Sangamo is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for various monogenic and infectious diseases with unmet medical needs. Our therapeutic products are based on our engineered zinc finger DNA-binding protein (ZFP) genome editing and adeno-associated virus (AAV) gene therapy platforms.
We are worldwide leaders in the fields of gene therapy and genome editing. Our AAV-based gene therapy platform uses our proprietary vector constructs to enable the delivery and potent expression of any therapeutic gene. Our proprietary ZFP technology platform enables highly specific genome modification and gene regulation. ZFPs are a naturally occurring class of transcription factors, which we can engineer to bind to any DNA sequence with singular specificity and drive desired therapeutic outcomes.
Our 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 endonuclease domains to create zinc finger nucleases (ZFNs) which enable precise genome editing in cells. Sangamo's engineered ZFNs can modify a cell's DNA at a precise location, thereby facilitating the correction or disruption of a specific gene or the targeted addition of a new DNA sequence, without the unwanted consequences of off-target DNA binding activity.
To learn more about our AAV and ZFP technology platforms and their applications, click here.
Sangamo was founded in 1995, went public in 2000, and trades on the Nasdaq Global Market (Ticker: 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.
Our Business Strategy
Our business strategy is to do what we do best — to develop our AAV gene therapy and ZFP platforms 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.
We have a partnership with Shire International GmbH (formerly Shire AG) to develop ZFP Therapeutics for Huntington's disease and one other gene target. We also have a collaboration with Biogen Inc. (formerly Biogen Idec Inc) to develop a ZFP Therapeutic approach for hemoglobinopathies, 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.
To learn more about our existing partnerships or to inquire about partnership opportunities with Sangamo, see our Partnerships page.