IND-enabling Studies of Somatic Genome Editing Therapeutic Leads (U19, Clinical Trial Not allowed)

This funding opportunity announcement (FOA) is part of a suite of FOAs of the NIH Common Fund Somatic Cell Genome Editing (SCGE) Phase II Program. The goal of this U19 FOA is to support team-based research Projects to accelerate further optimization and development of genome editing-based therapeutic leads towards IND-enabling studies and an IND (Investigational New Drug) application submission to the U.S. Food and Drug Administration (FDA). The proposed leads should demonstrate strong biological rationale and in vitro and/or in vivo proof of concept (POC) data generated in model system(s) each defined by the targeted cell or tissue type to establish a genome editing therapeutic approach that is potentially adaptable to target different pathogenic variants in the same cell or tissue types.
Categories: Health.