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Translational Capabilities: Target enabling tools (Milestone 4.O)


Timeline Start - End

2018 - 2022

Research Implementation Area

Translational Tools, Infrastructure, and Capabilities

De-risk novel candidate targets by supporting the development of high quality, open source, target enabling tools that can serve as starting points for drug discovery campaigns or as research tools to understand the biology of “dark targets”.

Success Criteria

  • Enable the development of target enabling packages (TEPs) for up to 100 novel, disease-relevant, candidate targets derived via unbiased, data-driven approaches.

  • These tools should include: purified target proteins/crystal structures, biochemical assays suitable for functional characterization and for compound screens; validated antibodies; cell-based assays for target modulation; potent and specific small molecules or biologics. 

  • Ensure that all data and reagents are made available to the wide community of researchers with no restriction on use.

Summary of Key Accomplishments

To diversify and re-invigorate the therapeutic pipeline for AD, NIA established the TREAT-AD Centers. The two centers are developing target enabling packages, a set of research tools necessary for studying the biology of novel targets and as starting points for drug discovery, for over 80 novel targets discovered through AMP®AD and other target discovery programs. All data and target enabling packages developed by the TREAT-AD centers will be made available to researchers from academia and biotech/industry without restriction. The first set of 20 target enabling packages will be released at the end of 2022. In addition, the TREAT-AD centers will initiate drug discovery efforts to develop preclinical drug candidates for a subset of high priority novel targets. Two novel neuroimmune targets (INPP5D and PLCG2) have already been advanced into drug discovery.

The key accomplishments summary is current as of March 2022. 

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