Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access (X01, Clinical Trial Not Allowed)
ID: 357815Type: Posted
Overview

Buyer

National Institutes of Health (HHS-NIH11)

Eligible Applicants

Others

Funding Category

Health

Funding Instrument

Grant

Opportunity Category

Discretionary

Cost Sharing or Matching Requirement

Yes

Original Source

https://grants.gov/search-results-detail/357815

Additional Information

http://grants.nih.gov/grants/guide/pa-files/PAR-25-326.html
Timeline
    Description

    The National Institutes of Health (NIH) is inviting applications for the Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access program under funding opportunity number PAR-25-326. This initiative aims to facilitate access to contract research organizations and subject matter experts to support nonclinical development activities for gene-based therapies targeting ultra-rare neurological and neuromuscular diseases. The program emphasizes the urgency of developing tailored therapies due to the critical nature of these conditions and the lack of existing commercial therapeutic options, encouraging proposals that demonstrate proof of concept data for defined patient populations. Applications are accepted on a rolling basis, with a close date of January 31, 2028, and interested parties can reach out to NIH Grants Information at grantsinfo@nih.gov for further inquiries. Additional details can be found at the provided link: https://grants.nih.gov/grants/guide/pa-files/PAR-25-326.html.

    Point(s) of Contact
    grantsinfo@nih.gov
    Files
    Title
    Posted
    PAR-25-326-Full-Announcement.html
    PAR-25-326.html
    The National Institutes of Health (NIH) is inviting applications for the Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access program under the funding opportunity number PAR-25-326. This initiative aims to facilitate access to contract research organizations and subject matter experts to support nonclinical development activities for gene-based therapies targeting ultra-rare neurological and neuromuscular diseases. The program encourages proposals that demonstrate proof of concept data for a defined patient population, addressing significant unmet medical needs. Key activities include planning and limited nonclinical studies that may lead to an Investigational New Drug (IND) application with the FDA. Applications are accepted on a rolling basis with specific submission deadlines, focused on enhancing access to URGenT resources, developing product plans, and ensuring regulatory compliance. The program does not provide direct funding but offers support through access to specialized resources to aid in the advancement of therapeutics for ultra-rare diseases. This NOFO emphasizes the urgency of developing tailored therapies due to the critical nature of these conditions and the lack of existing commercial therapeutic options.
    Similar Opportunities
    Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
    National Institutes of Health
    The National Institutes of Health (NIH) is offering a funding opportunity titled "Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders" (PAR-25-327), aimed at supporting Investigational New Drug (IND)-enabling studies and preparations for First-in-Human (FIH) clinical trials. This initiative focuses on advancing innovative gene-based therapies, such as oligonucleotides and viral vector approaches, for ultra-rare disorders affecting fewer than 6,000 patients, with an emphasis on projects that demonstrate robust proof of concept data and readiness for clinical trials. Eligible applicants include a wide range of organizations, including higher education institutions and various nonprofit and for-profit entities, with applications requiring detailed project planning and compliance with Good Manufacturing Practices (GMP). The application period opens on January 9, 2025, with a close date of October 8, 2027, and interested parties can reach out to NIH Grants Information at grantsinfo@nih.gov for further inquiries.
    Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
    National Institutes of Health
    The National Institutes of Health (NIH) has announced a funding opportunity titled "Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)" aimed at supporting clinical projects that enhance readiness for clinical trials in rare diseases. This initiative seeks applications that focus on developing and testing biomarkers and clinical outcome measures, as well as defining the characteristics of rare diseases to facilitate the design of future clinical trials. The program is particularly important for addressing the needs of rare diseases affecting fewer than 200,000 individuals in the U.S., with a maximum budget of $275,000 for direct costs over a project period of up to two years. Interested applicants can find more information and submit inquiries via email to grantsinfo@nih.gov, with the application deadline set for July 20, 2028.
    GREGoRi Innovation Projects (U01 Clinical Trial Optional)
    National Institutes of Health
    The National Institutes of Health (NIH) is set to announce a funding opportunity titled "GREGoRi Innovation Projects (U01 Clinical Trial Optional)" aimed at soliciting applications for research on proof of concept studies that explore innovative strategies for identifying genes or variants associated with rare genetic disorders. This initiative seeks to advance beyond traditional DNA sequencing methods by incorporating emerging molecular technologies, such as multi-omics and RNA-seq, to enhance the understanding of these disorders. The funding will be provided through a cooperative agreement, with no cost-sharing requirement, and is particularly targeted at small businesses engaged in health-related research. Interested applicants should prepare for the anticipated Notice of Funding Opportunity (NOFO) publication in 2025, with applications due in 2026, and can reach out to Lisa H. Chadwick at lisa.chadwick@nih.gov or by phone at 301-435-7275 for further information.
    GREGoRi Data Coordination and Outreach Center (U01, Clinical Trials Not Allowed)
    National Institutes of Health
    The National Institutes of Health (NIH) is offering a funding opportunity for the establishment of the GREGoRi Data Coordination and Outreach Center, aimed at advancing the Genomics Research to Elucidate the Genetics of Rare Disease (GREGoR) program. This initiative seeks to enhance the diagnosis of rare genetic disorders by developing innovative tools and analytical methods to identify causal genes and variants, with the center serving as the primary coordination hub for data and resources generated by the GREGoRi program. The total estimated funding for this cooperative agreement is $2 million, with one award anticipated, and applications are expected to be due in 2026 following the publication of the Notice of Funding Opportunity (NOFO) in 2025. For further inquiries, interested applicants can contact Lisa H. Chadwick at lisa.chadwick@nih.gov or by phone at 301-435-7275.
