Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access (X01, Clinical Trial Not Allowed) | PAR 25 326

Opportunity Information: Apply for PAR 25 326

The Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access opportunity (PAR 25-326) is a National Institutes of Health (NIH) funding announcement that gives researchers a formal way to tap into specialized development resources housed in the NINDS URGenT Network. Rather than being a traditional cash award meant to broadly fund a research program, this mechanism is designed to connect investigators to a coordinated set of contract research/medical organizations (CROs/CMOs) and highly relevant subject matter experts (SMEs). The main goal is to help teams working on gene-based therapies for ultra-rare neurological diseases move through the early, practical steps that often block progress, especially the planning and technical work required to get a program ready for later-stage development.

In terms of what the award supports, the emphasis is on planning, manufacturing-related support, and limited nonclinical therapeutic development activities. In plain language, the URGenT Network is positioned to help applicants navigate the real-world requirements that sit between an academic proof-of-concept and a product that is manufactured and characterized in a way regulators and future clinical partners will recognize. That can include coordinating with manufacturing organizations and experts who understand gene therapy production considerations, quality expectations, and development strategy. At the same time, this opportunity explicitly does not allow clinical trials, so the scope is restricted to preclinical and preparatory work rather than enrolling human participants or running interventional clinical studies.

The funding instrument is an NIH grant using the X01 activity code, listed under the health funding activity category and CFDA number 93.853. The opportunity is categorized as discretionary, with NIH as the sponsoring agency, and it was created on December 18, 2024. The original closing date is January 31, 2028, which signals a multi-year window during which applications may be accepted under the announcement’s schedule and submission policies. Details like the award ceiling and expected number of awards are not specified in the provided source text, which is common for resource-access style announcements where support may be structured around services and network capacity rather than a fixed dollar cap.

Eligibility is broad across the typical NIH landscape and includes many types of domestic organizations and governments. Eligible applicants include 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 Native American tribal governments; tribal organizations that are not federally recognized; public housing authorities/Indian housing authorities; nonprofits with or without 501(c)(3) status (when not institutions of higher education); for-profit organizations other than small businesses; small businesses; and other eligible entities. The announcement also explicitly calls out additional eligible applicant categories such as Alaska Native and Native Hawaiian Serving Institutions, AANAPISISs, 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, Indian/Native American Tribal Governments (other than federally recognized), and U.S. territories or possessions.

There are important limits related to foreign participation. Non-domestic (non-U.S.) entities (foreign organizations) are not eligible to apply, and non-domestic components of U.S. organizations are also not eligible to apply. However, foreign components as defined by the NIH Grants Policy Statement are allowed. Practically, that means the applicant organization must be U.S.-based and meet NIH’s domestic eligibility rules, but the proposed work may still include certain foreign elements if they qualify as foreign components under NIH policy and are justified and allowable within the application.

Overall, this NOFO is best understood as a pathway for ultra-rare gene therapy teams to gain structured access to specialized expertise and contracted capabilities needed for early development readiness, particularly around manufacturing planning and limited nonclinical steps, while staying clearly on the non-clinical side of the translational pipeline due to the clinical trial prohibition.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access (X01, 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): 93.853.
• This funding opportunity was created on 2024-12-18.
• Applicants must submit their applications by 2028-01-31.
• 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 PAR 25 326

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