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Clinical Trials associated with ALS20-transduced Hematopoietic Progenitor Cells(The Children's Hospital of Philadelphia)Phase 1/2 Study Evaluating the Safety and Efficacy of Gene Therapy Employing Lentiviral Vector ALS20-transduced Hematopoietic Progenitor Cells in Subjects With Transfusion-dependent-thalassemia
The main goal of this study is to find out if the blood disorder called transfusion-dependent beta thalassemia can be safely treated by modifying blood stem cells. This is done by collecting blood stem cells from the subject, modifying those cells, adding a healthy beta globin gene, and then giving them back to the subject. It is hoped that these modified cells will decrease the need for blood transfusions. The gene modified blood stem cells are called CHOP-ALS20 ("study drug"). This experimental gene therapy has not been tried on human beings before and is not FDA approved.
100 Clinical Results associated with ALS20-transduced Hematopoietic Progenitor Cells(The Children's Hospital of Philadelphia)
100 Translational Medicine associated with ALS20-transduced Hematopoietic Progenitor Cells(The Children's Hospital of Philadelphia)
100 Patents (Medical) associated with ALS20-transduced Hematopoietic Progenitor Cells(The Children's Hospital of Philadelphia)
100 Deals associated with ALS20-transduced Hematopoietic Progenitor Cells(The Children's Hospital of Philadelphia)