Orphan target

As mentioned above, a differentiating peculiarity of this type of library is that it is constituted by compounds that have already been safely given to humans. Thus, if a compound were to hif with sufficient potency on an orphan target, there is a high chance that it could rapidly be tested in patients for Proof of Principle. This possibility represents another advantage of the SOSA approach. 

It is one thing to have millions of individnal small molecules at one s disposal, but quite another to select the compounds yon want from this growing pool of diversity The first question to be answered is, of conrse, do we need to select compounds at all , because the answer is not always yes, we do Very often a random selection will perform as well as any pre-selected set, particularly when assaying orphan targets or those for which little or no information is available regarding endogenous ligands or 3D stmcmres. 

Wassermann, A. M., Geppert, H., and Bajorath, J. 2009. Ligand prediction for orphan targets using support vector machines and various target-hgand kernels is dominated by nearest neighbor effects. J. Chem. Inf. Model. 49 2155-2167. 

Tang Herbal, 147 Target-based drug discovery biological targets, 180-184 chemical end point in, 180 chemical tools, 178-179 definition of, 5 description of, 175-177 linear approach, 176 orphan receptors, 180 preclinical process in, 176-177 random variation in gene expression, 178... 

The emphasis of the program is to support projects that complement, rather than compete with the private sector. Some examples are projects related to high-risk and novel targets, orphan diseases, and areas in which the monetary return may not be immediately clear. 

Wilson S, Bergsma DJ Orphan G-pro-tein-coupled receptors novel drug targets for the pharmaceutical industry. Drug Des Discov 2000 17 105-114. 

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