DRUGS4RARE: Drug Discovery Platform for Rare Diseases
Jordi Quintana, Director of Business Development and Innovation, Parc Cientific De Barcelona
We have developed a platform for Drug Discovery for Rare Diseases, Drugs4Rare, with three main actions: a) to gather information on the chemical structures, biological targets, and orphan diseases, associated to chemical compounds of therapeutic interest; b) to identify the potential protein targets for compounds by means of state-of-the-art in silico target profiling approaches; and c) to validate experimentally the predicted chemico-biological interactions. The results of these three consecutive actions are deployed in an integrated chemical biology annotated Drugs4Rare database, which contains key virtual and experimental information in the drug discovery process for new orphan drugs for rare diseases, or for repurposing of therapeutically active compounds towards rare diseases. The Drugs4Rare Drug Discovery pilot platform has been developed analyzing 49 compounds approved in Europe for the treatment of certain rare diseases. We have gathered information on these compounds and associated rare diseases from the Orphanet database (www.orpha.net), and additional information on their chemical structures, biological targets, and mechanisms of action in public (Pubchem, ChEMBL) and private access (SciFinder, Integrity) databases. Then we used the virtual polypharmacology predictive platform developed in the Chemogenomics Laboratory at IMIM, to generate a profiling of the 49 compounds in front of around 5000 biological targets.
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