This URGenT Network grant offers funding to advance gene-based and transcript-directed therapies for ultra-rare neurological and neuromuscular disorders. The program specifically supports Investigational New Drug (IND)-enabling studies and crucial planning activities required for First-in-Human (FIH) clinical testing. Its core purpose is to accelerate promising clinical candidates – including oligonucleotides and viral-based gene therapies – that exhibit robust biological rationale and demonstrated proof-of-concept data in relevant model systems. The ultimate goal is to facilitate an IND filing and the subsequent initiation of a clinical trial. This initiative underscores a vital commitment to translating innovative research into clinical applications for patients with significant unmet medical needs in these specific ultra-rare conditions.
Opportunity ID: 336476
General Information
| Document Type: | Grants Notice |
| Funding Opportunity Number: | PAR-22-030 |
| Funding Opportunity Title: | Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 – Clinical Trial Optional) |
| Opportunity Category: | Discretionary |
| Opportunity Category Explanation: | – |
| Funding Instrument Type: | Cooperative Agreement |
| Category of Funding Activity: | Health |
| Category Explanation: | – |
| Expected Number of Awards: | – |
| Assistance Listings: | 93.853 — Extramural Research Programs in the Neurosciences and Neurological Disorders |
| Cost Sharing or Matching Requirement: | No |
| Version: | Synopsis 2 |
| Posted Date: | Nov 09, 2021 |
| Last Updated Date: | Dec 01, 2023 |
| Original Closing Date for Applications: | Jun 07, 2024 |
| Current Closing Date for Applications: | Oct 11, 2024 |
| Archive Date: | Nov 10, 2024 |
| Estimated Total Program Funding: | – |
| Award Ceiling: | – |
| Award Floor: | – |
Eligibility
| Eligible Applicants: | County governments State governments Public housing authorities/Indian housing authorities Independent school districts Nonprofits having 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 Small businesses Private institutions of higher education City or township governments Native American tribal governments (Federally recognized) 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) For profit organizations other than small businesses Others (see text field entitled “Additional Information on Eligibility” for clarification) |
| Additional Information 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 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. |
Additional Information
| Agency Name: | National Institutes of Health |
| 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. |
| Link to Additional Information: | http://grants.nih.gov/grants/guide/pa-files/PAR-22-030.html |
| Grantor Contact Information: | If you have difficulty accessing the full announcement electronically, please contact:
NIH OER Webmaster
OERWebmaster03@od.nih.gov Email:OERWebmaster03@od.nih.gov |
Version History
| Version | Modification Description | Updated Date |
|---|---|---|
| updated expiration date | Dec 01, 2023 | |
| Nov 09, 2021 |
DISPLAYING: Synopsis 2
General Information
| Document Type: | Grants Notice |
| Funding Opportunity Number: | PAR-22-030 |
| Funding Opportunity Title: | Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 – Clinical Trial Optional) |
| Opportunity Category: | Discretionary |
| Opportunity Category Explanation: | – |
| Funding Instrument Type: | Cooperative Agreement |
| Category of Funding Activity: | Health |
| Category Explanation: | – |
| Expected Number of Awards: | – |
| Assistance Listings: | 93.853 — Extramural Research Programs in the Neurosciences and Neurological Disorders |
| Cost Sharing or Matching Requirement: | No |
| Version: | Synopsis 2 |
| Posted Date: | Nov 09, 2021 |
| Last Updated Date: | Dec 01, 2023 |
| Original Closing Date for Applications: | Jun 07, 2024 |
| Current Closing Date for Applications: | Oct 11, 2024 |
| Archive Date: | Nov 10, 2024 |
| Estimated Total Program Funding: | – |
| Award Ceiling: | – |
| Award Floor: | – |
Eligibility
| Eligible Applicants: | County governments State governments Public housing authorities/Indian housing authorities Independent school districts Nonprofits having 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 Small businesses Private institutions of higher education City or township governments Native American tribal governments (Federally recognized) 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) For profit organizations other than small businesses Others (see text field entitled “Additional Information on Eligibility” for clarification) |
| Additional Information 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 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. |
Additional Information
