Hauke Cornils, Evotec

Phenotypic screening approaches provide the possibility to identify novel small molecules based on the desired cellular effects in an unbiased fashion. However, despite progress in the development and analysis of phenotypic screens, the identification of the molecular targets of screening ‚hits‘ provide a significant technical hurdle.

Target-based drug discovery on the other hand allows for High-Throughput Screening approaches but depend on the identification and validation of disease relevant targets.

Genetic screening approaches allow bridging phenotypic and target-based drug discovery approaches. Especially the CRISPR system with its potential to induce loss of functions mutations offers the possibility to identify and validate novel targets contributing to disease relevant phenotypes in a genome-wide fashion. Importantly, CRISPR technology can also be applied in advanced cell models such as primary cells or iPS derived systems. In this presentation, we are going to discuss an example of successful application of CRISPR in a high content-based whole-genome wide arrayed screen in primary cells.

The presented data shows that Evotec has integrated CRISPR based screening and target validation into its existing portfolio of target identification and validation platforms to further expand the space for target-based drug discovery.