Sangamo Therapeutics (NASDAQ:SGMO) is a clinical-stage biotechnology company headquartered in Brisbane, California, that specializes in the development of genomic therapies based on its proprietary zinc finger nuclease (ZFN) technology. Founded in 1995, Sangamo pioneered ZFN-based genome editing to precisely alter DNA sequences for the treatment of serious genetic and rare diseases. The company’s platform encompasses in vivo genome editing, ex vivo cell therapy, and genome regulation approaches, with a focus on durable therapeutic effects through permanent genetic modification or sustained gene expression control.
Through its genome editing programs, Sangamo is advancing multiple product candidates into clinical trials for conditions such as hemophilia A and B, mucopolysaccharidosis types I and II, and lysosomal storage disorders. The company’s ex vivo programs leverage ZFN technology to engineer patient-derived cells for applications in immuno-oncology and regenerative medicine. Sangamo also develops gene regulation therapeutics using zinc finger transcription factors designed to upregulate or silence disease-related genes without altering the underlying DNA sequence.
Over the years, Sangamo has formed strategic partnerships with leading pharmaceutical and biotechnology firms to expand its research capabilities and accelerate the development of its pipeline. Collaborators have included major global players in hemophilia, metabolic disorders, and neuroscience, supporting clinical development, manufacturing scale-up, and regulatory filings. Sangamo maintains research operations and manufacturing facilities in the San Francisco Bay Area and collaborates with academic and industry partners worldwide.
Sangamo is led by President and Chief Executive Officer Sandy Macrae, under whose guidance the company has continued to innovate in genome engineering and advance its most promising candidates through regulatory milestones and clinical proof-of-concept studies. With a commitment to addressing unmet medical needs, Sangamo Therapeutics leverages its ZFN platform to develop next-generation therapies aimed at transforming the treatment landscape for patients with genetic and rare diseases.