Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
Department of Health and Human Services
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.
Funding
Award size
Not specified
Type
Cooperative Agreement
Dates
Deadline
Later
Posted
12/18/2024
Closes
2027-10-08
Eligibility
Eligibility
Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher educationSmall businessesNative American tribal organizations (other than Federally recognized tribal governments)For profit organizations other than small businessesCounty governmentsPublic and State controlled institutions of higher educationIndependent school districtsPrivate institutions of higher educationSpecial district governmentsPublic housing authorities/Indian housing authoritiesOthers (see text field entitled "Additional Information on Eligibility" for clarification)City or township governmentsNative American tribal governments (Federally recognized)Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher educationState governments
Program
Funding area
Health - HL
Opportunity #
PAR-25-327
Source
Status
Open
Market
Government
Level
Federal
Source
Grants.gov