TX200 is an investigational therapy being developed by Sangamo Therapeutics to prevent an immune-mediated rejection of a kidney transplant.

How does TX200 work?

Alport syndrome patients often progress to end-stage kidney disease. At this point, kidney function is severely impaired and patients require dialysis and eventually a kidney transplant.

An organ transplant always carries the risk of rejection because the patient’s immune system recognizes the transplanted organ as foreign. One reason why organs are rejected is that the donor’s and the recipient’s human leukocyte antigen (HLA) proteins do not match. The immune system recognizes this mismatch and attacks the foreign HLA proteins on the transplanted organ.

TX200 is a chimeric antigen receptor (CAR) T-cell therapy that uses a subset of the patient’s regulatory T-cells, which are part of the immune system. The regulatory T-cells are first collected from the patient and then genetically engineered to target HLA-A2, a type of HLA protein. TX200 binds to HLA-A2 molecules on the transplanted kidney, suppressing the immune response against the organ.

If treatment with TX200 turns out to be effective, it may reduce or replace the use of immunosuppressant medications that organ transplant patients have to take for the rest of their lives.

TX200 in clinical trials

TX200 has not yet been tested in clinical trials.

Sangamo plans to conduct a multicenter, open-label, first-in-human Phase 1/2 clinical trial called STEADFAST to assess the potential of TX200 in preventing immune-mediated rejection of kidney transplants.

The study, which will test single ascending doses of TX200, will be carried out in the U.K., France, the Netherlands, Germany, and Belgium. The first trial sites are expected to open in 2020.

 

Last updated: Jan. 2, 2019

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