This is an open label study to evaluate the safety and efficacy of α-globin Restored Autologous Hematopoietic Stem Cells in α-Thalassemia Major Patients
Aged 12-35 years (inclusive), ICF can be provided by the patient and/or legal guardian;
Definitively α- thalassemia diagnosed with severe TDT without genotype restriction, and a valid test report can be provided;
Average transfusion volume > 100 mL/kg/year or transfusion frequency > 8 times/year within 2 years prior to enrollment, or has been definitively diagnosed with TDT;
At least 3 months of full volume transfusion (verification of blood transfusion records can be provided) prior to screening, and Hb is maintained at ≥ 9.0 g/dL;
Patients with fully HLA-matched donors;
Received allogeneic transplantation, which needs to be weighed and evaluated by an expert committee; received other gene therapies;
Have previously undergone splenectomy;
Uncorrected bleeding disorder;