Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
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.
Eligibility
See official grant page for eligibility requirements
Industry Tags
About This Grant
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 Information
Eligibility Overview
This opportunity from National Institutes of Health is open to eligible applicants nationwide. Organizations working in Healthcare are especially encouraged to review the requirements.
Federal grant opportunities like this one are typically open to a range of applicant types. Common eligible organizations include:
- Nonprofits and 501(c)(3) organizations
- Small businesses and startups (especially for SBIR/STTR programs)
- State, local, and tribal governments
- Colleges, universities, and research institutions
- Individuals (for select programs in education, arts, and research)
Eligibility requirements vary by opportunity. Always review the official listing before investing time in an application.
How to Apply
- 1
Review the full opportunity
Read the complete Notice of Funding Opportunity (NOFO) for this National Institutes of Health program on its official page (https://www.grants.gov/search-results-detail/357817).
- 2
Confirm eligibility
Check that your organization meets every requirement set by National Institutes of Health — applicant type, location, registration status, and any prior-award restrictions.
- 3
Prepare required documents
Gather your budget narrative, needs statement, organizational details, SAM.gov registration, and any required certifications.
- 4
Submit before the deadline
This opportunity closes on October 8, 2027. Submit at least 48 hours early — portal systems are often slow near closing time.
Grant Writing Tips
Proposals for healthcare programs such as those from National Institutes of Health are competitive — these tips can strengthen your application:
- ✓Start your application at least 4 weeks before the deadline — rushed proposals score lower.
- ✓Tailor your needs statement to match the funder's stated priorities, using their exact language where possible.
- ✓Have a colleague outside your team review your budget narrative before submitting — fresh eyes catch errors reviewers penalize.
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Frequently Asked Questions
Who is eligible for Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)?
This opportunity from National Institutes of Health is open to eligible applicants nationwide. Organizations working in Healthcare are especially encouraged to review the requirements. Always confirm the full eligibility criteria on the official listing before applying.
How much funding does Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) provide?
A specific award amount is not listed for Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional). See the official grant page for funding details.
When is the deadline for Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)?
The application deadline for Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) is October 8, 2027. Submit at least 48 hours early to avoid last-minute portal issues.
How do I apply for Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)?
Apply for Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) through the official grant page (https://www.grants.gov/search-results-detail/357817). Confirm your eligibility and prepare your application materials before the deadline.
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