Sangamo BioSciences Inc was incorporated in the State of Delaware on June 22, 1995. The Company is a clinical stage biopharmaceutical company engaged in the research, development and commercialization of engineered DNA-binding proteins for therapeutic genome editing and gene regulation. Its proprietary zinc finger DNA-binding proteins (ZFP) technology enables efficient and specific genome editing and gene regulation and it is developing ZFP Therapeutics; novel therapeutic products for the treatment of genetic disease. It has several proprietary clinical and preclinical programs in development and has strategically partnered certain programs with biopharmaceutical companies to obtain funding for its own programs and to expedite clinical and commercial development. The Company and its licensed partners are engaged in the research, development and commercialization of ZFPs, a naturally occurring class of proteins found in humans. It has used its knowledge and expertise to develop a proprietary technology platform in both genome editing and gene regulation. ZFPs can be engineered to make ZFP nucleases (ZFNs), proteins that can be used to specifically modify DNA sequences by adding or knocking out specific genes (genome editing) and ZFP transcription factors (ZFP TFs), proteins that can be used to turn genes on or off (gene regulation). As ZFPs act at the DNA level, it potentially has broad and fundamental applications in several areas, including human therapeutics, plant agriculture and research reagents, including the production of transgenic animals and cell-line engineering. In the process of developing this platform the Company has accrued scientific, manufacturing and regulatory capabilities and know-how that is generally applicable in the broader field of gene therapy. The Company is engaged in the development of novel human therapeutics. Its ZFP Therapeutic, SB-728, a ZFN-modified autologous cell product for the treatment of HIV/AIDS, is the first therapeutic application of its ZFN genome editing technology and is being evaluated in an ongoing Phase 2 study of ZFN-modified T-cells (SB-728-T-1101, Cohort 3) and a Phase 1/2 study of modified hematopoietic stem cells (SB-728mR-HSPC) in HIV-infected subjects. It is also initiating Phase 1/2 studies of in vivo genome editing applications of ZFP Therapeutics for hemophilia B and MPS I, a lysosomal storage disorder (LSD). In addition, it has proprietary preclinical programs in hemophilia A and other LSDs and research stage programs in other monogenic diseases, including certain central nervous system (CNS) disorders and cancer immunotherapy. The Company's competitors are - Protein pharmaceuticals under development at pharmaceutical and biotechnology companies such as Pfizer, Baxter, Bayer, Novo Nordisk, Genzyme, Shire, BioMarin, Biogen, Acceleron and numerous other pharmaceutical and biotechnology firms; Gene therapy companies developing gene-based products in clinical trials. Competitors in this category may include, but not be limited to, uniQure, BioMarin, bluebird bio, RegenX, Spark Therapeutics, Dimension Therapeutics, and Voyager Therapeutics; Cell therapy companies developing cell-based products. Competitors in this category may include Novartis, Adaptimmune, bluebird bio, Cellectis SA, Juno Therapeutics, Kite Pharma and Lion Biotechnologies; Nuclease technologies under development for therapeutic applications of genome modification including companies such as Editas Medicine, CRISPR Therapeutics, Caribou BioSciences and Intellia Therapeutics developing the CRISPR/Cas9 system, Cellectis SA developing TALE nucleases and meganucleases, bluebird developing Homing Endonucleases and MegaTALs and Precision BioSciences, Inc. developing meganucleases.
Benzinga is a fast-growing, dynamic and innovative financial media outlet that empowers investors with high-quality, unique content.