Gene therapy aims to cure Transfusion-Dependent thalassemia in a single shot

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Subjects between 12 and 45 years of age at the time of consent and are able to provide written informed consent. 2. Diagnosis of TDT, also known as β-thalassemia major, without genotype restriction, and a valid test report can be provided. 3. A history of at least 100 mL/kg/year of pRBCs transfusion or ≥ 8 transfusions of pRBCs per year for previous 2 years. 4. Sufficient blood transfusion for at least 3 months before screening (transfusion records can be provided), and Hb is maintained ≥9.0 g/dL before each transfusion. 5. The level of ferritin \<5000ng/mL; Cardiac magnetic resonance imaging (MRI) T2 and liver MRI T2 findings suggest iron overload at a moderate level or below. 6. Clinically stable, and eligible for autologous hematopoietic stem cell transplant (auto-HSCT). 7. Adequate organ function for the conditioning with busulfan. Exclusion Criteria: 1. Uncorrected bleeding disorder; 2. Uncontrolled epilepsy or mental disorders; 3. Received hydroxyurea, ruxolitinib, decitabine, or cytarabine within 3 months prior to enrollment; 4. Usage of psychoactive substance, drug, or alcohol abuse within six months prior to screening. 5. Pulmonary hypertension without effective intervention. 6. Persistent toxicity (≥ CTCAE grade 2) induced by previous treatment. 7. Positive for anti-RBC antibodies. 8. Positive for hepatitis B surface antigen (HBsAg) and HBV DNA copy number \> upper limit of normal (ULN) (HBV DNA test not required for patients negative for HBsAg), positive for hepatitis C virus (HCV) antibody, unless HCV RNA is negative (HCV RNA-negative subjects are not excluded), positive human immunodeficiency virus (HIV), or positive for Treponema pallidum antibody (TP-Ab) (subjects who are positive for the antibody due to vaccination can be enrolled). 9. Has or has had malignant tumors or myeloproliferative disease or immunodeficiency disease; 10. Clinically significant and active bacterial, viral, fungal, or parasitic infection.