Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells

Inclusion Criteria: * Age 3-35 years old (inclusive), male or female; * The subject and/or his/her legally recognized representative/parent/guardian fully understands the study and all information related to the study and has signed the informed consent form; * Clinical diagnosis of transfusion-dependent β-thalassemia (TDT) with a blood transfusion record within 2 years (inclusive) prior to screening showing a history of ≥ 10 units (U)/kg/year (or ≥ 100 mL/kg/year) or ≥ 8 times/year of suspended RBC transfusions in at least 1 consecutive 12-month period; * Karnofsky score (for subjects aged ≥ 16 years) or Lansky score (for subjects aged \< 16 years) of ≥ 80; * Subjects in stable disease state who are eligible for hematopoietic stem cell transplantation as per investigator's judgment; * Access to diagnosis and treatment records issued by medical professional institutions within 2 years prior to screening, including the records of blood transfusions, hematology, serum chemistry, and other examinations; * Willing and able to comply with study procedures, with good compliance, and willing to receive and complete the follow-up study with a duration of at least 2 years; * Subjects of childbearing potential (including female subjects of childbearing potential and male subjects whose partners are of childbearing potential) must use effective contraception within 12 months of treatment. Exclusion Criteria: * Diagnosis of associated α-thalassemia: \> 1 alpha chain deletion or alpha …