PROJECT SUMMARY / ABSTRACT There is a critical need for investigators to have ready access to a dedicated resource to evaluate new regenerative medicine and gene therapy/somatic cell genome editing applications with nonhuman primates for the treatment of human diseases. This application is focused on meeting this need through the improvement and validation of the rhesus monkey model and related tools and technologies to address new approaches to treat inherited disorders that impact a range of organ systems; and by providing investigators with opportunities to obtain data for new NIH grant applications and for the conduct of investigational new drug (IND)-enabling studies. The potential ramifications of gene transfer/genome editing at any age underscores the importance of rigorous assessments of safety in the rhesus monkey model system which closely recapitulates human physiology. The proposed resource program will be of significant interest to investigators and a range of NIH Institutes as the opportunities will cut across a spectrum of organ systems and diseases. The goals will be accomplished through the following Specific Aims: (1) Enhance tools and technologies for translational gene- based approaches in the rhesus monkey model for utilization by the research community; (2) Improve preclinical xenogeneic models for use by the research community to study human hematopoietic cells in the rhesus host; and (3) Launch the Translational Nonhuman Primate Regenerative Medicine and Gene Therapy/Somatic Cell Genome Editing Resource Program for model validation and therapeutic testing. Our translational team will meet the goals of the RFA by improving and validating the monkey model system for translational research across the lifespan; enhancing biological resources, tools, technologies, and research protocols; and utilizing an established and proven infrastructure that has a successful track record in providing collaborative research opportunities to investigators nationwide. To ensure accessibility for investigators, a call for validation studies will be circulated to launch the resource. The program will provide a pipeline for preclinical and IND-enabling investigations for the development and testing of new treatments for a range of common and rare diseases.

PROJECT NARRATIVE There is a critical need for investigators to have access to a dedicated resource for evaluating new regenerative medicine and gene therapy/somatic cell genome editing approaches with nonhuman primates for the treatment of human diseases. The overarching goals of this application are to provide models, tools, and technologies to address safe and effective treatments across age groups, and ensure opportunities are available to obtain preclinical data that will lead to human clinical trials.