Study Summary
It is a single-arm, single-center, open-label, single-dose study. A total of three subjects with severe sickle cell disease (SCD), aged 12-50 years (inclusive), are planned to receive cell infusion. After successful hematopoietic stem cell engraftment is achieved in the first subject, cell infusion will be initiated for subsequent subjects.
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KL003 Cell InjectionGENETIC
KL003 is an autologous CD34⁺ hematopoietic stem cell gene therapy product in which the βA-T87Q-globin gene is transduced via a lentiviral vector. Through genetic modification, the patient's autologous CD34⁺ hematopoietic stem cells are engineered to differentiate into red blood cells expressing functional β-globin, thereby increasing overall hemoglobin levels, improving anemia, and ultimately eliminating transfusion dependence.
Study Locations
No locations listed.