Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
NIH cooperative agreements fund IND-enabling studies and planning for first-in-human gene-based or transcript-directed therapies aimed at ultra-rare neurological or neuromuscular disorders.
⚑ Clinical Trial Optional · Supports IND-enabling studies and planning toward first-in-human clinical testing; cooperative agreement mechanism · Foreign organizations are listed as eligible, but non-domestic components of U.S. organizations are not eligible · No award ceiling provided in notice
Unit fits — one characterization, each unit's own rules
| Physical Sciences & Engineering (demo) | 65 good | technical depth: minor; funds applied research |
| IPPRA | 45 partial | peripheral portfolio topic: public_health; social/behavioral work is none; funds applied research; biomedical core — IPPRA health lane is communication/crisis/policy (capped); clinical-trial/biomedical core — IPPRA angle is policy/community (capped); capped at 45 (limited social-science role) |
| Tom Love Innovation Hub | 45 partial | funds applied research; prototyping/demonstration stage; deep-tech content |
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
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.
Apply
View on Grants.gov → CONTACT: National Institutes of Health <grantsinfo@nih.gov>
Proposal brief SEE AN EXAMPLE →
A one-page internal memo: fit assessment, submission requirements, document scaffold, and next steps dated back from the deadline — tailored to your project idea if you add one.
Proposal shell · National Institutes of Health conventions SEE AN NIH EXAMPLE →
Funder-faithful document skeletons — National Institutes of Health's document set with section headings, page limits, reviewer guidance, and writing prompts; add a project idea to get [DRAFT] starter bullets. Download as .md for Word or Overleaf.