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

Korábbi kezelés

• Prior allo-HSCT or gene therapy.
  • Other protocol defined Exclusion criteria may apply.

Más betegség

• Active bacterial, virus, fungal or parasitic infection, including HIV infection, HbsAg and HBV DNA positive, HCV DNA positive, or Treponema Pallidum infection;

Egyéb

• Subjects with associated α-thalassemia;
• HLA identical sibling or unrelated donors are available;

• 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.