Therapeutics Development Core (Core B)

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PROJECT SUMMARY TO THERAPEUTICS DEVELOPMENT CORE The goal of the Therapeutics Development Core (Core B) is to establish an efficient platform for supporting the development and validation of a pipeline of chemical, biological, and cellular therapeutics for the treatment of a wide spectrum of kidney diseases. Northwestern University (NU) has been at the forefront of drug discovery for a wide range of diseases, and state-of-the-art infrastructures have been built that collectively have significantly expanded our therapeutics armamentarium for many diseases including hematological, oncological, neurological, and rheumatological conditions. However, they have not been prioritized and tailored to kidney-specific therapeutics development. We propose to fill this gap by establishing a Therapeutics Development Core within the NU-GoKIDNEY Center. The Therapeutics Development Core will function as an efficient translational conduit between the Preclinical Models Core (Core A) and the Clinical and Translational Core (Core C). This Core proposes to provide a “one-stop” shop for concierge services for access and expert guidance to kidney therapeutics development, with the ultimate goal of clinical translation. It will be constructed to provide centralized consultation services for developing novel kidney disease-specific therapeutics to the NU-GoKIDNEY Center investigators for: (1) designing and testing experimental biological and small molecule therapeutics; (2) designing and testing cellular therapeutics; and (3) designing and testing nanotechnology-based precision targeting of therapeutic “payloads” to the desired cells and tissues. Core B will implement a Ph.D.-level Navigator system for maximizing service efficiency and promoting innovative collaborations. Core B will also use the soon-to-be-launched nephro-HUB to expand the core user base, track core services, enhance user interactions and promote user collaborations. Core B Directors and Navigators collectively possess extensive expertise encompassing novel cellular therapeutics development, virtual drug and biologics design, high-throughput screening platforms for efficiency determination, and nanotechnology-based precision drug targeting. Core B will formalize and capture this collective expertise under one roof, and provide investigators of the NU-GoKIDNEY Center with superior customer service combined with expert guidance in choosing the most efficient route for novel therapeutics development. By providing these core services, we anticipate that the Therapeutics Development Core will: (1) accelerate kidney therapeutics development by providing target-specific consultations and navigation through the processes of therapeutics design, development, validation and production; (2) elevate kidney disease- specific therapeutics development to the forefront of existing cutting-edge expertise and state-of-the-art infrastructures in therapeutics development at Northwestern; (3) catalyze and synergize innovative collaborations within a dynamic network of NU-GoKIDNEY Center investigators via a common platform provided by the Therapeutics Development Core.