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.
Resources and Tools
Contacts
Chris Boshoff, Ph.D.
Program Director
chris.boshoff@nih.gov
Tjerignimin Silue, Ph.D.
Health Program Specialist
tjerignimin.silue@nih.gov
Program Inquiries Contact:
URGenTMailbox@ninds.nih.gov
Funding Opportunities
PAR-22-030: Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
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.
Events
URGenT Network Information Session
April 21, 2022 | 3PM EST
Related Topics
URGenT Network Information Session slides(pdf, 977 KB)