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.
Pre-submission Communication form(pdf, 267 KB)
Resources and Tools
PAR-22-028: Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access (X01, Clinical Trial Not Allowed)
All applicants are strongly encouraged to contact URGenT program staff prior to submitting an application.
News & Events
URGenT Network Information Session
April 21, 2022 | 3PM EST
URGenT Network Information Session slides(pdf, 977 KB)