This funding opportunity supports IND-enabling studies and planning activities for first-in-human clinical testing of gene-based or transcript-directed therapeutics, including oligonucleotides and viral-based gene therapies. It targets ultra-rare neurological and neuromuscular disorders, aiming to accelerate development of clinical candidates with strong biological rationale and proof of concept data toward IND filing and clinical trial initiation.
Eligible applicants include a wide range of U.S. government entities, nonprofits, educational institutions, small businesses, tribal organizations, and other specified organizations. Non-domestic entities and foreign components of U.S. organizations are not eligible to apply, though foreign componen
