Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
The URGenT network offers funding for studies and planning activities that support the development of new gene-based or transcript-directed therapies for ultra-rare neurological or neuromuscular disorders. The aim is to advance promising therapies that have strong biological support and proven effectiveness in relevant model systems, helping them progress towards regulatory approval and the st…
The Ultra-Rare Gene-Based Therapy (URGenT) network supports Investigational New Drug (IND)-enabling studies and planning activities for First-in-Human (FIH) clinical testing of gene-based or transcript-directed therapeutics, such as oligonucleotides and viral-based gene therapies, for ultra-rare…
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