Rapid advances in life sciences are uncovering an increasing number of molecular targets relevant to human diseases. While genetic approaches are useful to evaluate the role of these molecular targets in cellular processes and human diseases, small molecules represent valuable orthogonal reagents permitting the interrogation of cellular processes and therapeutic development. Recent advances in laboratory automation, coupled with the ability to assemble and manage large libraries of small molecule compounds, have created unprecedented opportunities for new biomedical discoveries. It is now possible to screen hundreds of thousands of small molecules in assays involving purified proteins, cultured cell lines, or whole organisms.

Recognizing the importance of small molecules in basic and translational research, Indiana University School of Medicine has committed significant resources to establish the Chemical Genomics Core Facility in order to facilitate the identification of chemical tools to study biological pathways and the discovery of lead compounds for the development of novel therapeutics. This will help to put IU scientists at the forefront of this emerging area of research in chemical biology.

The mission of the Chemical Genomic Core is to provide IU investigators with cost-effective access to high throughput screening of structurally-diverse, drug-like small molecules in biological assays provided by the investigators. This would enable the investigators to discover small molecule tools for basic research, therapeutic development and diagnostic applications. Facility staff will work closely with each investigator through all stages of the screening process, providing an opportunity for IU students and fellows to gain experience and training in high throughput screening at the facility.

In the near future, the core facility will also develop capabilities for targeted chemical synthesis and chemi-informatics. Targeted chemical synthesis at an early stage after large scale screens is the major unmet need in current chemical biology efforts. The focused chemical synthesis programs will be dedicated to the optimization of lead compounds discovered from screening efforts. Optimization will focus on enhancement of therapeutic properties including potency and selectivity, as well as bioavailability and toxicity. A long term goal for the core is to facilitate the development of new therapeutic agents to treat a variety of human diseases. A more immediate goal is the development of chemical tools that are suitable for mechanistic studies of biological processes. Such tools can be very important in the development of therapeutic agents since they can be used to test the effects of altering biological processes in cells, which can lead to the identification of validated targets for drug development.