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Protecting The Gift


For patients with a failing organ, such as kidney, liver, heart or lung, the only currently-available long-term treatment option to restore normal or near-normal organ function is the transplant of a healthy organ. We estimate that over 130,000 solid organ transplants were performed worldwide in 2016. Of these, 72,960 were performed in the United States, the European Union, Canada and Japan, of which 43,876, or 60%, were kidney transplants, according to the Global Observatory on Donation and Transplantation, or GODT. According to the Scientific Registry of Transplant Recipients, or SRTR, the 10-year rates for kidney transplant patients in the United States who were alive with a functional transplant kidney from 2006 are approximately 48% and 66% for deceased and living donor kidney transplants, respectively. Organ failure and death rates can be even higher for other types of organ transplants.

Long-term immunosuppressive therapy is required for transplant patients and can cause significant side effects, such as increased risk of developing lymphomas and other malignancies, opportunistic infections, cardiovascular disease, new-onset diabetes, tremor, headache, insomnia, diarrhea, nausea and hypertension. Aside from these potentially severe side effects, immunosuppressive therapies also involve significant expenses. The side effects and high treatment costs are a contributing factor to the low compliance rate by patients with their immunosuppressive treatment regimens, which in kidney transplant patients is estimated to be between 22% and 28%. In turn, noncompliance with a patient’s prescribed immunosuppressive drug regimen increases the risk of costly acute rejection episodes and eventual organ rejection and loss.

We are focused on the discovery, development and commercialization of personalized cellular immunotherapies engineered to reprogram the immune system in order to improve outcomes in organ transplant recipients. Our lead product candidate, MDR-101, is a cellular immunotherapy for the treatment of genetically-matched, or HLA-matched, living donor kidney transplant recipients that is manufactured from the blood and bone marrow cells of the kidney donor. A single dose of MDR-101 is intended to prevent transplant organ loss, preserve long-term transplant kidney function and eliminate the need for chronic immunosuppressive drugs. Beyond MDR-101, we are developing a pipeline of product candidates for recipients of HLA-mismatched living donor kidney transplants, the delayed therapy of prior recipients of living donor kidney transplants and recipients of deceased donor kidney transplants. We believe our platform has broad applicability beyond kidney transplantation and are evaluating other types of solid organ transplants, such as liver transplants, as well as product candidate development opportunities beyond organ transplantation.