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 and efficacy of lentiviral vector transduction of β-globin genetically modified autologous CD34+ hematopoietic stem cells in patients with transfusion-dependent 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 and efficacy of lentiviral vector transduction of β-globin genetically modified autologous CD34+ hematopoietic stem cells in patients with transfusion-dependent β-thalassemia.
A Phase 1 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+Stem Cells Transduced With a Lentiviral Vector Encoding βA-T87Q-Globin
This is a Phase 1,open label,safety,and efficacy study in subjects with non-β0/β0 TDT β-thalassemia Major by transplanting BD211 drug product which is for autologous use only,via a single IV administration.