Beyond vaccines: Developing new therapeutics based on RNA and other oligonucleotides

The toolbox for drug discovery is continuously expanding beyond traditional small molecules, peptide- and protein-based therapies.

The development of the mRNA vaccines against COVID-19 are probably the most well-known example of RNA-based therapeutics. However, therapeutics based on RNA and other oligonucleotides has the potential for a much broader application than vaccines.

Therapeutics based on RNA and other oligonucleotides constitute a rapidly evolving area that may accelerate drug development processes from discovery to clinic.

Among other uses, RNA therapeutics has great potential within personalised medicine because the technology makes it possible to adjust a treatment to a patient’s genetic background.

To advance the translational potential of RNA-based therapeutics, this Challenge Programme 2023 theme will support projects developing fundamental research and thus new insights in this field.

The specific challenge of this theme is to support the basic science that facilitate development of RNA- and Oligonucleotide-based therapeutics beyond treatment of rare diseases and vaccine purposes. Such research is required to develop novel therapeutics for common clinical conditions, primarily cardiometabolic diseases, that is efficiently targeting the relevant tissue, cell types and subcellular localisation.

Some examples of research within the scope of this Challenge theme could be:

• Research on cellular targeting to optimise delivery into specific cell types, such as adipocytes, endothelia or skeletal muscles, and control of subcellular localisation. This may include studies on chemical modification of RNA and oligonucleotides, development of nanoparticles, and cellular adaptation to RNA/oligonucleotide exposure.
• Research that encourages development of novel RNA/oligonucleotide-based therapeutics and explores mechanisms to obtain increased transcription and protein expression.
• Research that pursues therapeutics in novel RNA/oligonucleotide-based modalities such as aptamers, circular RNA, miRNA, and lncRNA.
• Research in cellular adaptation to the RNA and oligonucleotides.

Find more information about the Challenge Programme theme RNA and Oligonucleotide-based Therapeutics, including the application process, here.