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 in subjects with transfusion-dependent β-thalassemia.
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Percentage of participants with successful lentiviral vector transduced CD34+ stem cell engraftment
Timeframe: Up to 42 days post transplant
Engraftment time of neutrophil
Timeframe: Up to 42 days post transplant
Engraftment time of platelet
Timeframe: Up to 42 days post transplant
Transplant-related mortality within 100 days and within 1 year after reinfusion of KL003 drug product
Timeframe: Up to 1 year post transplant
The number, frequency and severity of adverse events (AE) within 1 year after reinfusion of KL003 drug products
Timeframe: Up to 24 months post transplant