Genomic and Biologic Therapies for ADRD (U01 Clinical Trial Optional)

This Notice of Funding Opportunity (NOFO) supports the translation of genome editing and other biologic therapies for Alzheimer's Disease-Related Dementias (ADRD). The program spans the full translational pipeline, from therapeutic lead optimization and drug product development through IND-enabling studies and early clinical evaluation. Supported activities may include optimization of editing constructs and delivery systems; assessment of bioactivity, target engagement, durability, pharmacokinetics, and bioavailability; demonstration of in vivo efficacy; execution of safety pharmacology and toxicology studies; and development of scalable manufacturing processes, formulation strategies, and quality attributes aligned with regulatory expectations. ADRD include Frontotemporal Dementia (FTD); Lewy Body Dementias (LBD), including Dementia with Lewy Bodies (DLB) and Parkinson's Disease Dementia (PDD); Vascular Contributions to Cognitive Impairment and Dementia (VCID); and Multiple Etiology Dementias (MED).

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