Gamida Cell developed CordIn, a variation of NiCord®, adapted for transplantation in patients with chronic non-malignant diseases.
CordIn is currently being tested as a potential cure for sickle cell disease (SCD) and thalassemia. A phase I/II in pediatric patients with SCD is ongoing in the US. Five patients were enrolled to date, with encouraging preliminary data. A second phase I/II in patients with SCD and thalassemia was recently initiated in the US and in Europe.
An investigator initiated study in patients with aplastic anemia is planned to begin soon, in collaboration with the NIH (Prof. Richard Childs).
Future studies will test the safety and efficacy of CordIn in additional non-malignant, chronic orphan indications, such as: genetic metabolic diseases, refractory autoimmune diseases and bone marrow failure syndromes
Sickle cell disease and thalassemia are two rare genetic blood disorders, associated with high morbidity, frequent hospitalizations and a low life expectancy. Only symptomatic treatments are available to these patients. Cost of treating a patient with SCD is approximately 9 million USD over his life time. Hematopoietic cell transplantation is considered the only potential cure, but due to the challenge in finding a matched donor, very few patients are getting a transplant.