A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaemia.

A Single Site, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassemia

This is an open label, single site study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.

Key Inclusion Criteria:

• Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms;
• 6~35 years old, all gender;
• Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol;
• Eligible for autologous stem cell transplant;
• Organs in good function.

Other protocol defined Inclusion criteria may apply.

Key Exclusion Criteria:

• Subjects with associated α-thalassemia;
• Active bacterial, virus, fungal or parasitic infection, including HIV infection, HbsAg and HBV DNA positive, HCV DNA positive, or Treponema Pallidum infection;
• HLA identical sibling or unrelated donors are available;
• Prior allo-HSCT or gene therapy.

Other protocol defined Exclusion criteria may apply.