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Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)

National Institutes of Health · Opportunity number PAR-25-327

View the official notice on Grants.gov
357 days left (deadline Oct 8, 2027)

Details summarized from the official notice as of Oct 15, 2026. Always confirm eligibility, deadlines and requirements in the official notice before applying.

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Key facts

Opportunity numberPAR-25-327
AgencyNational Institutes of Health
Department of Health and Human Services
Assistance listing (ALN, formerly CFDA)93.853
PostedDec 18, 2024
DeadlineOct 8, 2027
Cost sharing or matching requiredNo
Funding typeCooperative agreement
CategoryHealth
Last updated hereOct 15, 2026

Summary from the notice

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.

Who can apply

The official notice lists these applicant types as eligible:

  • City or township governments
  • For-profit organizations other than small businesses
  • Small businesses
  • Others named in the notice (see the eligibility text)
  • Private colleges and universities
  • Independent school districts
  • County governments
  • Special district governments
  • Public and Indian housing authorities
  • Nonprofits without 501(c)(3) status (other than colleges)
  • State governments
  • Nonprofits with 501(c)(3) status (other than colleges)
  • Public and state colleges and universities
  • Federally recognized tribal governments
  • Other Native American tribal organizations

Who should take a closer look

  • Small businesses
  • Nonprofits
  • Colleges and universities
  • State governments
  • Local governments
  • Tribal governments and organizations
  • Larger for-profit companies

Not listed as eligible

  • Individuals

Unless the eligibility text in the notice names you, this opportunity is probably not for you.

Eligibility details from the notice

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.

Registrations you will likely need

Most applications submitted through Grants.gov need the following. Registration can take several weeks, so start early. The notice lists the exact requirements.

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