The aim of this work package is to provide contribution to the CDR by design the Platform ‘Medicines discovery and early drug development’ that will cover all aspects of early-stage, preclinical drug discovery for pediatric therapeutics. It is well established that molecular changes that occur in children differ to the general population and additional considerations such as growth and development are important in the discovery campaign design. New emergent technologies such as use of pluripotent stem cell, 3D cell cultures, target validation, patient derived cell assays, micro-fluidics, high-throughput cell image analysis, are pillars of novel precision medicine and are timely that children can benefit from this new wave of molecular science.
It is thus important to focus on specific targets for pediatric indications that should be considered independent of the existing knowledge for adults; special attention in diseases such as cancer, rare pediatric diseases, asthma and inflammatory conditions, infections and sepsis. The proposed design will be focused on five topics, namely Informatics, Medicinal Chemistry, Chemical Biology, Molecular and Experimental Pharmacology and Immunology. This Platform will be set up in collaboration with the existing RIs and in particular EATRIS-ERIC.
The reference person for this WP is Giulia Chiaruttini ([email protected]).