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Ultra-rare Gene-based Therapy (URGenT) Network
The Ultra-rare Gene-based Therapy (URGenT) program will support 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. Altogether, around 7,000 known rare and ultra-rare diseases affect 30 million people in the US. Many are life-threatening and few have FDA-approved treatments. About 45% of rare diseases, including ultra-rare diseases, are neurological disorders, and 90% of rare childhood disorders have major neurological effects. 85% of rare and ultra-rare diseases are single gene disorders, making them excellent candidates for gene therapy.The URGenT program will provide funding and resources to advance gene-based therapies for ultra-rare neurological diseases from late-stage pre-clinical development into first-in-human clinical testing.
The goals of the URGenT program are to:
Accelerate advancement of discoveries into the clinic.
Provide resources and expertise not currently available to applicants.
Deliver therapeutics to patients with ultra-rare neurological diseases.
Standardize and harmonize best practices and protocols for the development of gene-based therapies for ultra-rare diseases.