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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

PAR-25-327

HHS-NIH11

General Information

Est. Program Funding:

October 8, 2027

Closing Date:

Expected No. of Awards

Award Ceiling

Opportunity Number:
PAR-25-327

Owning Agency:
HHS-NIH11

Opportunity Category:
Discretionary (D)

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.

Response Date	Oct 08, 2027 12:00:00 AM EDT
Posting Date	Dec 18, 2024 12:00:00 AM EST
Archive Date	Nov 13, 2027 12:00:00 AM EST
Award Ceiling
Award Floor
Cost Sharing	No

Eligibility

Eligible Applicants

Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education, Native American tribal organizations (other than Federally recognized tribal governments), Independent school districts, County governments, Public and State controlled institutions of higher education, Special district governments, Small businesses, For profit organizations other than small businesses, State governments, Others (see text field entitled "Additional Information on Eligibility" for clarification), Private institutions of higher education, Public housing authorities/Indian housing authorities, Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education, City or township governments, Native American tribal governments (Federally recognized)

Additional Info on Eligibility:

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.

Additional Information

Agency Name	National Institutes of Health
Link to Additional Information:	https://grants.nih.gov/grants/guide/pa-files/PAR-25-327.html
Grantor Contact Information:	If you have difficulty accessing the full announcement electronically, please contact: GrantSolutions Help Desk Phone: 301-402-2541

CFDA Program(s)

93.853: Extramural Research Programs in the Neurosciences and Neurological Disorders
:

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