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Main focus: Gene editing using zing-finger nucleases for novel gene-ediing and cell therapies.
Company stage: Clinical
Diseases: Solid graft transplantations, beta-thalassemia, sickle cell disease, inflammatory bowel disease, multiple sclerosis, solid tumours, haematological cancers
Genome editing technology: Zinc finger nucleases (ZNFs)
Funding stage: Public (NASDAQ:SGMO)
Location: South San Francisco, CA, USA
Sangamo Therapeutics is the only company solely focused on the development of zinc finger nucleases (ZFNs) as a tool for genetic engineering. The company applies the tool for both ex vivo and in vivo-based gene-editing therapies, as well as a tool for genome regulation. The company is currently sponsoring clinical trials for sickle cell disease and beta-thalassemia, where ZFNs are applied for gene editing. The company holds multiple substantial collaborations with companies such Pfizer, Biogen, Novartis, Takeda, Sanofi and Kite Pharma.