Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)

Funding Agency:
National Institutes of Health

The Ultra-Rare Gene-Based Therapy (URGenT) network supports Investigational New Drug (IND)-enabling studies and planning activities for First-in-Human (FIH) clinical testing of gene-based or transcript-directed therapeutics, such as oligonucleotides and viral-based gene therapies, for ultra-rare neurological or neuromuscular disorders. The goal of this funding opportunity announcement (FOA) is to accelerate the development of a promising clinical candidate with robust biological rationale and demonstrated proof of concept (POC) data for the intended approach in a model system relevant to a specified patient population towards an IND filing and the initiation of a clinical trial.

Companion FOA: PAR-22-028 , X01 Resource Access Program


  • Letter of Intent Due Date(s): 30 days prior to the application due date.

  • Application Due Dates: Dec. 22, 2021; Feb. 9, 2022; June 9, 2022; Oct. 8, 2022; Feb 8, 2023; June 9, 2023; Oct 10, 2023; Feb 8, 2024; June 7, 2024

PAR-22-030 Expiration Date June 08, 2024




Medical - Basic Science
Medical - Clinical Science
Medical - Translational

External Deadline

February 8, 2023