048 Chemically modified mRNA for therapeutic applications

Author: Jacek Jemielity

The Laboratory of Chemical Biology at Centre of Nw Technology University of Warsaw is interested in chemically modified nucleotides and nucleic acids (https://chembiobiochem.com). Due to the biological role of these molecules, their chemically modified analogs have tremendous applications in the development of molecular tools for research, diagnostic and therapeutic applications.

The team is particularly famous for modifying mRNA with a focus on improving its therapeutic properties. During the pandemic, mRNA technology had experienced its spectacular use in covidien vaccines, but its potential is much greater. For more than 20 years, Jemielity and his colleagues have been designing, synthesizing and studying the properties of nucleotide analogs of the 5′ end of mRNA (so-called cap analogs). Their inventions are licensed to leaders in the field. One of them, beta-S-ARCA, an invention that increases mRNA persistence and translation efficiency, has been licensed to BioNTech, which is using it in a total of 12 clinical trials on anti-cancer vaccines. The potential of mRNA is much broader, encompassing not only preventive antiviral vaccines or therapeutic cancer vaccines, but can also be used in protein replacement strategies, genome editing, or cell therapies.