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

The Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access (X01) opportunity, administered by the National Institute of Neurological Disorders and Stroke (NINDS) under the National Institutes of Health (NIH), provides a critical support mechanism for researchers working on gene-based or transcript-directed therapies for ultra-rare neurological or neuromuscular diseases. This initiative acknowledges the unique challenges in addressing ultra-rare diseases—those affecting fewer than 6,000 individuals in the United States—and supports investigators with planning, manufacturing, and limited nonclinical development efforts necessary to advance toward Investigational New Drug (IND) applications. It serves as a reissue of PAR-22-028 and is supported by NINDS contract resources, including Contract Research Organizations (CROs), Contract Manufacturing Organizations (CMOs), and Subject Matter Experts (SMEs), at no cost to the awardees. The purpose of the program is to accelerate the preclinical development of patient-specific therapeutics by leveraging NINDS infrastructure and expertise. The URGenT Network provides in-kind access to planning services, manufacturing capabilities, and nonclinical testing resources. Eligible proposals must present viable therapeutic candidates with proof-of-concept data and a defined pathogenic variant for an ultra-rare condition. The support offered under this X01 mechanism can be used for manufacturing process development, confirmatory pharmacological studies, toxicity assessments, regulatory strategy planning, and assistance with FDA pre-IND and IND submission packages. The award does not provide monetary funds but facilitates access to high-value development resources. Participation requires that applicants have identified a therapeutic candidate supported by rigorous in vivo and/or in vitro data and are positioned to pursue regulatory engagement with the FDA. Entry into the network includes assembling a multidisciplinary project team with NIH staff and consultants, which then collaboratively defines a development plan and timeline. Activities must be completed within a two-year project period. The X01 mechanism is intended for planning and early implementation phases, rather than full-scale development or clinical trials. Applications proposing activities such as target discovery, in vitro assay development, basic research, or human clinical research are not considered responsive and will not be reviewed. The funding opportunity was originally scheduled to remain active through 2028 but was set to expire early on March 12, 2026, per notice NOT-NS-26-032. Multiple application cycles were offered annually with cut-off dates every few months between 2025 and 2028. As of March 13, 2026, the opportunity is expired, though NIH policy allows for limited late or continuous submission under certain circumstances with prior approval through the eRA Service Desk. All applications were required to be submitted electronically via ASSIST, institutional system-to-system (S2S) solutions, or Grants.gov Workspace. Eligibility for this program includes a broad spectrum of applicant types across public and private institutions, including higher education institutions, nonprofits, for-profits (small and large businesses), local and state governments, and Native American tribal organizations. However, non-U.S. entities are not eligible to apply as primary applicants. Intramural NIH scientists may participate under limited funding structures. Intellectual property developed through the URGenT Network is retained by the recipient institution, and SMEs involved in the project maintain confidentiality agreements to protect sensitive data. Contact for scientific questions is Dr. Chris Boshoff at NINDS. Applications are subject to a two-step review process, beginning with internal evaluation for feasibility and fit, followed by peer review for scientific merit. Projects are expected to lead toward FDA regulatory submissions and eventually clinical trials. While the URGenT X01 does not directly provide funding, it represents a vital access point for resources that are often out of reach for researchers tackling some of the rarest and most urgent diseases.

Follow NIH’s application instructions closely. Scientific rigor (blinding, randomization, appropriate controls) is mandatory for preliminary data.