    Clinical Validation of a Candidate Biomarker for Neurological or Neuromuscular Disorders (U01 Clinical Trial Optional)
    National Institutes of Health
    The National Institutes of Health (NIH) is offering a funding opportunity titled "Clinical Validation of a Candidate Biomarker for Neurological or Neuromuscular Disorders (U01 Clinical Trial Optional)." This initiative aims to support the clinical validation of strong candidate biomarkers for neurological and neuromuscular disorders, focusing on establishing their clinical sensitivity and specificity in accordance with FDA guidelines. The program is particularly significant as it seeks to enhance public health outcomes by facilitating the implementation of validated biomarkers in clinical settings, thereby addressing critical healthcare needs in these areas. Interested applicants should note that the application period opens on January 21, 2025, and closes on June 22, 2026. For further inquiries, applicants can contact NIH Grants Information at grantsinfo@nih.gov, and additional details can be found at the provided link: https://grants.nih.gov/grants/guide/pa-files/PAR-25-055.html.
    Blueprint Neurotherapeutics Network (BPN): Biologic-based Drug Discovery and Development for Disorders of the Nervous System (UG3/UH3 Clinical Trial Optional)
    National Institutes of Health
    The National Institutes of Health (NIH) has announced a funding opportunity titled "Blueprint Neurotherapeutics Network (BPN): Biologic-based Drug Discovery and Development for Disorders of the Nervous System," aimed at supporting the development of biologic therapies for nervous and neuromuscular disorders. This initiative is structured as a cooperative agreement, divided into two phases: UG3 for lead optimization and UH3 for IND-enabling activities, with a focus on specific treatment applications for these disorders. The program encourages collaboration with NIH resources, including access to contract research organizations and experienced consultants, to facilitate the development of innovative therapies. Interested applicants, including higher education institutions and nonprofits, are encouraged to apply by August 18, 2027, and can find more information at the provided NIH grants link or contact NIH Grants Information at grantsinfo@nih.gov.
    GREGoRi Technology Integration Center (U01, Clinical Trials Optional)
    National Institutes of Health
    The National Institutes of Health (NIH) is announcing a funding opportunity for the establishment of the GREGoRi Technology Integration Center (TechIC) under the Genomics Research to Elucidate the Genetics of Rare Disease (GREGoR) program. This initiative aims to advance the diagnosis of rare genetic disorders by developing standards and best practices for new molecular methods, ultimately producing a multidimensional dataset to support the creation and evaluation of innovative diagnostic tools. The total estimated funding for this cooperative agreement is $3 million, with one award anticipated, and applications are expected to be due in 2026 following the publication of the Notice of Funding Opportunity (NOFO) in 2025. For further inquiries, interested applicants can contact Lisa H. Chadwick at lisa.chadwick@nih.gov or by phone at 301-435-7275.
    Notice of Intent to Publish the re-issuance of the Notice of Funding Opportunity (NOFO) Rare Diseases Clinical Research Consortia (RDCRC) for the Rare Diseases Clinical Research Network (RDCRN) (U54 Clinical Trial Optional)
    National Institutes of Health
    The National Institutes of Health (NIH) is set to re-issue the Notice of Funding Opportunity (NOFO) for the Rare Diseases Clinical Research Consortia (RDCRC) under the Rare Diseases Clinical Research Network (RDCRN), aimed at enhancing research in the diagnosis, management, and treatment of rare diseases. Each RDCRC is required to focus on at least three rare diseases and propose two to four clinical research projects, including one longitudinal study, emphasizing a collaborative, multi-site approach to address significant clinical trial readiness gaps. This initiative reflects the government's commitment to advancing public health through innovative methodologies and stakeholder engagement in the rare disease domain, with applications expected to be due in June and fall of 2025. Interested applicants can reach out to Dr. Tiina K. Urv at 301-827-2746 or via email at urvtiin@mail.nih.gov for further information.
    Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)
    National Institutes of Health
    The National Institutes of Health (NIH) has announced a funding opportunity for preclinical proof of concept studies targeting rare diseases under the R21 Exploratory/Developmental Research Grant mechanism. This initiative aims to support efficacy studies in established preclinical models for therapeutic agents, including small molecules and biologics, with the goal of advancing projects toward Investigational New Drug (IND) applications. The funding is particularly significant as it addresses the unmet medical needs of approximately 30 million individuals in the U.S. affected by rare diseases, enhancing the likelihood of progressing novel therapies to clinical trials. The total funding available is up to $1,200,000 for FY26, with a maximum budget of $275,000 per two-year project. Interested applicants must submit their letters of intent by May 1, 2025, and can find more information by contacting NIH Grants Information at grantsinfo@nih.gov or visiting the opportunity's webpage at https://grants.nih.gov/grants/guide/rfa-files/RFA-TR-25-002.html.
    Clinical Validation of a Candidate Biomarker for Neurological or Neuromuscular Disorders (U01 Clinical Trial Optional)
    National Institutes of Health
    The National Institutes of Health (NIH) has announced a funding opportunity titled "Clinical Validation of a Candidate Biomarker for Neurological or Neuromuscular Disorders (U01 Clinical Trial Optional)" under Funding Opportunity Number PAR-24-097. This initiative aims to clinically validate candidate biomarkers for neurological diseases, focusing on establishing their clinical sensitivity and specificity, provided that preliminary conditions such as detection method development are met. The program emphasizes the importance of multidisciplinary collaborations and requires applicants to include a Plan for Enhancing Diverse Perspectives (PEDP) to promote inclusivity in research teams. Interested applicants must submit their proposals by February 22, 2024, with project durations capped at five years. For further inquiries, applicants can contact NIH Grants Information at grantsinfo@nih.gov, and additional details can be found at the provided link: https://grants.nih.gov/grants/guide/pa-files/PAR-24-097.html.