A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia

A Multicenter, Open Label Phase 1 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects with Transfusion Dependent Β-Thalassaemia

This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01.

Key Inclusion Criteria:

• Subjects and/or legal representative fully understand and voluntarily sign informed consent forms.
• Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol.
• Be not appropriate to receive conventional allogeneic hematopoietic stem cell transplantation.
• Lansky/Karnofsky score ≥ 70%.
• Eligible for hematopoietic stem cell transplantation and conditioning with busulfan as per investigator's judgement.

Other protocol defined inclusion criteria may apply.

Key Exclusion Criteria:

• Subjects with associated α-thalassemia.
• Subjects with any clinically significant acute or uncontrolled infections.
• History of uncontrolled epilepsy or other mental disorders.
• Previous treatment with allogeneic bone marrow transplantation or gene therapy.

Other protocol defined exclusion criteria may apply.