ATIR201 is being developed for inherited blood disorders with an initial focus on thalassemia, an inherited blood disorder which results in improper oxygen transport and destruction of red blood cells in a patient. Replacing the diseased bone marrow through an HSCT and restoring the proper production of hemoglobin, would provide a cure for this disease. The addition of ATIR201 to transplant regimes in this indication should provide a more effective immune response and reduce mortality without the risk of GVHD until the immune system has fully re-grown from stem cells in a transplanted graft.

Clinical development of ATIR201 for thalassemia has recently been initiated with a Phase I/II clinical trial, regulatory approvals having been received in various European countries to start the trial.