Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access (X01, Clinical Trial Not Allowed)
This grant provides access to specialized resources and expertise for researchers developing gene-based therapies for extremely rare neurological disorders, helping them prepare for regulatory submissions and advance their therapeutic projects.
Description
The purpose of this FOA is to provide investigators with a mechanism to access contract research/medical organizations (CROs/CMOs) and subject matter experts (SMEs) within the NINDS Ultra-Rare Gene-based Therapy (URGenT) Network to support planning, manufacturing, and limited nonclinical therapeutic development efforts.