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

National Institutes of Health

Summary

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).

Eligibility

Small businesses
Schools & universities
State governments
Nonprofits
Local governments
Tribal governments & organizations
For-profit organizations

Other Eligible ApplicantsEligible Agencies of the Federal Government;U.S. Territory or Possession;Faith-based or Community-based Organizations;Regional Organizations;Non-domestic (non-U.S.) Entities (Foreign Institutions).