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Illuminating the Druggable Genome

Illuminating the Druggable Genome (IDG)

Results from the Human Genome Project revealed that the human genome contains approximately 20,000 genes. A gene contains (encodes) the information that each cell uses to make (express) a protein, which is essential for the body to function properly. Abnormal protein expression is associated with many human diseases, which makes proteins key targets for therapeutic agents.

Approximately 3,000 genes are considered part of the “druggable genome,” a set of genes encoding proteins that scientists can or predict they can modulate using experimental small molecule compounds. Yet the existing clinical pharmacopeia is represented by only a few hundred targets, leaving a huge swath of biology that remains unexploited.  Therefore, a large number of proteins remain for scientists to explore as potential therapeutic targets. Much of the druggable genome encodes three key protein families: non-olfactory G-protein-coupled receptors, ion channels and protein kinases. Researchers lack crucial knowledge about the function of many proteins from these families and their roles in health and disease. Better understanding of how these proteins work could shed light on new avenues of investigation for basic science and therapeutic discovery.

New Area of Discovery

To improve scientific understanding of the four understudied protein families, NIH launched an effort called Illuminating the Druggable Genome (IDG) in 2013, with the first awards made in 2014. The IDG was originally funded as a three-year pilot program sponsored by the NIH Common Fund and designed to create a centralized information repository and develop new technology platforms to study the protein families.  Now, the current implementation phase of the IDG Program aims to build on the knowledge and tools developed during the pilot phase and disseminate these IDG-generated resources to the greater scientific community. 

NCATS Director Christopher P. Austin, M.D., co-chairs NIH’s IDG Working Group with Griffin P. Rodgers, M.D., M.A.C.P., director of the National Institute of Diabetes and Digestive and Kidney Diseases. The group includes representatives from the National Institute of Mental Health, the National Cancer Institute, the National Institute of Neurological Disorders and Stroke, and other Institutes and offices at NIH.

By expanding the potential therapeutic space through the IDG program, NIH is clearing a path for more efficient disease-related research and more effective treatments for patients.

For more information about IDG, visit the NIH Common Fund website.

Funded Research

 Knowledge Management Center for Illuminating the Druggable Genome
(U24) RFA-RM-16-024

PI Name Institution Name Title
Tudor Oprea University of New Mexico Health Science Center Knowledge Management Center for Illuminating the Druggable Genome (Center Award)

Avi Ma'ayan

Icahn School of Medicine at Mount Sinai Knowledge Management Center for Illuminating the Druggable Genome (Component Award)


 Resource Dissemination and Outreach Center for Illuminating the Druggable Genome
(U24) RFA-RM-16-025

PI Name Institution Name Title

Stephan Schurer, Tudor Oprea and Larry Sklar

University of Miami School of Medicine Illuminating the Druggable Genome Resource Dissemination and Outreach Center (IDG-RDOC)


Data and Resource Generation Centers for Illuminating the Druggable Genome
(U24) RFA-RM-16-026

PI Name Institution Name Title
Gary L. Johnson University of North Carolina Chapel Hill Illuminating Function of the Understudied Druggable Kinome

Michael T. McManus and Lily Jan

University of California, San Francisco Illuminating Druggable Dark Matter

Bryan L. Roth and Brian K. Shoichet

University of North Carolina Chapel Hill Illuminating the Druggable GPCR-ome