IND-enabling Studies for Platform Clinical Trials of Genome Editing in Multiple Diseases (U01 Clinical Trial Not Allowed)

Funding Opportunity RFA-RM-24-001 from the NIH Guide for Grants and Contracts. The purpose of this NOFO is to provide support for applications for IND-enabling studies for the development of a novel in vivo genome editing therapeutic platform (genome editor plus delivery system) for two or more disease indications, using the same genome editor, route of administration, and delivery system. Through this initiative, NIH aims to explore the extent to which the use of a therapeutic genome editing platform can streamline the regulatory path for multiple disease indications and to disseminate this regulatory information (and supporting documentation) to the scientific community.