Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access (X01)

NOTE: This funding opportunity expired March 12, 2026. Limited case-by-case submissions may be accepted; contact the eRA Service Desk.

Research Focus

This program supports investigators developing gene-based and transcript-directed therapeutics for ultra-rare neurological and neuromuscular disorders (affecting ≤6,000 people in the U.S.). The URGenT network provides access to contract research organizations (CROs), contract manufacturing organizations (CMOs), and subject matter experts (SMEs) to accelerate precision medicine approaches. Supported activities include manufacturing, nonclinical toxicology testing, and other IND-enabling activities—but clinical trials are not allowed under this mechanism. The goal is to generate data sufficient for a pre-Investigational New Drug (pre-IND) meeting with the FDA and eventual IND submission, addressing the critical unmet need in ultra-rare disease communities where ~95% lack FDA-approved therapeutics.

Eligible therapeutic modalities include oligonucleotide-based approaches (antisense oligonucleotides, siRNA, shRNA), viral vector-based approaches (adeno-associated viruses), cell therapy-based approaches (gene-modified ex vivo), genome editing platforms (CRISPR, ZFNs, TALENs), and RNA-targeted small molecules. Applicants work with URGenT SMEs to identify gaps in existing data and protocols before framing focused FDA discussions.

At a Glance

• Who can apply: Extramural researchers funded by NINDS; clinical trials not permitted under this award
• Funding & project length: Not stated; in-kind contract access at no cost to investigators
• Award mechanism: X01 Resource Access Award
• Key dates: Expired March 12, 2026 (original expiration February 1, 2028); rolling submission windows through January 31, 2027 (case-by-case review only now)
• Best fit for: Neuroscience, genetic medicine, and translational researchers preparing IND packages for ultra-rare gene therapies; planning and nonclinical development phases