Optimization of Genome Editing Therapeutics for Alzheimer's Disease and Alzheimer's Disease-Related Dementias (AD/ADRD) (U01 - Clinical Trials Not Allowed)

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This Funding Opportunity Announcement (FOA) supports the optimization of promising genome editing-based therapeutic leads for Alzheimer's Disease and Alzheimer's Disease-Related Dementias (AD/ADRD), towards IND-enabling studies. Specifically, it supports the characterization and optimization of therapeutic lead(s) that show promise as potential genome editing therapeutics, as evidenced by convincing proof-of-concept studies in appropriate models. At the end of the funding period, successful projects will have optimized a genome editing therapeutic candidate with demonstrated bioactivity, manufacturability, biodistribution, in vivo efficacy, and/or target engagement (measurement of proximal downstream effects) and optimal dosing, combined with other properties consistent with the desired clinical application. This FOA is not specific for any one or group within the AD/ADRD spectrum of disorders. Disorders, and includes genome editing therapies for: Frontotemporal dementia (FTD), Lewy body dementias (LBD) (including Dementia with Lewy bodies (DLB), Parkinson Disease Dementia (PDD)), Vascular contributions to Cognitive Impairment and Dementia (VCID), Alzheimers Disease (AD) and Multiples Etiology Dementias (MED).

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