Optimization of Genome Editing Therapeutics for Alzheimer's Disease-Related Dementias (ADRD) (U01 - Clinical Trials Not Allowed) | RFA NS 24 037

Opportunity Information: Apply for RFA NS 24 037

The NIH funding opportunity titled "Optimization of Genome Editing Therapeutics for Alzheimer's Disease-Related Dementias (ADRD) (U01 - Clinical Trials Not Allowed)" (Funding Opportunity Number RFA-NS-24-037) is a discretionary, cooperative agreement program designed to move promising genome editing therapeutic leads closer to the point where they can support IND-enabling work. The core idea is not to fund early discovery from scratch, but to take genome editing approaches that already have convincing proof-of-concept data in appropriate models and then do the practical, translational optimization needed to make them realistic drug candidates.

The program focuses on characterizing and improving one or more genome editing-based therapeutic leads so that, by the end of the award, the project has produced an optimized candidate with properties that look more like a development-ready therapy. NIH emphasizes outcomes such as demonstrated bioactivity, a plausible manufacturing path (manufacturability), acceptable biodistribution (getting to the right tissues and cell types), in vivo efficacy and/or solid target engagement data (clear measurement of proximal downstream biological effects that show the edit is doing what it is supposed to do), and an optimized dosing strategy. In other words, the work should reduce risk and answer the kinds of questions that typically block a genome editing concept from advancing toward regulatory studies, without actually running a clinical trial under this award.

The NOFO is intentionally broad across the ADRD landscape rather than being restricted to a single diagnosis. Applications can target genome editing therapeutic strategies relevant to multiple ADRD categories, including frontotemporal dementia (FTD), Lewy body dementias (LBD) such as dementia with Lewy bodies (DLB) and Parkinson disease dementia (PDD), vascular contributions to cognitive impairment and dementia (VCID), and multiple etiology dementias (MED). This broad scope signals that NIH is prioritizing platform and target opportunities wherever the biology supports a credible genome editing therapeutic rationale, as long as the project is positioned at the optimization stage and is aimed at advancing toward IND-enabling readiness.

Mechanistically, the award uses the U01 cooperative agreement format, which usually means substantial NIH program involvement compared with a standard research grant. While the NOFO summary does not list specific milestone language, the intent is consistent with milestone-driven translational development: measurable progress toward a defined optimized candidate rather than open-ended basic research. Clinical trials are explicitly not allowed under this opportunity, so the funded work is expected to remain in the preclinical optimization space.

Eligibility is broad and includes many U.S.-based organization types: state, county, city, township, and special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; federally recognized tribal governments; certain tribal organizations; public housing authorities/Indian housing authorities; nonprofits with and without 501(c)(3) status (outside of higher education); for-profit organizations other than small businesses; and small businesses. The NOFO also highlights additional eligible applicant categories such as Alaska Native and Native Hawaiian Serving Institutions, Asian American Native American Pacific Islander Serving Institutions (AANAPISI), Hispanic-serving Institutions, Historically Black Colleges and Universities (HBCUs), Tribally Controlled Colleges and Universities (TCCUs), faith-based or community-based organizations, regional organizations, eligible federal agencies, and U.S. territories or possessions. Foreign organizations are not eligible to apply, and non-U.S. components of U.S. organizations are not eligible; however, foreign components as defined under the NIH Grants Policy Statement are allowed, which typically means limited, well-justified work performed outside the U.S. as part of an otherwise eligible U.S. applicant organization.

Key administrative details in the source information include the sponsoring agency (National Institutes of Health), activity area (health), and CFDA numbers 93.853 and 93.866. The original application due date is listed as 2024-11-19, and the award ceiling is stated as $1,000,000. The listing does not provide a number of expected awards in the excerpt provided. Overall, the opportunity is aimed at teams that already have a genome editing therapeutic concept with strong preclinical proof-of-concept and now need focused resources to optimize the candidate characteristics that regulators, manufacturers, and translational development programs typically require before advancing into IND-enabling efforts.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "Optimization of Genome Editing Therapeutics for Alzheimer's Disease-Related Dementias (ADRD) (U01 - Clinical Trials Not Allowed)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.853, 93.866.
• This funding opportunity was created on 2024-08-13.
• Applicants must submit their applications by 2024-11-19. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• Each selected applicant is eligible to receive up to $1,000,000.00 in funding.
• Eligible applicants include: State governments, County governments, City or township governments, Special district governments, Independent school districts, Public and State controlled institutions of higher education, Native American tribal governments (Federally recognized), Public housing authorities/Indian housing authorities, Native American tribal organizations (other than Federally recognized tribal governments), Nonprofits having a 501 (c) (3) status with the IRS, other than institutions of higher education, Nonprofits that do not have a 501 (c) (3) status with the IRS, other than institutions of higher education, Private institutions of higher education, For-profit organizations other than small businesses, Small businesses, Others.

Apply for RFA NS 24 037

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