Open · 594 days left D National Institutes of Health

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

Funding
Not specified
Deadline
--
Days
--
Hrs
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Min
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Sec
Oct 08, 2027
Posted Dec 18, 2024 (429 days ago)
Closes Oct 8, 2027 (in 594 days)

Grant Details

Opportunity Number
PAR-25-327
CFDA / ALN
93.853
Opportunity Category
Discretionary (D)
Funding Category
Health (HL)
Funding Instrument
Cooperative Agreement (CA)
Cost Sharing
No Cost Sharing (No)

Eligibility

State governments (00) County governments (01) City or township governments (02) Special district governments (04) Independent school districts (05) Public and State controlled institutions of higher education (06) Native American tribal governments (Federally recognized) (07) Public housing authorities / Indian housing authorities (08) Native American tribal organizations (11) Nonprofits having a 501(c)(3) status with the IRS (12) Nonprofits without 501(c)(3) status (13) Private institutions of higher education (20) For-profit organizations other than small businesses (22) Small businesses (23) Others (25)

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.

Description

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.