A target is a cellular structure that is involved at he molecular-level of a specific disease and can be modulated by a therapeutic. It could be an enzyme, receptor, nucleic acid, hormone, ion channel, membrane protein, etc. There are several ways to identify a ‘druggable’ target implicated in the disease process or generation of symptoms. Here you will find information about methods, tools and strategies such as: in silico screening, next generation screening, phenotypic screening and structural analysis.
Once identified, the next step is to demonstrate that drug effects on the target can provide a therapeutic benefit with an acceptable safety window through a multifunctional process using validation techniques in disease models, in vitro tools and in vivo models.