The National Institute of Neurological Disorders and Stroke (NINDS) recently created the Ultra-Rare Gene-Based Therapy (URGenT) Network. The URGenT Network supports the development of state-of-the-art gene-based therapies for ultra-rare neurological diseases, which affect as few or fewer than one in fifty thousand people. It 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 neurological or neuromuscular disorders.
It provides funding and resources (X01 and U01) to advance gene-based therapies for ultra-rare neurological diseases from late-stage pre-clinical development into first-in-human clinical testing, to accelerate the development of a promising clinical candidate with robust biological rationale and to demonstrate proof of concept (POC) data for the intended approach in a model system relevant to a specified patient population towards an IND filing and the initiation of a clinical trial.
With future receipt dates approaching, please feel free to contact NINDS with any questions regarding the program and any interest in submitting an application.