Opportunity Information: Apply for PAR 25 327
The NIH funding opportunity PAR-25-327 is a cooperative agreement (U01, clinical trial optional) focused on speeding up translation of gene-based and transcript-directed therapies for ultra-rare neurological and neuromuscular disorders. It sits within the Ultra-Rare Gene-Based Therapy (URGenT) network and is specifically aimed at taking a therapy that already looks genuinely promising and pushing it through the remaining preclinical and operational steps needed to reach an Investigational New Drug (IND) application and begin a first-in-human (FIH) clinical study. In practical terms, this is meant for projects that have moved beyond early discovery and now need a structured, milestone-driven path to IND-enabling studies and clinical trial readiness.
The scientific scope centers on gene-based or transcript-directed therapeutic modalities, with examples including oligonucleotide approaches and viral-based gene therapies. The emphasis is on ultra-rare disorders affecting the nervous system or neuromuscular function, where traditional commercial development pathways are often difficult due to small patient populations. To fit the intent of the program, an application should be anchored in a well-supported biological rationale for the target disease and mechanism, and it should already have proof-of-concept (POC) evidence for the proposed therapeutic strategy in a model system that is relevant to the intended patient population. The core expectation is that the proposed work will materially de-risk and advance a defined clinical candidate toward IND submission, rather than exploring broad platform concepts without a clear, near-term candidate and development plan.
Because the mechanism is a cooperative agreement, NIH involvement is typically more active than in a standard research grant, with an expectation of coordinated planning, progress tracking, and alignment with network goals. The opportunity explicitly supports IND-enabling studies and planning activities required for FIH testing, meaning projects should be framed around the kinds of deliverables regulators expect before human dosing (for example, the preclinical evidence package, readiness for manufacturing and quality considerations, and clinical planning). While clinical trials are listed as optional under the U01, the stated program goal is to move projects to the point where a clinical trial can be initiated, so strong applications generally align their timelines and milestones with that end point.
Eligibility is broad across public and private sectors. Eligible applicants include state, county, and local governments; special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; federally recognized Native American tribal governments; tribal organizations (other than federally recognized tribal governments); public housing authorities/Indian housing authorities; nonprofits with or without 501(c)(3) status (other than institutions of higher education); for-profit organizations (other than small businesses); small businesses; and other organizations. The announcement also calls out additional eligible applicant types such as Alaska Native and Native Hawaiian Serving Institutions, AANAPISIs, Hispanic-serving Institutions, HBCUs, Tribally Controlled Colleges and Universities, faith-based or community-based organizations, regional organizations, eligible federal agencies, tribal governments that are not federally recognized, and U.S. territories or possessions.
Foreign eligibility is constrained. Non-domestic (non-U.S.) entities are not eligible to apply, and non-domestic components of U.S. organizations are not eligible to apply. However, foreign components are allowed when they meet the NIH Grants Policy Statement definition, which generally means a U.S.-based applicant organization can include a justified foreign component in the project when it is necessary for the project and offers special expertise, resources, populations, or other unique capabilities not readily available in the United States.
Administratively, the sponsoring agency is the National Institutes of Health, the opportunity is categorized as discretionary funding, the activity category is health, and the CFDA (Assistance Listing) number provided is 93.853. The opportunity was created on 2024-12-18, and the original closing date listed is 2027-10-08. An award ceiling and expected number of awards are not specified in the provided source text, which usually signals that applicants should consult the full notice for budget guidance, project period expectations, and any institute-specific limits or milestones tied to URGenT network participation.
Overall, this opportunity is best understood as a translational bridge for ultra-rare neuro and neuromuscular gene-based therapy programs that already have convincing early evidence and now need coordinated support to complete IND-enabling work and be ready to launch a first-in-human trial.Apply for PAR 25 327
- The National Institutes of Health in the health sector is offering a public funding opportunity titled "Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)" and is now available to receive applicants.
- Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.853.
- This funding opportunity was created on 2024-12-18.
- Applicants must submit their applications by 2027-10-08.
- Eligible applicants include: State governments, County governments, City or township governments, Special district governments, Independent school districts, Public and State controlled institutions of higher education, Native American tribal governments (Federally recognized), Public housing authorities/Indian housing authorities, Native American tribal organizations (other than Federally recognized tribal governments), Nonprofits having a 501 (c) (3) status with the IRS, other than institutions of higher education, Nonprofits that do not have a 501 (c) (3) status with the IRS, other than institutions of higher education, Private institutions of higher education, For-profit organizations other than small businesses, Small businesses, Others.
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FAQs: NIH PAR-25-327 (URGenT Network) Cooperative Agreement (U01, Clinical Trial Optional)
What is PAR-25-327?
PAR-25-327 is a National Institutes of Health (NIH) funding opportunity that uses a cooperative agreement mechanism (U01; clinical trial optional). It is designed to speed the translation of gene-based and transcript-directed therapies for ultra-rare neurological and neuromuscular disorders by supporting the remaining preclinical and operational steps needed to reach an Investigational New Drug (IND) application and begin a first-in-human (FIH) clinical study.
What is the overall purpose of this funding opportunity?
