Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
National Institutes of Health (NIH)
No specific budget limits but budgets must reflect actual project needs.
Project duration must not exceed 3 years.
To support IND-enabling studies and planning activities for gene-based therapies targeting ultra-rare neurological and neuromuscular disorders.
To accelerate the development of promising clinical candidates with robust biological rationale and proof of concept data.
Individuals suffering from ultra-rare neurological and neuromuscular disorders.
Research institutions and organizations involved in gene therapy development.
Eligible organization types include higher education institutions, nonprofits, for-profit organizations, small businesses, local, state, and federal governments.
Non-domestic (non-U.S.) entities are not eligible to apply.
U.S. based organizations are eligible; non-domestic components of U.S. organizations are not eligible.
Projects must focus on IND-enabling studies and clinical trial planning for gene-based therapies.
Applicants must provide proof of concept data for the proposed therapeutic approach.
Budgets are not limited but must reflect the actual needs of the proposed project.
Application deadlines are set for various cycles, with the latest due date being October 8, 2027.
Projects are expected to be completed within 3 years.
No specific restrictions on prior funding mentioned.
Applications must include detailed project plans, budgets, and proof of concept data.
Pre-IND meeting minutes with the FDA must be included if available.
Applications will be evaluated based on scientific merit, innovation, and feasibility.
Applications will undergo a peer review process, with feedback provided to applicants.
Priority will be given to applications demonstrating strong proof of concept and feasibility.
The program emphasizes collaboration with NIH program staff and external experts.
High-risk nature of therapeutic development may lead to project attrition.
Projects that align with NIH priorities in rare disease research are encouraged.
Strong emphasis on proof of concept data may disadvantage early-stage projects without substantial data.
Demonstrating robust proof of concept data is crucial for application success.
Avoid submitting applications that lack scientific rigor or do not adhere to submission guidelines.
Engage with NIH staff early in the application process for guidance.
Highlight unique aspects of the proposed therapeutic approach and its potential impact on ultra-rare diseases.