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Clinical Trials associated with BD-211BD211静脉输注治疗输血依赖型β-地中海贫血患者安全性和有效性的I期临床研究
[Translation] Phase I clinical study on the safety and efficacy of intravenous infusion of BD211 in the treatment of patients with transfusion-dependent β-thalassemia
主要目的是评估编码人βA-T87Q-珠蛋白基因的BD211慢病毒载体体外转导修饰自体CD34+造血干细胞(BD211注射液)静脉回输到输血依赖型β-地中海贫血(TDT)受试者体内后的安全性和耐受性。次要目的是评估BD211静脉回输到TDT受试者体内后的有效性和BD211静脉回输到TDT受试者体内后的药效学特征。
[Translation] The primary objective is to evaluate the safety and tolerability of autologous CD34+ hematopoietic stem cells (BD211 injection) modified by BD211 lentiviral vector encoding human βA-T87Q-globin gene after intravenous infusion into subjects with transfusion-dependent β-thalassemia (TDT). The secondary objective is to evaluate the efficacy of BD211 after intravenous infusion into TDT subjects and the pharmacodynamic characteristics of BD211 after intravenous infusion into TDT subjects.
A Phase 1 Clinical Trail of the Safety and Efficacy of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Intravenous Infusion for the Treatment of Transfusion-dependent β-thalassaemia Patients
This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.
Safety and Efficacy of Lentiviral Vector Transduction of β-globin Genetically Modified Autologous CD34+ Hematopoietic Stem Cells in Patients With Transfusion-dependent β-thalassemia
This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.
100 Clinical Results associated with BD-211
100 Translational Medicine associated with BD-211
100 Patents (Medical) associated with BD-211
100 Deals associated with BD-211