples and bioinformatic algorithms to identify potentially immunogenic epitopes defined by the individual mutations with our mRNA platform, which allows encoding of multiple epitopes on one mRNA molecule, we have implemented and verified this approach in a first-in-concept clinical trial. This has demonstrated that mRNA is a versatile platform technology especially applicable for manufacturing of individualized therapeutics.