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Drug Development/Novel Targets: Target validation (Milestone 6.C)


Timeline Start - End

2014 - 2020

Research Implementation Area

Drug Development - Novel Targets

Identify, characterize, and complete early validation for at least 6 novel therapeutic targets for AD and AD-related dementias (a minimum of 3 targets for pre-symptomatic and early stage disease and a minimum of 3 for advanced disease). These efforts should include therapeutic targets for the neuropsychiatric and behavioral disturbances in AD and ADRDs.

Success Criteria

  • Validation based on availability of the following for each novel target: a systems-level understanding of the gene, protein and metabolic networks within which they operate, one or more cell based/animal models that are freely available to the research community, a quantitative assessment of the integrative response to the modulation of the target in one or more model organisms, and identification of pharmacodynamic biomarker(s) for target engagement.

Summary of Key Accomplishments

Through the AMP®AD Target Discovery Program NIA has supported the discovery of over 550 novel candidate targets for AD. More than twenty of these candidate targets have been characterized in cell and animal models. The targets delivered by the AMP AD program along with the supporting data have been made available to researchers in academia and in the biotech/pharmaceutical industry through centralized data infrastructure. Over twelve AMP AD targets are being pursued by industry and in academic labs for new drug discovery efforts targeting multiple aspects of the disease process, such as neuroinflammation, bioenergetic failure and synaptic pathology. The AMP AD data resources have been used by more than 3000 researchers from the US and internationally to advance basic research and therapy development for AD/ADRD.

The key accomplishments summary is current as of March 2022. 

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