Targeting RNA with small molecules

ongoing

RNA molecules play key roles in biological processes and diseases. An emerging avenue to develop innovative medicines is therefore to design small molecule drugs that bind to RNA and affect downstream biological functions. However, to advance this field of research, a new generation of scientists is needed that can further develop and ultimately master the challenging process of drug design applied to RNA. Therefore, the TargetRNA network aims to educate and nurture the next generation of scientists with cutting-edge interdisciplinary skills in the discovery and biological characterization of RNA modulators and thus generate fundamental knowledge on what drives affinity and specificity of small molecules binding to RNA. The objective of TargetRNA is to develop compounds with antimicrobial activity through their interaction with RNA targets, and to use these to selectively reshape the human gut microbiome, an area with vast implications for medicine. Training through research activities, spanning from computational and medicinal chemistry over structural and molecular biology to microbiology and in vivo models, will be supplemented by an innovative training programme in specific and transferable skills for interdisciplinary drug discovery strategies to target RNA, including FAIR data principles, open science practices as well as responsible development and sustainable implementation of new innovations. To achieve the ambitious research and training goals, the TargetRNA network unites leading scientists from 13 academic partners (8 beneficiaries, 5 associated) and 4 industry partners (1 beneficiary, 3 associated) based in 9 countries.