Cell Therapy :: Sangamo Therapeutics, Inc. (SGMO)

Sangamo has significant experience in process development and manufacturing of modified cell therapy products gained through its T-cell and hematopoietic stem cell (HSC) programs in HIV, which were the first genome editing products to enter human clinical trials. In collaboration with Bioverativ we are also developing modified HSC treatments for beta-thalassemia and sickle cell disease. Cells are removed from the body and undergo ZFN-mediated genome editing. In these autologous therapies, the modified cells are grown and tested before being infused back into the patient.

Modified T-cells have demonstrated spectacular success in treating some cancers. With the exception of two cases these have been autologous therapies. A more useful product would be an off-the-shelf or allogeneic product that could be administered to any patient on diagnosis rather than after precious weeks of manufacturing their own cells. Using our ZFN-mediated genome editing technology to knock out genes that identify these cells as foreign to a patient, we are working to make this possibility a reality.

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Cell Therapy :: Sangamo Therapeutics, Inc. (SGMO)