Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access (X01, Clinical Trial Not Allowed)
National Institutes of Health (NIH)
Funding is not awarded via the X01 mechanism; access to resources is provided at no cost.
Maximum project period is two years.
To provide investigators access to contract research/medical organizations and subject matter experts within the NINDS URGenT Network.
Support planning, manufacturing, and limited nonclinical therapeutic development efforts.
Investigators working on ultra-rare neurological disorders.
Patients diagnosed with debilitating ultra-rare diseases.
Eligible organization types include public/state controlled institutions of higher education, private institutions of higher education, nonprofits, for-profit organizations, small businesses, local governments, state governments, county governments, city or township governments, special district governments, Indian/Native American tribal governments, and eligible agencies of the federal government.
Non-domestic (non-U.S.) entities are not eligible to apply.
Applications are limited to organizations based in the U.S.
Non-domestic components of U.S. organizations are not eligible.
Projects must focus on gene-based or transcript-directed therapeutics for ultra-rare neurological disorders.
Proposals should include rational proof of concept data for a viable therapeutic candidate.
No specific budget or funding amount is provided as funds are not awarded via the X01 mechanism.
Application deadlines are rolling, with specific due dates listed for each application cycle.
All applications are due by 5:00 PM local time of the applicant organization.
No restrictions on prior grant funding are mentioned.
Applicants must follow the instructions in the Research (R) Instructions in the How to Apply - Application Guide.
A letter of intent is encouraged but not required.
Applications will be evaluated based on scientific and technical merit, including significance, investigator qualifications, innovation, approach, and environment.
Applications will undergo a two-step review process involving evaluation by NIH staff and external experts.
Focus on the clinical rationale, potential patient benefit, and the novelty of the proposed therapeutic candidate.
Access to NINDS contract resources is provided at no cost to the investigators.
Access to resources does not guarantee a successful therapy development plan.
Encourages collaboration with URGenT SMEs for strategic therapeutic development plans.
Projects must demonstrate a clear understanding of the targeted ultra-rare diseases and the proposed therapeutic approaches.
Demonstrating preliminary data supporting the proposed therapeutic candidate is crucial.
Avoid submitting applications that include activities considered non-responsive to the NOFO.
Engage with URGenT SMEs early in the planning process to identify potential issues.
Highlight the innovative aspects of the proposed therapeutic approach and its potential impact on ultra-rare diseases.