The purpose is to take a therapy that already appears genuinely promising and advance it through a structured, milestone-driven path toward IND submission and FIH clinical trial readiness. It is intended as a translational bridge for programs that have moved beyond early discovery and now need IND-enabling work and clinical planning to initiate a first-in-human study.
What kinds of diseases are in scope?
The scope is ultra-rare disorders that affect the nervous system or neuromuscular function. The opportunity emphasizes conditions where traditional commercial development is challenging due to very small patient populations.
What therapeutic approaches are in scope?
The scientific scope centers on gene-based or transcript-directed therapeutic modalities. Examples mentioned include oligonucleotide approaches and viral-based gene therapies.
Is this opportunity meant for early discovery research?
No. The intent is not to explore broad platform concepts without a defined, near-term clinical candidate and development plan. Applications are expected to focus on work that will materially de-risk and advance a defined clinical candidate toward IND submission.
What level of existing evidence is expected before applying?
An application should be anchored in a well-supported biological rationale for the target disease and mechanism and should already have proof-of-concept (POC) evidence for the proposed therapeutic strategy in a model system relevant to the intended patient population.
What does "IND-enabling" mean in the context of this opportunity?
Within the provided description, IND-enabling refers to the kinds of preclinical and operational work products regulators typically expect before initiating human dosing. This includes building the preclinical evidence package and being ready for manufacturing and quality considerations, along with clinical planning needed to support an IND application and begin a first-in-human study.
Are clinical trials required under this funding opportunity?
Clinical trials are listed as optional under the U01 mechanism. However, the stated program goal is to move projects to the point where a clinical trial can be initiated, so strong applications generally align timelines and milestones with readiness to begin first-in-human testing.
What is the URGenT network and how does it relate to PAR-25-327?
This opportunity sits within the Ultra-Rare Gene-Based Therapy (URGenT) network. The program description emphasizes coordinated planning, progress tracking, and alignment with network goals as part of the cooperative agreement approach.
What does it mean that this is a "cooperative agreement" (U01)?
A cooperative agreement typically involves more active NIH involvement than a standard research grant. The description highlights expectations for coordinated planning, progress tracking, and alignment with the URGenT network goals, reflecting a milestone-driven approach.
Who is eligible to apply?
Eligibility is broad across public and private sectors. Eligible applicants include:
- State, county, and local governments
- Special district governments
- Independent school districts
- Public and state-controlled institutions of higher education
- Private institutions of higher education
- Federally recognized Native American tribal governments
- Tribal organizations (other than federally recognized tribal governments)
- Public housing authorities/Indian housing authorities
- Nonprofits with or without 501(c)(3) status (other than institutions of higher education)
- For-profit organizations (other than small businesses)
- Small businesses
- Other organizations
Are any additional applicant types specifically called out as eligible?
Yes. The announcement also calls out the following as eligible applicant types:
- Alaska Native and Native Hawaiian Serving Institutions
- Asian American Native American Pacific Islander Serving Institutions (AANAPISIs)
- Hispanic-serving Institutions
- Historically Black Colleges and Universities (HBCUs)
- Tribally Controlled Colleges and Universities
- Faith-based or community-based organizations
- Regional organizations
- Eligible federal agencies
- Tribal governments that are not federally recognized
- U.S. territories or possessions
Can non-U.S. (foreign) organizations apply directly?
No. Non-domestic (non-U.S.) entities are not eligible to apply.
Can a U.S. organization include a non-U.S. component?
Non-domestic components of U.S. organizations are not eligible to apply. However, foreign components are allowed when they meet the NIH Grants Policy Statement definition, generally meaning the U.S.-based applicant can include a justified foreign component when it is necessary for the project and offers special expertise, resources, populations, or other unique capabilities not readily available in the United States.
Which agency is sponsoring this opportunity?
The sponsoring agency is the National Institutes of Health (NIH).
How is this opportunity categorized administratively?
Based on the provided information, the opportunity is categorized as discretionary funding, with an activity category of health.
What is the Assistance Listing (CFDA) number?
The Assistance Listing (CFDA) number provided is 93.853.
When was this opportunity created?
The opportunity was created on 2024-12-18.
What is the listed closing date?
The original closing date listed is 2027-10-08.
Is an award ceiling provided?
No award ceiling is specified in the provided text.
Is the expected number of awards provided?
No. The expected number of awards is not specified in the provided text.
What does it mean that budget limits and award counts are not specified?
In the provided description, the absence of an award ceiling and expected number of awards is presented as a signal that applicants may need to consult the full notice for budget guidance, project period expectations, and any institute-specific limits or milestones tied to URGenT network participation.
What type of project is this opportunity best suited for?
This opportunity is best suited for translational programs in ultra-rare neurological or neuromuscular disorders where there is convincing early evidence and a clear plan to complete IND-enabling studies and operational readiness steps needed to submit an IND and initiate a first-in-human clinical study.
What is a common misalignment with the program intent?
A common misalignment, based on the description, would be proposing broad platform development or exploratory work without a defined clinical candidate and a near-term, milestone-driven development plan aimed at IND submission and first-in-human readiness.
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