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 transfusion-dependent β-thalassemia.
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The number, frequency and severity of adverse events (AE) after reinfusion of KL003 drug products
Timeframe: within 6 months
The proportion of participants who meet the definition of transfusion independence (TI) for at least 6 months
Timeframe: Up to 24 months post transplant