This opportunity supports IND-enabling studies and planning activities for first-in-human clinical testing of gene-based or transcript-directed therapies, including oligonucleotides and viral-based gene therapies, targeting ultra-rare neurological and neuromuscular disorders. The goal is to accelerate promising 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. entities such as nonprofits, small businesses, educational institutions, tribal governments, and various government bodies. Specific eligibility details are provided in the announcement.
