URGenT Resource Access (X01): Nonclinical & Manufacturing Support for Ultra‑Rare Gene Therapies (No Clinical Trials)
This NINDS X01 gives investigators no-cost access to URGenT CRO/CMO resources and subject-matter experts to support planning, manufacturing, and limited nonclinical development of gene- or transcript-directed therapies for ultra-rare neurological and neuromuscular diseases (clinical trials not allowed). Awardees receive a multidisciplinary project team to develop IND-enabling activities, prepa…
Background
According to the definition created by Congress in the Orphan Drug Act of 1983 and adopted by the FDA, a rare disease is a condition that affects fewer than 200,000 people in the United States. Ultra-rare diseases affect substantially fewer people, less than or equal to 6,000; …
Funding Source
Eligibility
- Applicant eligibility is restricted to organizations that administer an eligible parent award; this NOFO provides administrative supplements to existing awards.
- Foreign/Non-domestic entities are NOT eligible to apply. Non-domestic (non-U.S.) components of U.S. organizations are NO…
Dates
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