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 Notice of Funding Opportunity (NOFO) 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.
Companion FOA: PAR-25-326 , X01 Resource Access Program
Deadlines:
- Letter of Intent Due Date(s): 30 days prior to the application due date.
- Application Due Dates: Feb 10, 2025; June 9, 2025; Oct 9, 2025; Feb 9, 2026; June 9, 2026; Oct 9, 2026; Feb 9, 2027; June 9, 2027; Oct 8, 2027
PAR-25-327 Expiration Date October 09, 2027