Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
- Posted
- November 9, 2021
- Closed
- October 11, 2024
- Cost sharing
- No
- Instrument
- Cooperative Agreement
- Assistance listing
- 93.853
- Category
- Health
- Archives
- November 10, 2024
Program funding history
Awards made under Assistance Listing 93.853 across FY2025–FY2027, from public federal spending records.
- FY2025 obligated
- $1.8B
- FY2026 obligated
- $2B
- FY2027 (to date) obligated
- $0
- Awards in window
- 8,124
Top recipients: The General Hospital Corporation, Regents of the University of California, San Francisco, the, The Johns Hopkins University, Regents of the University of Michigan, Washington University, the
Source: USAspending.gov · refreshed October 2026 · Program page: awards, recipients and the opportunities under this listing →
Synopsis
The Ultra-Rare Gene-Based Therapy (URGenT) network supports Investigational New Drug (IND)-enabling studies and planning activities for First-in-Human (FIH) clinical testing of gene-based or transcript-directed therapeutics, such as oligonucleotides and viral-based gene therapies, for ultra-rare neurological or neuromuscular disorders. The goal of this funding opportunity announcement (FOA) is to accelerate the development of a promising clinical candidate with robust biological rationale and demonstrated proof of concept (POC) data for the intended approach in a model system relevant to a specified patient population towards an IND filing and the initiation of a clinical trial.
Who can apply
- Independent school districts
- Private institutions of higher education
- Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education
- For profit organizations other than small businesses
- City or township governments
- Native American tribal governments (Federally recognized)
- Public and State controlled institutions of higher education
- Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education
- County governments
- Others (see text field entitled "Additional Information on Eligibility" for clarification)
- Public housing authorities/Indian housing authorities
- Special district governments
- Native American tribal organizations (other than Federally recognized tribal governments)
- Small businesses
- State governments
Other Eligible Applicants include the following: Alaska Native and Native Hawaiian Serving Institutions; Asian American Native American Pacific Islander Serving Institutions (AANAPISISs); Eligible Agencies of the Federal Government; Faith-based or Community-based Organizations; Hispanic-serving Institutions; Historically Black Colleges and Universities (HBCUs); Indian/Native American Tribal Governments (Other than Federally Recognized); Non-domestic (non-U.S.) Entities (Foreign Organizations); Regional Organizations; Tribally Controlled Colleges and Universities (TCCUs) ; U.S. Territory or Possession; Non-domestic (non-U.S.) Entities (Foreign Institutions) are not eligible to apply. Non-domestic (non-U.S.) components of U.S. Organizations are not eligible to apply. Foreign components, as defined in the NIH Grants Policy Statement, are allowed.
The official record
Grants.gov published the official terms and dates of this opportunity; this page keeps them as they were listed.
View on Grants.gov Full announcement
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Similar grants
- Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
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- Innovation Grants to Nurture Initial Translational Efforts (IGNITE): Neurotherapeutic Agent Characterization and In vivo Efficacy Studies (R61/R33 Clinical Trial Not Allowed)
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