Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 – Clinical Trial Optional)
Can you apply?
This grant is for researchers and institutions advancing gene-based treatments for rare neurological and neuromuscular disorders. Applicants must have institutional support and access to research infrastructure. U.S. academic institutions, research hospitals, and eligible nonprofits may apply.
The program emphasizes translational research moving from laboratory findings toward clinical application. Funding supports multi-year projects that bridge basic science and human trials.
This is a competitive NIH mechanism requiring strong scientific innovation and feasibility plans. Clinical trial components are optional but supported.
This grant is for researchers and institutions advancing gene-based treatments for rare neurological and neuromuscular disorders. Applicants must have institutional support and access to research infrastructure. U.S. academic institutions, research hospitals, and eligible nonprofits may apply.
The program emphasizes translational research moving from laboratory findings toward clinical application. Funding supports multi-year projects that bridge basic science and human trials.
This is a competitive NIH mechanism requiring strong scientific innovation and feasibility plans. Clinical trial components are optional but supported.
Program 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.
Who can apply
Eligible applicants
- 501(c)(3) Public Charity
- City / Municipal Government
- County Government
- Nonprofits
- Private University
- Public Authority
- Public K-12 School
- Public University
- Small Business (SBA-defined)
- Special District
- State Government
- Tribal Nation
- Tribal Organization
Details
This grant is for researchers and institutions advancing gene-based treatments for rare neurological and neuromuscular disorders. Applicants must have institutional support and access to research infrastructure. U.S. academic institutions, research hospitals, and eligible nonprofits may apply.
The program emphasizes translational research moving from laboratory findings toward clinical application. Funding supports multi-year projects that bridge basic science and human trials.
This is a competitive NIH mechanism requiring strong scientific innovation and feasibility plans. Clinical trial components are optional but supported.
How to apply
Application links
Required documents
- SF-424 (R&R) Federal Form
- Project Narrative (specific aims, significance, innovation, approach)
- Detailed Budget and Budget Justification
- Biosketch for Key Personnel (NIH Format)
- Supporting Letters of Commitment
- IRB/IACUC Documentation (if applicable)
Program contact
- 👤 National Institutes of Health
- 📧 grantsinfo@nih.gov
- 📞 301-402-2541
Funding track record
Recent awards under CFDA 93.853 from the last 3 years — real organizations that won funding through this same program.
Top 10 Largest Recent Awards
-
$82,511,281
-
$67,362,785
-
$62,646,087
-
$56,144,651
-
$45,268,737
-
$40,959,789
-
$35,655,349
-
$35,655,116
-
$35,335,145
-
$34,183,297
Top States by Funding
- MA 6 awards $186.5M
- CA 4 awards $129.9M
- OH 4 awards $112.5M
- FL 3 awards $100.3M
- MN 2 awards $99.4M
Source: USAspending.gov — federal spending transparency. Data covers last 3 years.
Funding history
Annual funding for this program — Federal obligations (CFDA 93.853). How funding has trended year over year.
| 2024 | $2,362,835,459 | |
| 2025 | $2,345,500,401 |
FAQ
Who can apply for this grant?
Academic institutions, research hospitals, and eligible nonprofits with research capacity. Applicants must demonstrate institutional support and access to necessary research infrastructure.
What is the typical deadline and project timeline?
Deadlines are annual, typically in October. Projects usually run 3-5 years with milestone-based progress reviews.
What types of research activities are funded?
Translational research on gene therapies for ultra-rare neurological and neuromuscular disorders. Clinical trial components are optional but can strengthen applications.
How competitive is this program?
Very competitive. NIH U01 mechanisms typically fund 10-15% of applications. Strong preliminary data and innovation are essential.
What is the typical funding range?
U01 awards generally range from $400,000 to $600,000 annually, with total project costs varying by scope.
💡 Tips for applicants
- Start with preliminary data showing proof of concept. Reviewers expect solid evidence your approach is feasible.
- Clearly explain the translational path from research to potential clinical use. Show specific milestones.
- Build a strong team with genetics, neurology, and clinical expertise. Collaborative strength matters.
- Address safety and regulatory considerations early. Gene therapy faces scrutiny on these fronts.
- Use your institutional environment section to highlight resources. Access to patient populations and core facilities strengthens competitiveness.
⚠️ Common mistakes
Weak preliminary data or unclear translational pathway to clinical application. Insufficient detail on safety assessment and regulatory strategy. Underestimating timeline or overestimating feasibility of moving to human trials.
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