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

The National Institutes of Neurological Disorders and Stroke (NINDS) is interested in supporting research projects proposing the optimization and translational development of genomic and biologic therapeutic candidates for Alzheimer’s Disease-Related Dementias (ADRD). Breakthroughs in genomic and biologic therapies are creating new opportunities to treat, prevent, and potentially modify the course of dementias; however, progress is limited by challenges in delivery, safety, manufacturability, and readiness for clinical testing. This program is intended to support projects across the translational pipeline, from optimization through IND development and early clinical evaluation, for genomic and biologic therapeutic candidates with compelling preclinical evidence. Eligible projects may include activities to refine therapeutic design and manufacturability; establish pharmacology, toxicology, biodistribution, and in vivo efficacy; to generate the full body of data required to support regulatory interactions and IND submissions, and, when appropriate, first-in-human (FIH) clinical studies. The program is aligned with priorities from the ADRD Summits, including acceleration of disease-modifying therapeutic development in frontotemporal degeneration (FTD), Lewy body dementia (LBD), and vascular contributions to cognitive impairment and dementia (VCID). Applications are not being solicited at this time. Notice is being provided to allow potential applicants sufficient time to develop meaningful collaborations and responsive projects.

Other Eligible ApplicantsIndian/Native American Tribal Governments (Other than Federally Recognized);Eligible 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).

The major elements in evaluating proposals include assessments of: (1) the scientific merit and general significance of the proposed study and its objectives; (2) the technical adequacy of the experimental design and approach; (3) the competency of the proposed investigator or group to successfully pursue the project; (4) the adequacy of the available and proposed facilities and resources; (5) the necessity of the budget components requested in relation to the proposed project; and (6) the relevance and importance to announced program objectives. The following criteria will be used in considering the scientific and technical merit of SBIR/STTR Phase I grant applications: (1) the soundness and technical merit of the proposed approach; (2) the qualifications of the proposed principal investigator, supporting staff, and consultants; (3) the technological innovation of the proposed research; (4) the potential of the proposed research for commercial application; (5) the appropriateness of the budget requested; (6) the adequacy and suitability of the facilities and research environment; and (7) where applicable, the adequacy of assurances detailing the proposed means for (a) safeguarding human or animal subjects, and/or (b) protecting against or minimizing any adverse effect on the environment. Phase II grant applications will be reviewed based upon the following criteria: (1) the degree to which the Phase I objectives were met and feasibility demonstrated; (2) the scientific...