Originally founded as Sangamo BioSciences, Inc. organized around developing new technologies for genome editing, Sangamo Therapeutics, Inc. (NASDAQ:SGMO) is clinical stage biopharmaceutical company, focuses on translating ground-breaking science into genomic therapies that transform patients' lives using platform technologies in genome editing, gene therapy, gene regulation, and cell therapy. The companyÂs proprietary zinc finger DNA-binding protein (ZFP) technology enables specific genome editing and gene regulation. The ZFPs could be engineered to make ZFP nucleases (ZFNs), proteins that could be used to specifically modify DNA sequences by adding or knocking out specific genes; and ZFP transcription factors (ZFP TFs), proteins that can be used to turn genes on or off. Its therapeutic products include SB-728-T, a ZFN-mediated autologous T-cell product for human immunodeficiency virus and acquired immunodeficiency syndrome (HIV/AIDS), which is in Phase II and Phase I clinical trials; and SB-728-HSPC that is in Phase I/II clinical trials for HIV/AIDS. The company also engages in Phase I/II studies of in vivo genome editing applications of ZFP Therapeutics for hemophilia B, Hemophilia A, and Mucopolysaccharidosis I (MPS) and MPS II, which are lysosomal storage disorder (LSD); proprietary preclinical programs in other LSDs; and research stage programs in certain central nervous system disorders and cancer immunotherapies. It has collaborative partnerships with Biogen Inc. to develop therapeutic genome editing products in hemoglobinopathies; and with Shire International GmbH to develop the preclinical development program in HuntingtonÂs disease, as well as license agreement with Sigma-Aldrich Corporation to develop ZFP-based laboratory research reagents and Dow AgroSciences, LLC to modify the genomes or alter protein expression of plant cells, plants, or plant cell cultures.
