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

ID: PAR-22-030 • Type: Posted
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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 funding opportunity announcement (FOA) 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.

Overview

Category of Funding
Health
Funding Instruments
Cooperative Agreement
Grant Category
Discretionary
Cost Sharing / Matching Requirement
False
Source
On 11/9/21 the National Institutes of Health posted grant opportunity PAR-22-030 for Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional). The grant will be issued under grant program 93.853 Extramural Research Programs in the Neurosciences and Neurological Disorders.

Timing

Posted Date
Nov. 9, 2021, 12:00 a.m. EST
Closing Date
Oct. 11, 2024, 12:00 a.m. EDT Past Due
Last Updated
Dec. 1, 2023, 12:03 p.m. EST
Version
2
Archive Date
Nov. 10, 2024

Eligibility

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

Contacts

Contact
National Institutes of Health
Email Description
See Section VII. Agency Contacts within the full opportunity announcement for all other inquires.
Contact Phone
(301) 402-2541

Documents

Posted documents for PAR-22-030

Grant Awards

Grants awarded through PAR-22-030

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