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Cambridge MedChem Consulting

Target Validation

There has been much discussion about the attrition of drugs in development due to lack of efficacy in man and this in part can be due to poor target validation. That is proof that modulation of the identified target in a model system has the desired impact on biological activity and can be linked to therapeutic utility.

This is an absolutely critical step, almost everything else can be fixed.

For this reason two new resources seem particularly valuable.

The Centre for Therapeutic Target Validation platform ( brings together information on the relationships between potential drug targets and diseases. The core concept is to identify evidence of an association between a target and disease from various data types.

A target can be a protein, protein complex or RNA molecule, but we integrate evidence through the gene that codes for the target. In the same way, we describe diseases through a structure of relationships called the Experimental Factor Ontology (EFO) that allows us to bring together evidence across different but related diseases.The platform supports workflows starting from either a target or disease and presents the evidence for target – disease associations in a number of ways through association and evidence pages.

DisGeNET( is a discovery platform integrating information on gene-disease associations (GDAs) from several public data sources and the literature doi.

The current version contains (DisGeNET v3.0) contains 429111 associations, between 17181 genes and 14619 diseases, disorders and clinical or abnormal human phenotypes.