Cell Therapy

Sangamo is aiming to pioneer the next generation of cell therapy using regulatory T cells, or Tregs.

Tregs are a subset of immune cells with potent immunomodulatory functions. These functions include maintaining immune homeostasis and immune tolerance, as well as regulating proinflammatory immune responses.

Overview

CAR-Tregs are regulatory T cells (Tregs) engineered with a chimeric antigen receptor (CAR), designed to localize and accumulate at the site of disease.

CAR-Tregs are expected to utilize multiple mechanisms of immune regulation to reduce inflammation locally.

For example, in inflammatory bowel diseases (IBD) such as Crohn’s disease, the inflammation is located in the gastrointestinal (GI) track.

We engineer Tregs with a CAR targeted to IL23R, which is found in the GI tract and overexpressed by inflammatory cells in the gut of Crohn’s disease patients. When the CAR-Tregs are infused into the patient, they are expected to migrate to the GI tract, engage IL23R and suppress the local inflammation.

CAR-Tregs engineered by Sangamo using Zinc Finger technology are positioned to be the new frontier in cell therapy to potentially address inflammatory and immune-mediated diseases.

Sangamo’s strategy is to first evaluate first-in-human proof-of-concept in an autologous study, and then potentially move to allogeneic approaches to address scalability.

For more information on our approach in Treg cell therapy, watch the short video below.

In addition to Tregs, Sangamo is also developing potential cell therapies using other types of cells, such as effector T cells for oncology, as part of a collaboration with Kite; and hematopoietic stem cells for sickle cell disease.

Sangamo Announces Updated Phase 1/2 STAAR Study Data in Fabry Disease Showing Sustained Benefit, Improvements in Kidney Function and a Favorable Safety Profile