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

The National Institutes of Health (NIH), through the National Institute of Neurological Disorders and Stroke (NINDS), has issued a forecasted opportunity titled "Genomic and Biologic Therapies for ADRD (U01 Clinical Trial Optional)." This funding opportunity reflects the NIH’s commitment to accelerating therapeutic development for Alzheimer’s Disease-Related Dementias (ADRD), a group of conditions that includes frontotemporal degeneration (FTD), Lewy body dementia (LBD), and vascular contributions to cognitive impairment and dementia (VCID). The initiative is designed to support translational research that advances genomic and biologic therapeutic candidates from early optimization through preclinical development and, where appropriate, first-in-human (FIH) clinical evaluation. Recognizing the transformative potential of genomic and biologic therapies, this program specifically targets projects that have already demonstrated compelling preclinical evidence and are ready for translational advancement. Eligible activities may include refining therapeutic design, improving manufacturability, conducting pharmacology and toxicology studies, evaluating biodistribution and in vivo efficacy, and compiling data necessary for regulatory submissions such as Investigational New Drug (IND) applications. Projects may also support early-phase clinical trials if warranted by the maturity of the therapeutic candidate. The scope of this funding opportunity is aligned with strategic priorities identified during recent ADRD Summits. These include the development of disease-modifying treatments and a focus on advancing therapies beyond traditional pharmacologic approaches. The NIH emphasizes the importance of overcoming challenges related to delivery, safety, and production scalability—key barriers that have historically limited the clinical applicability of genomic and biologic therapies in neurodegenerative diseases. The forecast indicates that approximately three awards will be made under this opportunity, with a total estimated program funding of $6,000,000. The expected post date for the official Notice of Funding Opportunity (NOFO) is July 1, 2026, and applications will be due by November 10, 2026. Award notifications and project start dates are both expected on July 1, 2027. While no cost sharing or matching is required, applicants are encouraged to build meaningful collaborations and prepare robust, responsive proposals. Eligibility for this opportunity is broad, including state and local governments, public and private institutions of higher education, nonprofits (with and without 501(c)(3) status), for-profit organizations, small businesses, federally recognized and non-federally recognized tribal governments and organizations, U.S. territories, and foreign institutions. This inclusive approach aims to encourage diverse perspectives and promote innovation in the development of novel ADRD therapies. Applications will be submitted through Grants.gov, and although specific application components are not yet listed, standard NIH cooperative agreement processes and requirements will likely apply. The NINDS ADRD Program should be contacted via email at [email protected] for questions or further information. This early forecast gives applicants ample time to plan and establish partnerships to prepare high-quality submissions once the full solicitation is released.