Grant✓ Small businesses may apply
Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
National Institutes of Health
- Opportunity #
- PAR-25-327
- Agency
- National Institutes of Health
- ALN (CFDA)
- 93.853
- Status
- posted
- Posted
- Dec 18, 2024
- Closes
- Oct 8, 2027
- Funding instrument
- Cooperative Agreement
- Category
- Health
- Cost sharing required
- No
Who can apply
- •Independent school districts
- •City or township governments
- •Private institutions of higher education
- •Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education
- •State governments
- •Public and State controlled institutions of higher education
- •Native American tribal governments (Federally recognized)
- •Public housing authorities/Indian housing authorities
- •For profit organizations other than small businesses
- •Native American tribal organizations (other than Federally recognized tribal governments)
- •Small businesses
- •County governments
- •Special district governments
- •Others (see text field entitled "Additional Information on Eligibility" for clarification)
- •Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education
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.
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 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.
Contact
National Institutes of Health · grantsinfo@nih.gov