Ultra-rare Gene-based Therapy (URGenT) Network

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.

URGenT will support PIs with a lead gene therapy candidate from start-to-finish over a 3-year period. Through a phased program with multiple entry points, URGenT will provide support beginning at the planning phase and moving into a pre-clinical therapeutic development phase to include manufacturing, IND-enabling PK/toxicology studies, and IND submission, and finally into an early clinical trials phase. The URGenT program anticipates an initial application deadline of November 2021, starting with the pre-clinical phase.