The overall goal of the project is to build an RNA-focused innovation ecosystem where excellent labs will realize strong research programs in RNA biology and RNA therapeutics in cooperation with industrial partners and qualified intellectual property and technology transfer management to make the Czech Republic a recognized player in RNA therapeutics and strengthen its competitiveness in the pharmaceutical industry.
- To exploit medically relevant aspects of RNA biology through research on disease-related RNA metabolism and by combining excellent expertise in nucleic acid chemistry and RNA biology (including biochemistry, molecular, structural and cell biology).
- To have a competitive and unique research programme with strong innovation potential in several directions:
- unique synthesis of modified RNA for therapeutic purposes based on unique nucleotide derivatives and customized enzymatic synthesis,
- testing the application potential of RNA analogues and modified oligonucleotides along the entire gene expression pathway: during transcription, post-transcriptional processing and translation,
- deciphering the underlying role of RNA metabolism in specific neurodegenerative disorders,
- identification of novel therapeutic targets based on RNA metabolism in models of the human pathogens trypanosomatid and Bordetella pertussis for the development of new therapeutic strategies,
- investigation of mammalian siRNA biogenesis and formation of RNAi effector complexes with implications for RNAi-based therapeutics,
- development of new strategies for the regulation of prokaryotic and eukaryotic translation to be utilized in RNA-based therapies.
- To bring original and critical know-how to Czech research and development through:
- establishment of a functional collaboration in the field of RNA therapy with the Abisch-Frenkel Center for RNA Therapy of the Weizmann Institute, an Israeli research powerhouse,
- collaboration with companies, especially Alnylam, a leader in RNAi-based therapies.
- To provide excellent training to graduate students and early career scientists participating in this project with the focus on the field of RNA therapeutics.
- To improve undergraduate and postgraduate education programs in the field of RNA biology at four Czech universities (Charles University, University of Chemistry and Technology, Masaryk University, University of South Bohemia).