Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
National Institutes of Health · Extramural Research Programs in the Neurosciences and Neurological Disorders (93.853)
Posted 18 Dec 2024 · About 12 months left
Summary
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.
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Eligibility and conditions
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Applicant types: Special district governments; For profit organizations other than small businesses; City or township governments; Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education; County governments; Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education; Small businesses; Others (see text field entitled "Additional Information on Eligibility" for clarification); Independent school districts; Public housing authorities/Indian housing authorities; State governments; Native American tribal organizations (other than Federally recognized tribal governments); Private institutions of higher education; Native American tribal governments (Federally recognized); Public and State controlled 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.