Gamida Cell said today that the first patient has been transplanted in a study of its CordIn product for patients with severe aplastic anemia or hypoplastic myelodysplastic syndrome.
The product is designed for patients with rare genetic diseases who have no fully-matched donors for a bone marrow transplantation. Gamida Cell said it is also evaluating its CordIn therapy for patients with sickle cell disease.
The Phase I/II aplastic anemia trial is being conducted by Dr. Richard Childs – clinical director of the National Heart, Lung and Blood Institute’s division of intramural research. Childs is also the assistant U.S. surgeon general.
“Severe aplastic anemia and myelodysplastic syndrome are life-threatening bone marrow disorders with few optimal treatment options. Many patients with these diseases fail conventional therapy. Amongst those with severe AA who respond to conventional treatment, up to 30% will suffer relapse or evolve to myelodysplastic syndrome or leukemia which is often fatal,” Childs said in prepared remarks.
“Promising preclinical and clinical data have shown the efficacy of Gamida Cell’s ex-vivo hematopoietic stem cell technology. Based upon exciting prior data, we are now conducting a clinical trial at the NHLBI testing whether umbilical cord blood transplantation using CordIn can be used to improve the results of conventional cord blood transplantation for patients with these life-threatening conditions who lack an available matched donor.”
“The study, announced today, represents a significant milestone for Gamida Cell, as we continue our important mission of expanding access to curative transplantation in patients for which engraftment has been historically difficult,” president & CEO Yael Margolin added. “We highly value our collaboration with the NIH and with Dr. Childs, who is one of the world experts in the field, and we look forward to evaluating the potential of CordIn in patients with life-threatening hematologic diseases.”