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A - Viral Vector Development -> AAV Vectors – Clinical/Non-Human Primate Studies

1545: Translational Programs in Gene Therapy and Somatic Cell Genome Editing: Nonhuman Primate Research Opportunities

Type: Poster Session

Poster Board Number: 1545
Presentation Details
Session Title: Friday Poster Session
Start Time: 5/19/2023 12:00
End Time: 5/19/2023 14:00

Clinical translation of emerging gene therapy and somatic cell genome editing strategies for the treatment of a range of human diseases requires access to nonhuman primate models. Nonhuman primates represent a critical bridge for translational applications because they can provide a path to understand human biological functions, study complex human diseases, and address the safety of new diagnostics and therapies proposed for human use across the lifespan. Of primary concern is ensuring safe and efficient editing, which represents the two most fundamental barriers to human clinical use. The Nonhuman Primate Testing Center for Evaluation of Somatic Cell Genome Editing Tools was established in 2019 to develop safe and efficient genome editing methods to treat patients with common or rare diseases. The Testing Center supports a range of NIH-funded projects targeting a variety of organ systems (e.g., brain/CNS, heart, skeletal muscle, lung, liver, kidney, hematopoietic system) through an efficient collaborative process, and is a Large Animal Testing Center in the NIH Somatic Cell Genome Editing (SCGE) Consortium. The SCGE Consortium includes 72 investigators, 45 projects, and 38 institutions across the U.S. focused on developing targeted systems for the delivery of new editors and improved human genome editing tools, and exploring new methods to assess unintended biological effects. A significant concern for the clinical use of gene editors is the potential for immune responses and pre-existing immunity. The Testing Center has a range of capabilities including total-body positron emission tomography (PET) imaging to identify edited cells and assess inflammation and T-cell trafficking. Detection of T-cell reactivity in blood may not accurately reflect local immunologic events at the target site that can reduce efficacy or result in toxicity. In the context of future human gene-editing trials, it will be important to understand how persistent gene expression leads to immune responses and the consequences of these responses for transduced/edited cells. The Testing Center collaborates with members of the SCGE Consortium through their NIH-funded grants and Collaborative Opportunity Fund projects to address these and related questions in nonhuman primate models from early prenatal stages to infants, juveniles, and adults. The Nonhuman Primate Testing Center for Evaluation of Somatic Cell Genome Editing Tools ensures contributions will have a sustained, powerful impact on new treatments for patients across all age groups, including the youngest patients in need.

Alice F. Tarantal1, Michele Martinez1, Lionel Sanz1, David J. Segal2, Dennis J. Hartigan-O'Connor3

1Pediatrics, Cell Biology and Human Anatomy, and California National Primate Research Center, University of California, Davis, Davis, CA,2Biochemistry and Molecular Medicine, and Genome Center, University of California, Davis, Davis, CA,3Medical Microbiology and Immunology, and California National Primate Research Center, University of California, Davis, Davis, CA
 A.F. Tarantal: None.

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