Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 – Clinical Trial Optional)

Official funding announcement — preliminary draft

Funder: National Institutes of Health

Opportunity number: PAR-25-327

Official listed status: posted

Closing information: 2027-10-08 (official deadline clock time and time zone must be checked in NOFO)

Award disclosure: Applicant-level ceiling not stated in the retrieved federal synopsis; verify in full NOFO.

Applicant categories in federal synopsis: Public housing authorities/Indian housing authorities; For profit organizations other than small businesses; Public and State controlled institutions of higher education; Private institutions of higher education; Others (see text field entitled "Additional Information on Eligibility" for clarification); Native American tribal governments (Federally recognized); State governments; County governments; Independent school districts; Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education; Small businesses; Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education; City or township governments; Native American tribal organizations (other than Federally recognized tribal governments); Special district governments

Federal synopsis

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.

Draft intake record. Eligibility, program terms, current amendments and applicant ceiling must be independently verified before publishing.

Official Grants.gov opportunity and NOFO