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OpenOpportunity PAR-25-327

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

National Institutes of Health · Department of Health and Human Services

At a glance

Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) is an open federal cooperative agreement opportunity from the National Institutes of Health (Department of Health and Human Services). Applications are due October 8, 2027. Applicant types listed as eligible include state governments, county governments, city or township governments, special district governments, independent school districts and 10 more. Cost sharing is not required. It is funded under Assistance Listing 93.853 (Extramural Research Programs in the Neurosciences and Neurological Disorders).

Summary assembled from the official notice's fields. The official notice governs.

Key facts

Posted
Dec 18, 2024
Close date
Oct 8, 2027
Archive date
Nov 13, 2027
Award floor
Not specified
Award ceiling
Not specified
Est. total funding
Not specified
Expected awards
Not specified
Cost sharing
Not required
Funding instrument
Cooperative Agreement
Opportunity category
Discretionary

Who can apply

  • State governments
  • County governments
  • City or township governments
  • Special district governments
  • Independent school districts
  • Public colleges & universities
  • Federally recognized tribal governments
  • Public & Indian housing authorities
  • Native American tribal organizations
  • 501(c)(3) nonprofits
  • Nonprofits without 501(c)(3) status
  • Private colleges & universities
  • For-profit organizations (not small businesses)
  • Small businesses
  • Others (see notice)

Additional information on eligibility (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.

Based on the published notice. Review the official notice for complete eligibility requirements.

What it funds

Official synopsis as published by the agency, formatted for readability.

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.

Additional information from the agency ↗

Assistance Listing

What is an ALN?

Reported obligations under ALN 93.853

Historical data · not a prediction
FY24$2.36B
FY25$2.35B
FY26— est.
Source: SAM.gov Assistance Listing 93.853. Program-wide totals, not awards from this specific opportunity.

Agency contact (as published)

National Institutes of Health
grantsinfo@nih.gov
301-402-2541

Quick answers

When is the deadline for this opportunity?

According to the official notice, applications are due October 8, 2027. Confirm the deadline and submission time on Grants.gov before applying.

Who is eligible to apply for this opportunity?

The official notice lists these applicant types as eligible: State governments, County governments, City or township governments, Special district governments, Independent school districts, Public colleges & universities, Federally recognized tribal governments, Public & Indian housing authorities, Native American tribal organizations, 501(c)(3) nonprofits, Nonprofits without 501(c)(3) status, Private colleges & universities and 3 more. Additional restrictions may apply; review the full notice.

How do I apply for this opportunity?

Applications are submitted through the official source, not through GrantsJunction. Open the official notice on Grants.gov (opportunity PAR-25-327), and review the full announcement and application package. Organizations applying through Grants.gov generally need an active SAM.gov registration and a Grants.gov account, which can take several weeks to set up.

Matched on shared Assistance Listings, agency, categories and eligible applicant types.