Sangamo is a clinical stage biopharmaceutical company focused on the research, development and commercialization of engineered DNA-binding proteins for the development of novel therapeutic strategies for unmet medical needs.
We are worldwide leaders in the development of a proprietary technology platform that enables specific regulation of gene expression and gene modification. The basis of this platform is a naturally occurring class of transcription factors, zinc finger DNA-binding proteins (ZFPs) which we can engineer to drive desired therapeutic outcomes. Engineered ZFPs can be linked to functional domains that normally activate or repress gene expression to create ZFP transcription factors (ZFP TFs) capable of turning genes on or off. We can also link ZFPs to nuclease domains to create zinc finger nucleases (ZFNs) which enable precise gene-editing in cells. Engineered ZFNs can modify a cell's DNA at a precise location, thereby facilitating correction or disruption of a specific gene or the targeted addition of a new DNA sequence.
A New Class of Therapeutics
Our primary mission is to develop ZFP Therapeutics®. We have ongoing clinical programs to evaluate ZFP TFs and ZFNs as novel approaches to unmet medical needs where we believe we have a differential technical advantage to impact the outcome of disease by functioning at the DNA level.
Our Business Strategy
Our business strategy is to do what we do best — to develop highly specific ZFP TFs and ZFNs through early stage clinical testing and strategically partner with biopharmaceutical companies at key value inflection points to execute late-stage clinical trials and commercial development.
Our ZFP technology can be broadly applied to the development of novel human therapeutics. We have proprietary programs in HIV/AIDs and monogenic diseases that we are developing internally. With positive clinical data we intend to work with partners to develop programs through late-stage clinical trials. We have a preclinical pipeline that is focused in monogenic and rare diseases and neurologic indications. In early 2012, we established a partnership with Shire International GmbH (formerly Shire AG) to develop ZFP Therapeutics for hemophilia, Huntington's disease and other monogenic diseases. In January 2014, we established a partnership with Biogen Idec to develop a ZFP Therapeutic approach for hemoglobinopathies, such as beta-thalassemia and sickle cell disease.
As Sangamo's internal therapeutic development programs progress, we will continue our strategy of balancing collaborative development programs with retention of ownership of select assets that fit our strategic vision and intend to pursue late-stage development and commercialization in order to become a fully integrated biopharmaceutical company.
We have successfully partnered non-therapeutic applications of our ZFP technology and have out-licensed applications in high value laboratory research reagents, transgenic animals and commercial production cell lines to Sigma-Aldrich Corporation, and in plant agriculture to Dow AgroSciences LLC, a subsidiary of Dow Chemical Corporation.
Sigma is developing and marketing ZFN-derived gene editing tools under the trademark CompoZr®, ZFP-modified cell lines for commercial production of protein pharmaceuticals through its SAFC® business unit, and ZFP-engineered transgenic animals through its SAGE™ Labs business unit.
Dow AgroSciences has the exclusive right to use Sangamo’s ZFP technology in plants and is marketing ZFP-derived plant products under the trademark EXZACT™ Precision Technology.
Sangamo was founded in 1995, went public in 2000, and trades on the Nasdaq Global Market (SGMO). We are located in Richmond, California, in the Bay Area, the birthplace of biotechnology. We are intimately involved in our community through partnerships with academic institutions, relationships with non-profit organizations and ties to advocacy organizations aligned with improving the quality of life for patients with unmet medical needs within our strategic vision.