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

This grant supports IND-enabling studies and planning for first-in-human testing of gene-based or transcript-directed therapeutics, such as oligonucleotides and viral gene therapies, aimed at ultra-rare neurological or neuromuscular disorders. It funds work that accelerates development of candidates with strong biological rationale and proof-of-concept data toward IND filing and clinical trial initiation.