Assay Development and Screening for Discovery of Validated Chemical Hits for Brain Disorders (R01 Clinical Trial Not Allowed) | PAR 25 063

Opportunity Information: Apply for PAR 25 063

The funding opportunity titled "Assay Development and Screening for Discovery of Validated Chemical Hits for Brain Disorders (R01 Clinical Trial Not Allowed)" (PAR 25-063) is a National Institutes of Health (NIH) discretionary grant program designed to push early-stage drug discovery forward for brain and behavior-related conditions, with a specific emphasis on psychiatric disorders. At its core, the NOFO is trying to accelerate the creation and improvement of laboratory assays and screening strategies that can identify new small-molecule "hits" that are credible starting points for future therapeutic development. These chemical hits are intended to be used both as potential leads for treatment-oriented research and as practical tools to probe disease biology in ways that clarify which mechanisms truly matter.

A central theme of the program is target validation and biological insight. Many promising ideas in brain disorders fail because the underlying biological target or pathway is not actually causal, not druggable in practice, or not connected to meaningful disease-relevant outcomes. This NOFO supports projects that develop assays and run screens in a way that does more than just generate lists of active compounds. Applicants are expected to produce validated chemical hits that can reliably engage a relevant biological process and help answer whether a disease mechanism is worth pursuing. In other words, the goal is to fund research that produces usable, well-characterized chemical matter while simultaneously strengthening (or challenging) the rationale for the proposed target, pathway, or disease process.

The scientific scope focuses on discovery and development of novel small molecules. That typically means building or optimizing robust, reproducible assays (for example, biochemical, cell-based, phenotypic, or pathway-focused assays) and then using those assays in screening campaigns to find compounds that show activity consistent with the intended mechanism or phenotype. The funding is aligned with research that bridges basic neuroscience and translational discovery: projects should be grounded in disease relevance to psychiatric disorders, yet also be capable of generating generalizable insights into brain disease biology, especially in areas where drug targets have not yet been convincingly validated.

The mechanism is an NIH R01, which generally supports hypothesis-driven, multi-year research projects with enough resources to build substantial experimental programs. The announcement explicitly states "Clinical Trial Not Allowed," meaning the funded work must stay on the preclinical or non-clinical side. Studies can involve assay development, screening, hit confirmation, and related experimental validation activities, but they cannot include clinical trial activities that prospectively assign human participants to interventions to study health-related outcomes.

Eligibility is broad and includes many types of institutions and organizations. In addition to standard eligible applicants such as public and private institutions of higher education, nonprofit organizations (both with and without 501(c)(3) status), for-profit organizations (including entities other than small businesses and also small businesses), and various levels of government (state, county, city/township, special districts), the NOFO also welcomes applications from Native American tribal governments (federally recognized) and tribal organizations (including those other than federally recognized tribal governments), independent school districts, and public housing authorities/Indian housing authorities. The announcement also highlights additional eligible applicant categories such as Alaska Native and Native Hawaiian Serving Institutions, Asian American Native American Pacific Islander Serving Institutions (AANAPISIs), Hispanic-serving Institutions, Historically Black Colleges and Universities (HBCUs), Tribally Controlled Colleges and Universities (TCCUs), faith-based or community-based organizations, regional organizations, U.S. territories or possessions, and even non-domestic (non-U.S.) entities (foreign organizations). This wide eligibility is intended to broaden participation and encourage diverse scientific teams and settings to contribute to psychiatric drug discovery and target validation.

Administratively, the opportunity is listed under CFDA number 93.242, is run by the NIH, and was created on 2024-11-06. The original closing date shown is 2026-05-07. The listing does not provide an award ceiling or expected number of awards in the provided source data, so applicants would need to consult the full NOFO and NIH budgeting rules for typical R01 cost expectations and any institute-specific guidance.

Overall, this NOFO is best understood as support for rigorous, preclinical discovery work that transforms early ideas about psychiatric disease mechanisms into practical experimental systems and credible chemical starting points. Success under this program would look like an assay and screening workflow that holds up under repetition, yields confirmed and interpretable small-molecule hits, and produces evidence that meaningfully advances understanding of which biological processes in psychiatric disorders are truly promising for therapeutic exploration.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "Assay Development and Screening for Discovery of Validated Chemical Hits for Brain Disorders (R01 Clinical Trial Not Allowed)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.242.
• This funding opportunity was created on 2024-11-06.
• Applicants must submit their applications by 2026-05-07. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• 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.

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