| Agency Name: | National Institutes of Health |
| 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. |
| Link to Additional Information: | http://grants.nih.gov/grants/guide/pa-files/PAR-22-030.html |
| Grantor Contact Information: | If you have difficulty accessing the full announcement electronically, please contact:
NIH OER Webmaster
OERWebmaster03@od.nih.gov Email:OERWebmaster03@od.nih.gov |
DISPLAYING: Synopsis 1
General Information
| Document Type: | Grants Notice |
| Funding Opportunity Number: | PAR-22-030 |
| Funding Opportunity Title: | Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 – Clinical Trial Optional) |
| Opportunity Category: | Discretionary |
| Opportunity Category Explanation: | – |
| Funding Instrument Type: | Cooperative Agreement |
| Category of Funding Activity: | Health |
| Category Explanation: | – |
| Expected Number of Awards: | – |
| Assistance Listings: | 93.853 — Extramural Research Programs in the Neurosciences and Neurological Disorders |
| Cost Sharing or Matching Requirement: | No |
| Version: | Synopsis 1 |
| Posted Date: | Nov 09, 2021 |
| Last Updated Date: | Nov 09, 2021 |
| Original Closing Date for Applications: | – |
| Current Closing Date for Applications: | Jun 07, 2024 |
| Archive Date: | Jul 13, 2024 |
| Estimated Total Program Funding: | – |
| Award Ceiling: | – |
| Award Floor: | – |
Eligibility
| Eligible Applicants: | Nonprofits that do not have 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) City or township governments Small businesses Public and State controlled institutions of higher education For profit organizations other than small businesses Public housing authorities/Indian housing authorities State governments County governments Special district governments Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education Native American tribal organizations (other than Federally recognized tribal governments) Native American tribal governments (Federally recognized) Private institutions of higher education Independent school districts |
| Additional Information 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 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. |
Additional Information
| Agency Name: | National Institutes of Health |
| 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. |
| Link to Additional Information: | http://grants.nih.gov/grants/guide/pa-files/PAR-22-030.html |
| Grantor Contact Information: | If you have difficulty accessing the full announcement electronically, please contact:
NIH OER Webmaster
OERWebmaster03@od.nih.gov Email:OERWebmaster03@od.nih.gov |
Related Documents
Folder 336476 Full Announcement-PAR-22-030 -> PAR-22-030-Full-Announcement.pdf
Packages
| Agency Contact Information: | NIH OER Webmaster OERWebmaster03@od.nih.gov Email: OERWebmaster03@od.nih.gov |
| Who Can Apply: | Organization Applicants |
| Assistance Listing Number | Competition ID | Competition Title | Opportunity Package ID | Opening Date | Closing Date | Actions |
|---|---|---|---|---|---|---|
| FORMS-G | Use for due dates on or before January 24, 2023 | PKG00270430 | Nov 22, 2021 | Jan 24, 2023 | View | |
| FORMS-F | Use for due dates on or before January 24, 2022 | PKG00270049 | Nov 22, 2021 | Jan 24, 2022 | View | |
| FORMS-H | Use for due dates on or after January 25, 2023 | PKG00277954 | Nov 04, 2022 | Oct 10, 2024 | View |
Package 1
Mandatory forms
336476 RR_SF424_5_0-5.0.pdf
336476 PHS398_CoverPageSupplement_5_0-5.0.pdf
336476 RR_OtherProjectInfo_1_4-1.4.pdf
336476 PerformanceSite_4_0-4.0.pdf
336476 RR_KeyPersonExpanded_4_0-4.0.pdf
336476 PHS398_ResearchPlan_4_0-4.0.pdf
336476 PHSHumanSubjectsAndClinicalTrialsInfo_3_0-3.0.pdf
Optional forms
336476 RR_Budget_3_0-3.0.pdf
336476 RR_SubawardBudget30_3_0-3.0.pdf
336476 PHS398_ModularBudget_1_2-1.2.pdf
336476 PHS_AssignmentRequestForm_3_0-3.0.pdf
Package 2
Mandatory forms
336476 RR_SF424_2_0-2.0.pdf
336476 PHS398_CoverPageSupplement_5_0-5.0.pdf
336476 RR_OtherProjectInfo_1_4-1.4.pdf
336476 PerformanceSite_2_0-2.0.pdf
336476 RR_KeyPersonExpanded_2_0-2.0.pdf
336476 PHS398_ResearchPlan_4_0-4.0.pdf
336476 PHSHumanSubjectsAndClinicalTrialsInfo_2_0-2.0.pdf
Optional forms
336476 RR_Budget_1_4-1.4.pdf
336476 RR_SubawardBudget30_1_4-1.4.pdf
336476 PHS398_ModularBudget_1_2-1.2.pdf
336476 PHS_AssignmentRequestForm_3_0-3.0.pdf
Package 3
Mandatory forms
336476 RR_SF424_5_0-5.0.pdf
336476 PHS398_CoverPageSupplement_5_0-5.0.pdf
336476 RR_OtherProjectInfo_1_4-1.4.pdf
336476 PerformanceSite_4_0-4.0.pdf
336476 RR_KeyPersonExpanded_4_0-4.0.pdf
336476 PHS398_ResearchPlan_5_0-5.0.pdf
336476 PHSHumanSubjectsAndClinicalTrialsInfo_3_0-3.0.pdf
Optional forms
336476 RR_Budget_3_0-3.0.pdf
336476 RR_SubawardBudget30_3_0-3.0.pdf
336476 PHS398_ModularBudget_1_2-1.2.pdf
336476 PHS_AssignmentRequestForm_3_0-3.0.pdf
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