Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access (X01, Clinical Trial Not Allowed)
This NINDS X01 gives investigators no-cost access to URGenT CRO/CMO resources and subject-matter experts to support planning, manufacturing, and limited nonclinical development of gene- or transcript-directed therapies for ultra-rare neurological and neuromuscular diseases (clinical trials not allowed). Awardees receive a multidisciplinary project team to develop IND-enabling activities, prepa…
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