Posted opportunity — confirm at source
Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
National Institutes of Health · PAR-25-327
See source for award amount
2027-10-08-00-00-00
Confirm exact timezone in the announcement.Small businesses, County governments, Public housing authorities/Indian housing authorities, Public and State controlled institutions of higher education, State governments, 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, Special district governments, Independent school districts, Native American tribal governments (Federally recognized), For profit organizations other than small businesses, Others (see text field entitled "Additional Information on Eligibility" for clarification), Private institutions of higher education, Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education, City or township governments
Geographic eligibility and all attachment-specific conditions require review of the original announcement.
Eligibility details
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 Organizations) 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.
Matching funds & resources
Source matching-funds field: Not Required.
Matching funds and leveraged resources are different requirements. Read the description and reviewed rules below before determining resource commitments.
About this opportunity
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 announcement 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.
Funding geography: Not specified — review source. A funder’s address does not establish eligibility.
Review original source ↗Funding instrument: Cooperative Agreement. Issuing jurisdiction: Federal. This is not a geographic eligibility determination.
Source fields and evidence
id: 357817
Source ↗ · id
opportunityNumber: PAR-25-327
Source ↗ · opportunityNumber
opportunityTitle: Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
Source ↗ · opportunityTitle
revision: 0
Source ↗ · revision
agencyDetails: {"code":"NIH","seed":"HHS-NIH11","agencyName":"National Institutes of Health","agencyCode":"HHS-NIH11","topAgencyCode":"HHS"}
Source ↗ · agencyDetails
owningAgencyCode: HHS-NIH11
Source ↗ · owningAgencyCode
cfdas: [{"id":426611,"opportunityId":357817,"cfdaNumber":"93.853","programTitle":"Extramural Research Programs in the Neurosciences and Neurological Disorders"},{"id":426612,"opportunityId":357817}]
Source ↗ · cfdas
applicantTypes: [{"id":"23","description":"Small businesses"},{"id":"01","description":"County governments"},{"id":"08","description":"Public housing authorities/Indian housing authorities"},{"id":"06","description":"Public and State controlled institutions of higher education"},{"id":"00","description":"State governments"},{"id":"11","description":"Native American tribal organizations (other than Federally recognized tribal governments)"},{"id":"12","description":"Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education"},{"id":"04","description":"Special district governments"},{"id":"05","description":"Independent school districts"},{"id":"07","description":"Native American tribal governments (Federally recognized)"},{"id":"22","description":"For profit organizations other than small businesses"},{"id":"25","description":"Others (see text field entitled \"Additional Information on Eligibility\" for clarification)"},{"id":"20","description":"Private institutions of higher education"},{"id":"13","description":"Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education"},{"id":"02","description":"City or township governments"}]
Source ↗ · applicantTypes
applicantEligibilityDesc: 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 Organizations) 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.
Source ↗ · applicantEligibilityDesc
fundingInstruments: [{"id":"CA","description":"Cooperative Agreement"}]
Source ↗ · fundingInstruments
fundingActivityCategories: [{"id":"HL","description":"Health"}]
Source ↗ · fundingActivityCategories
awardFloor: none
Source ↗ · awardFloor
awardCeiling: none
Source ↗ · awardCeiling
responseDate: Oct 08, 2027 12:00:00 AM EDT
Source ↗ · responseDate
responseDateStr: 2027-10-08-00-00-00
Source ↗ · responseDateStr
costSharing: false
Source ↗ · costSharing
lastUpdatedDate: Dec 18, 2024 01:06:08 PM EST
Source ↗ · lastUpdatedDate
status: posted
Source ↗ · oppHits[id=357817].oppStatus
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