The rapid acceleration of genomic and proteomic technologies has provided new insight on the molecular basis of disease. However, validation of therapeutic targets from this enormous collection of information has been limited by the slow pace of existing in vivo biological tools.
The discovery of RNA interference has revolutionized molecular biology. It has also become clear that the combination of modern drug delivery systems and RNA interference will similarly revolutionize drug therapy and the study of biology.
Our mission is to develop the center of research, education and innovation within Skolkovo Institute of Science and Technology (Skoltech) which will fulfill main aspirations of translational medicine. To achieve these goals we set up a multidisciplinary research facility which allows us to develop novel RNA delivery systems, to perform effective study and validation of long non-coding RNAs, proteins and signaling pathways as targets for the therapy by RNA interference and CRISPR/Cas9 technologies both in vitro and in vivo, to test new small molecules and therapeutic platforms for further translation into clinical studies. The development of translational medicine is impossible without deep understanding of regulatory pathways in individual cells and in a whole organism. This is especially needed in studies of long non-coding RNAs, epitranscriptomics and short peptide regulators. Study of novel regulatory mechanisms in pathology will provide us new targets for drug development in agreement with the Strategy of Science and Innovation approved by President of Russia.
These instruments allow us addressing crucial liver and neuropsychiatric disorders. Currently our main targets are hepatocellular carcinoma and non-alcoholic fatty liver disease diseases (delivery to hepatocytes, Kupffer cells and hepatic stellate cells) and Parkinson’s diseases. We are on the way to modulate immune diseases with RNA technologies (spleen macrophages and other myeloid cells). All these results could be translated into clinical studies in collaboration with International Medical Cluster.
In 2018 we are going move to East Ring Skoltech Campus and to start a new SPF animal house (3000 sq meters) to develop new strains of transgenic animal models for topical diseases by using RNAi and CRISPR/Cas9 technology. Then we plan to start a Contract Research Organization (in collaboration with Biomedical Cluster of Skolkovo Foundation) for preclinical drug testing that will be extremely competitive in Russia.