Transfusion-dependent Beta-Thalassemia Clinical Trial
Official title:
Evaluation of the Safety and Efficacy of KL003 Gene Therapy in Patients With Transfusion-dependent β-thalassemia With No Conditioning Regimen
This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem cells transfusion in subjects with β-thalassemia major.
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| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Active, not recruiting |
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