PAR-25-327-Full-Announcement.html

This is a grant funding opportunity announcement (PAR-25-327) from the Department of Health and Human Services National Institutes of Health for the Ultra-Rare Gene-Based Therapy (URGenT) network. The program supports IND-enabling studies and planning activities for First-in-Human clinical testing of gene-based therapeutics for ultra-rare neurological or neuromuscular disorders, with a focus on oligonucleotides and viral-based gene therapies.

The funding is provided through a U01 cooperative agreement mechanism under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853). Eligible applicants include a broad range of organizations: state/local governments, tribal organizations, educational institutions, nonprofits, for-profits, and small businesses. The program aims to accelerate development of clinical candidates with proven biological rationale and proof of concept data toward IND filing and clinical trials, though specific award values are not provided in this excerpt.