RNA4T: RNA for therapy

RNA-based drugs could overcome the limits of conventional small-molecule drugs modulating protein functions by targeting their active sites, which are restricted by the druggability of protein targets, which harbors less than 30% of the 2500 disease-related proteins. RNA therapies might have a number of other inherent advantages, such as low immunogenicity, rapid design and adaptivity, fast and efficient production, long-term effect, and usefulness for rare diseases. Most RNA therapeutics rely on base-pairing of RNA analogs in various antisense mechanisms or, alternatively, the mRNA-based drugs are transiently translated. However, the full potential of this modern and safe technology remains unexplored as there are less than a dozen FDA-approved RNA therapeutics.

The project brings together 14 research groups working on several disease models and mastering different methodological approaches in organic chemistry, biochemistry and cell and molecular biology with a common denominator in RNA biology. It thus gives us a unique opportunity to conduct highly interdisciplinary research that will strengthen our efforts to firmly establish the Czech Republic on the map of RNA therapeutics development and thus significantly increase its scientific prestige.

Project RNA for therapy is funded by Programme Johannes Amos Comenius managed by the Ministry of Education, Youth and Sports of the Czech Republic.

Registration number: CZ.02.01.01/00/22_008/0004575

Implementation date: 1. 1. 2024 – 31. 10. 2028

Budget: CZK 499 640 882, 63

Main Beneficiary: Institute of Organic Chemistry and Biochemistry of CAS

Scientific manager: Prof. Ing. Michal Hocek, CSc. DSc.

Partners:

• Institute of Molecular Genetics of the CAS
• Masaryk University (CEITEC)
• Biology Centre of the CAS
• Institute of Microbiology of the CAS
• Charles University (Faculty of Science)