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:

  1. Accelerate advancement of discoveries into the clinic.
  2. Provide resources and expertise not currently available to applicants.
  3. Deliver therapeutics to patients with ultra-rare neurological diseases.
  4. Standardize and harmonize best practices and protocols for the development of gene-based therapies for ultra-rare diseases.


Projects enter before or after the planning stage, during which access to specialized consultants is available as needed
Overview of the URGenT network - URGenT will support PIs with a lead gene therapy candidate from start-to-finish over a 3-year period. Projects enter before or after the planning stage, during which access to specialized consultants is available as needed. Appropriate contract partners are provided to aid with different stages of therapeutic development (including manufacturing, IND-enabling PK/toxicology studies, and IND submission), and transition to clinical trials. All aspects will be overseen by an external consulting board. Centralized data management ensures facilitative sharing of data, resources and practices with other projects in the network. ADME, absorption, distribution, metabolism and excretion; IND, Investigational New Drug; tox, toxicology.


Pre-Submission Communications with URGenT Program Staff
Please provide preliminary information and supporting data as instructed by the relevant Notice of Funding Opportunity (NOFO) available in this form as you plan to submit your proposal.

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

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

Columbia Awarded $15 Million to Create Medicines for Ultra-Rare Forms of ALS | Columbia University Irving Medical Center

Fetal surgery expert Tippi MacKenzie shifts to in-utero gene therapy (statnews.com)

Resources and Tools


Chris Boshoff, Ph.D.
Program Director

Tjerignimin Silue, Ph.D.
Health Program Specialist

Program Inquiries Contact:

    Funding Opportunities

    URGenT 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.


    URGenT Network Information Session
    April 21, 2022 | 3PM EST


    Related Topics

    URGenT Network Information Session slides(pdf, 977 KB)

    URGenT Subject Matter (SME) Consultants

    URGenT External Consultant Board (ECB) Members