Sangamo Announces UK Authorization of a Phase 1/2 Clinical Trial Evaluating the CAR-Treg Cell Therapy TX200 for Kidney Transplantation
The STEADFAST clinical study of TX200 is the first-in-human trial to evaluate a CAR-Treg cell therapy
“Being the first company to test a CAR-Treg candidate in humans is an important milestone for Sangamo and this exciting new frontier of cellular therapy. We believe that the TX200 program will be invaluable in expanding our understanding of the safety and mechanism of action of CAR-Treg cells and their relevance in the clinic,” said
TX200 is an autologous (cells collected from the patient) HLA-A2-targeted CAR-Treg cell therapy. The patient’s regulatory T cells (Tregs), a type of white blood cell which plays a key role in regulating the immune response and inflammation, are collected and genetically engineered with a Chimeric Antigen Receptor (CAR) designed to bind to HLA-A2. HLA-A2 is a protein belonging to the human leukocyte antigen (HLA) system. Donor/recipient mismatch in HLA molecules is a primary contributor to organ transplant incompatibility and may ultimately lead to immune-mediated rejection of the transplanted organ. The recipient’s immune system can recognize this HLA-A2 mismatch and may attack the new kidney carrying the HLA-A2 protein, potentially leading to graft rejection.
TX200 HLA-A2 CAR-Treg cells are designed to accumulate and localize within the new kidney where the HLA‑A2 protein is present, thereby utilizing the ability of Tregs to suppress immune responses against the transplanted kidney. This approach aims to help the recipient’s immune system accept the new kidney and induce immunological tolerance, potentially allowing tapering and eventual elimination of accompanying standard-of-care immune suppressive treatments.
Kidney transplantation is the treatment of choice for patients with ESRD who must otherwise remain on long-term dialysis. To prevent graft rejection, transplanted patients are treated with lifelong immune suppressive therapy, which impacts the body’s immune system broadly and is associated with multiple side effects, including an increased risk of infectious complications, cancer, and other drug-related toxicities.
TX200, which has been developed in collaboration with Professor
About the STEADFAST study
The STEADFAST study is a multicenter, open-label, single ascending dose, dose-ranging Phase 1/2 study. Sangamo plans to run this study in five different countries in
- JCI Insight. 2019;4(6):e123672. https://doi.org/10.1172/jci.insight.123672.
Forward Looking Statements
This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements regarding the potential benefits of cell therapy, including but not limited to Treg therapy, the Company's ability to develop and commercialize product candidates to address genetic diseases with the Company's proprietary technologies and the timing of commencement or next stages of such programs and the anticipated benefits therefrom. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the outcomes of clinical trials, the uncertain regulatory approval process, uncertainties related to the execution of clinical trials and uncertainties that research outcomes will support clinical programs. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business environments. These risks and uncertainties are described more fully in Sangamo's Annual Report on Form 10-K for the year ended
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