Target validation

Objectives

Target validation aims to prove that a target gene and its products are directly involved in a disease process and are appropriate targets for the development of new therapeutic drugs.
Drug target validation has became a challenge for drug discovery as it has become crucial to pinpoint new targets that are most likely to have a pharmaceutical value in order to optimize the drug discovery pipeline as well as to shorten the time to market.

Description

Thanks to its unparalleled expertise in molecular biology and High Content Screening, Fluofarma offers high-throughput functional target validation services.
The combination between the silencing the expression of specific genes by siRNAs and the sensibility of our cell-based assays provides a solution to the bottleneck of the post genomic era. I.e. how to identify the best candidate among hundreds of possibilities. Thus, our approach allows validating the function of a gene at the same rate than the discovery of potential candidate by gene profiling techniques.

Example of target validation

In this exemple, we assessed in a single experiment the role of 60 different human kinases in cancer development by measuring in a multiplexed assay both apoptosis and cell proliferation in the presence of siRNAs which specifically target each kinase.

This method allows the identification of targets (in pink) whose inhibition induces the cell apoptosis or the inhibition of cell proliferation.

 

Plotting those data in a bidimensional space allows selecting among the candidate the most suitable for the desired therapeutic application. For example a pure antiproliferative agent (green circle), a strong pro-apoptotic (blue circle), or a target that is strongly involved both in apoptosis and in cell proliferation (purple circle).