NIDDK’s Therapeutics Discovery Translational Pipeline

May 2019 Council

Lead Division/Office

DEM / DDN / KUH

Point(s) of Contact

Aaron Pawlyk, Ph.D.

Executive Summary

Although our understanding of biology and disease continues to increase, there are still numerous failures to advance efficacious and safe therapeutics, leaving significant unmet medical needs. To tap in to the deep expertise and creativity of its scientific community, the NIDDK established a pipeline for Translational Research for Therapeutic Discovery and Development, which has been successful in promoting the translation of basic science discoveries into knowledge and development of tools that can be utilized to provide strong support for later-phase drug discovery and development efforts for diseases relevant to NIDDK. To date, this Pipeline has resulted in 36 awards and has brought in over a dozen medicinal chemistry groups to NIDDK mission areas, funding an array of novel therapeutic validation projects.

Recent significant advances in genetics, physiology, and the pathogenesis of disease coupled with technological advances have provided a rich knowledge base and strong toolbox to identify and pursue new therapeutics with the goal of generating new molecular, microbial, and cellular therapies for the treatment of disease. Despite this, there are numerous failures of potential therapeutics for reasons related to human safety or efficacy. It is necessary to develop better strategies to validate and prioritize therapeutic leads based on their likelihood of success to safely improve upon current standard of clinical care. Analyses of patent literature have indicated that academic laboratories and small biotechnology companies contribute significantly to novel targets and first-in-class drugs, highlighting the important role these sectors play. Unlike many other large institutes, the NIDDK has not historically had standing study sections for or a large footprint in therapeutics discovery.