Sangamo Therapeutics

Sangamo Therapeutics is a genomic medicine company conducting R&D across four distinct but complementary technology platforms — gene therapy, cell therapy, in vivo genome editing, and in vivo genome regulation. The inherent selectivity and diversity of Sangamo's technology platforms enable us to design therapeutic approaches to address the underlying genetic causes of diseases, utilizing whichever technology is best suited to deliver such treatment. As our technology platforms are interconnected, we are applying insights from research to optimize each individual platform and uncover future R&D pathways. Sangamo is best known for developing our proprietary gene editing technology, zinc finger nucleases (ZFNs). Our technical team continues to optimize the three key dimensions of ZFN genome editing: precision, efficiency, and specificity. Ex vivo gene-edited cell therapy represents the most direct application of ZFN technology, building on early experience in HIV clinical studies and expanding into hematology and oncology. Our cell therapy portfolio also includes ex vivo engineered regulatory T cells (CAR-Tregs) for autoimmune and inflammatory conditions. With deeper understanding of ZFN molecular biology, cassette engineering, and in vivo delivery, our gene therapy programs utilize AAV and are making clinical progress. Zinc finger protein engineering and AAV delivery also underpin our zinc finger protein transcription factor (ZFP-TF) genome regulation technology, which we are applying to CNS diseases.