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.
New Clinical Research Opportunity Announcements within NeuroNext
URGenT is pleased to announce two new Research Opportunity Announcements (ROAs) to support Stage 1 Preliminary and Stage 2 Protocol applications for clinical trials of gene-based and gene-targeted therapies for ultra-rare neurological diseases. The URGenT Clinical Trials program will utilize a two-stage application and review process for therapeutics that have an active Investigational New Drug (IND) designation. Stage 1 applications (OTA-24-011(pdf, 283 KB)(pdf, 283 KB)) will include scientific background information supporting the gene-therapy product/therapeutic and proposed clinical trial. Stage 2 Protocol Applications (OTA-24-012(pdf, 381 KB)(pdf, 381 KB)) will be accepted by invitation only and will include the submission of more detailed information on the proposed clinical trial and related activities. Funded studies will be conducted within the Network of Excellence in Neuroscience Clinical Trials (NeuroNEXT). Potential applicants are STRONGLY encouraged to contact NeuroNEXT/URGenT Program Staff to discuss their application and the application process prior to submission.
Prospective applicant contacts NINDS/DCR to discuss the proposed trial:
Sophie Cho, M.D.
Program Director, Division of Clinical Research
hyunjoo.cho@nih.gov
News & Publications
Next-generation strategies for gene-targeted therapies of central nervous system disorders: A workshop summary
ScienceDirect, December 2021
Personalized medicine is having its day | Nature Biotechnology
NINDS launches network to develop treatments for ultra-rare neurological diseases
November 4, 2021
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)