Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders
Funds IND-enabling studies and planning that take a gene-based therapy for an ultra-rare neurological or neuromuscular disorder toward its first human trial.
An ultra-rare neurological or neuromuscular disease rarely has the patient numbers to attract a company's own development budget on its own. This network exists to carry a promising candidate to human testing anyway.
What is funded is Investigational New Drug-enabling studies and planning activities for First-in-Human clinical testing of gene-based or transcript-directed therapeutics — oligonucle…
Investigational New Drug-enabling studies and First-in-Human planning for gene-based or transcript-directed therapeutics — including oligonucleotides and viral-based gene therapies — for ultra-rare neurological or neuromuscular disorders, requiring a robust biological rationale and demonstrated proof-of-concept data in a relevant model system. Clinical trials are optional.
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