Ultra-Rare Gene-Based Therapy Development for Neurological and Neuromuscular Disorders
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This opportunity supports the Ultra-Rare Gene-Based Therapy (URGenT) network, focused on advancing gene-based or transcript-directed therapeutics for ultra-rare neurological or neuromuscular disorders. The primary goal is to accelerate the development of promising clinical candidates towards an Investigational New Drug (IND) filing and the initiation of a clinical trial. This includes supporting Investigational New Drug (IND)-enabling studies and planning activities for First-in-Human (FIH) clinical testing. The scope encompasses therapeutics such as oligonucleotides and viral-based gene therapies. Applicants should demonstrate a robust biological rationale and proof of concept (POC) data in a relevant model system for the specified patient population. The funding instrument is a cooperative agreement, and the relevant Assistance Listing is 93.853 (Extramural Research Programs in the Neurosciences and Neurological Disorders).
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