Study Summary
This is a single group, open label study in 10 subjects who are 8 years of age or older with beta-thalassemia major. The objective of this study is to evaluate the safety and efficacy of autologous hematopoietic stem cell transduced with lentiviral vector for the treatment of beta-thalassemia major.
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Autologous CD34+ cells genetically modifiedGENETIC
Autologous hematopoietic stem cell transduced with lentiviral vector encoding the therapeutic beta-globin gene. The target dose in the transduced product is 3x10\^6 cells/Kg CD34+ cells, with a minimum dose of 2 x 10\^6/Kg and a maximum dose of 20 x 10\^6/Kg, depending on the yield of cells. The product will be injected intraosseously following intravenous BU ±Flu ±Cy.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Nanfang Hospial | Guangzhou | Guangdong | China |