Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access (X01, Clinical Trial Not Allowed) | PAR 22 028

Opportunity Information: Apply for PAR 22 028

The Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access opportunity (Funding Opportunity Number PAR 22 028) is an NIH National Institute of Neurological Disorders and Stroke (NINDS) program designed to help researchers move early gene-based therapy concepts for ultra-rare neurological diseases toward practical readiness. Rather than functioning like a typical research grant that mainly provides direct funding for investigator-led experiments, this X01 mechanism is structured to give investigators access to specialized resources and expertise housed within the URGenT Network. In practical terms, it is meant to reduce major barriers that often prevent ultra-rare disease therapy projects from advancing, especially when the work requires highly specialized development planning, manufacturing capabilities, and experienced subject matter guidance that many academic or small organizational teams do not have in-house. Clinical trials are explicitly not allowed under this opportunity, so the focus remains on preparatory and preclinical-enabling work instead of enrolling or treating human participants.

The core purpose of the FOA is to provide a formal pathway for investigators to work with contract research organizations (CROs), contract manufacturing organizations (CMOs), and subject matter experts (SMEs) connected to the NINDS URGenT Network. These resources are intended to support planning activities, manufacturing-related steps, and limited nonclinical therapeutic development efforts. In the gene therapy space, that typically means activities that help define what a viable therapy development package would look like and how it could realistically be produced and tested before any human study is contemplated. Examples of the kinds of support that fit this description often include early development roadmapping, refining a manufacturing strategy, addressing quality or process questions tied to vector or gene-based product production, and generating limited nonclinical data needed to make a program more mature and better positioned for later-stage development. The emphasis on "limited nonclinical therapeutic development" signals that this is not intended to fund broad discovery research or large, multi-year preclinical programs, but rather targeted work that meaningfully de-risks the path toward eventual translation.

This FOA is categorized as a discretionary grant opportunity in the health area (CFDA number 93.853) and is offered by the National Institutes of Health. The mechanism is X01, which generally indicates a resource access award rather than a conventional research project grant. The listing does not provide an award ceiling or an expected number of awards in the provided text, which typically means applicants need to rely on the full FOA details and URGenT program guidance for specifics about what level of resource support is available, what services can be requested, and how projects will be prioritized.

Eligibility is broad across U.S.-based organizations and includes many standard applicant types: state, county, and local governments; 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; public housing authorities/Indian housing authorities; tribal organizations that are not federally recognized governments; nonprofit organizations (both 501(c)(3) and non-501(c)(3) entities, excluding institutions of higher education in those nonprofit categories); for-profit organizations (other than small businesses); and small businesses. The FOA 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, eligible federal government agencies, regional organizations, and U.S. territories or possessions. This broad eligibility reflects an intent to make URGenT resources accessible to diverse institutions and organizations that may be positioned to lead ultra-rare disease therapy efforts, including community-embedded groups and mission-driven nonprofits that often play a central role in ultra-rare disease research ecosystems.

At the same time, foreign participation is limited in specific ways. Non-domestic (non-U.S.) entities (foreign institutions) are not eligible to apply, and non-domestic components of U.S. organizations are not eligible to apply. However, "foreign components," as NIH defines them in the NIH Grants Policy Statement, are allowed. In NIH terms, that typically means a U.S. applicant organization can include certain types of work performed in a foreign setting when it is justified and meets NIH policy requirements, but the applicant organization itself must be domestic and the structure must comply with the rules on foreign components. Anyone considering an international collaboration would need to pay close attention to how NIH distinguishes between a foreign institution applicant, a non-domestic component of a U.S. organization, and an allowable foreign component under policy.

The key dates in the provided source data indicate the opportunity was created on 2021-11-09, with an original closing date of 2025-01-24. Those dates matter because X01 opportunities can be updated or reissued over time, and applicants should verify whether there are multiple receipt dates, changes, or re-competition notices in the full announcement. Overall, the URGenT Network Resource Access FOA is best understood as a specialized on-ramp for teams working on ultra-rare gene-based therapies who need expert development help, access to manufacturing and contracted development capacity, and guidance to prepare a therapy program for later stages, while staying firmly on the nonclinical side and avoiding clinical trial activity under this mechanism.

• 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 2021-11-09.
• Applicants must submit their applications by 2025-01-24. (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.

Apply for PAR 22 028

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