OpenFederal grantOpen to for-profit businessesPAR-25-327

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

National Institutes of HealthCloses Oct 8, 2027(360 days left)

Does this fit your business?

This is an NIH research program for teams already holding a promising gene-based therapy candidate for an ultra-rare neurological or neuromuscular disorder, with proof-of-concept data in hand, who need IND-enabling studies and first-in-human trial planning. If you are a typical small business outside biotech or drug development, this is not your grant and applying wastes your week. Foreign organizations are not eligible.

Best for
Biotech and life-science companies with a gene-therapy candidate ready for IND-enabling work, Universities and academic medical centers running translational neuroscience research, Nonprofits and foundations focused on ultra-rare neurological or neuromuscular diseases, Small businesses doing preclinical R&D toward a first-in-human trial
Not for
Businesses outside biotech with no gene-therapy or drug-development pipeline, Foreign or non-U.S.-based organizations, Organizations seeking general operating funds or early discovery-stage research
Estimated fit by industry, best first
IndustryEstimated fit
Healthcare & BiotechMatches your industry (clinical)
80
Strong Match
Education & TrainingMatches your industry (stem)
69
Good Match
Technology & SoftwareCurrently accepting applications
68
Good Match
Agriculture & FarmingLimit: Not clearly about your industry or what you need funding for; check the…
54
Possible Match
Arts & CultureLimit: Nothing in this notice points to arts & culture
54
Possible Match
Construction & InfrastructureLimit: Not clearly about your industry or what you need funding for; check the…
54
Possible Match
Show all 15 industries
Estimated fit by industry, best first
IndustryEstimated fit
Energy & Clean TechLimit: Nothing in this notice points to energy & clean tech
54
Possible Match
Environment & ConservationLimit: Nothing in this notice points to environment & conservation
54
Possible Match
Finance & BankingLimit: Nothing in this notice points to finance & banking
54
Possible Match
Food & BeverageLimit: Not clearly about your industry or what you need funding for; check the…
54
Possible Match
Housing & Real EstateLimit: Nothing in this notice points to housing & real estate
54
Possible Match
ManufacturingLimit: Not clearly about your industry or what you need funding for; check the…
54
Possible Match
Retail & E-CommerceLimit: Not clearly about your industry or what you need funding for; check the…
54
Possible Match
Social Services & NonprofitLimit: Nothing in this notice points to social services & nonprofit
54
Possible Match
Transportation & LogisticsLimit: Not clearly about your industry or what you need funding for; check the…
54
Possible Match

These are our matcher’s estimates for a typical business in each industry. A real match depends on your size, your state and what you need the money for — check your own fit.

What this funds

Money can be used for
Research, R&D, Planning
Who can apply
Small businesses, For-profit companies, Nonprofits, State and local governments, Tribal governments and organizations, Colleges and universities, Other applicants (see eligibility)

Questions about this grant

When is the application deadline?
October 8, 2027.
Who can apply for this grant?
Small businesses, for-profit companies, nonprofits (with or without 501(c)(3) status), public and private universities, and federal, state, local, county, city, tribal, school-district, special-district, and public-housing government entities. Foreign organizations are not eligible, and non-U.S. components of U.S. organizations cannot apply.
Can a sole proprietor or single-member LLC apply?
The facts do not say. The eligibility list names small businesses and for-profit organizations other than small businesses, but it does not spell out whether a sole proprietor or single-member LLC qualifies, so check the full notice on Grants.gov before counting on it.
Can a nonprofit apply?
Yes. Nonprofits with 501(c)(3) status and nonprofits without 501(c)(3) status (other than institutions of higher education) are both listed as eligible.

Summary

NIH URGenT network funding for IND-enabling studies and planning for first-in-human trials of gene-based therapies for ultra-rare neurological/neuromuscular disorders. Open to small businesses, for-profits, nonprofits, universities, and state/local/tribal governments; foreign entities ineligible.

About this grant

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

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.

Topics and uses of funds

Topics

  • Health
  • Science
  • Technology

Program contact

National Institutes of Healthgrantsinfo@nih.gov301-402-2541

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