Small molecule chemical compounds, which can be used to test or “probe” the effects of increasing or decreasing the activity of a biological target in cells or animals, are some of the most powerful tools for target validation, which is the process of demonstrating that engaging a target provides meaningful therapeutic benefit. Probes enable researchers to investigate protein and cell functions and biological processes. If appropriate, probes can be optimized to become potential drug candidates.
Generating these chemical probes requires specialized expertise and facilities, and NCATS has built world-leading collaborative services to meet these needs. The NCATS Chemical Genomics Center (NCGC), formerly known as the NIH Chemical Genomics Center, was created in 2008 as a comprehensive screening center in the Molecular Libraries Probe Production Centers Network, part of the NIH Common Fund’s Molecular Libraries Program. The original goals of the NCGC were to translate the discoveries of the Human Genome Project into biology and disease insights and ultimately new therapeutics through small molecule assay development, high-throughput screening, informatics and chemistry. Learn more about the goals of the NCGC.
NCGC staff collaborate with more than 200 investigators at NIH and in the academic, biopharmaceutical and nonprofit sectors to generate probes for studying a diverse cross-section of human biology, focusing specifically on new targets and untreatable diseases. NCATS’ probe development activities also include finding more efficient ways to make probes, using probes to understand diseases and validating targets to treat diseases. Learn more about the NCGC operational model.