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Milestone 6.D

Identify, characterize, and complete early validation for at least 6 novel therapeutic targets for AD and 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.

Time Required

2014 to 2020

Research Implementation Area

Drug Development - Novel Targets

CADRO Category

C. Translational Research and Clinical Intervention

Activities, Funding Initiatives, and Resources

NIH Accelerating Medicines Partnership-AD – Target Discovery and Preclinical Va…
NIH RFA-AG-15-018: Immune and Inflammatory Mechanisms in Alzheimer’s Disease (R…
NIH RFA-AG-15-010: Interdisciplinary Research to Understand the Vascular Contri…
NIH RFA-AG-14-002: Optogenetic Tools for the Study of Neural Systems in Aging a…
NIH RFA-AG-13-013: Interdisciplinary Approach to Identification and Validation …

Funded Research Projects

IADRP – Category C. Translational Research and Clinical Interventions - 1. Drug…
IADRP – 2. Drug Development
NIH RFA-AG-15-018: Immune and Inflammatory Mechanisms in Alzheimer’s Disease (R…
NIH RFA-AG-15-010: Interdisciplinary Research to Understand the Vascular Contri…
NIH RFA-AG-14-002: Optogenetic Tools for the Study of Neural Systems in Aging a…
NIH RFA-AG-13-013: Interdisciplinary Approach to Identification and Validation …