Study Summary
The purpose of this Phase 1/2 study is to determine the feasibility and safety of stem cell collection and gamma-globin gene transfer, and success of gene correction in subjects with sickle cell disease
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ARU-1801GENETIC
Autologous CD34+ hematopoietic stem cells transduced ex-vivo with a gamma-globin lentiviral vector
* Subjects with sickle cell anemia will undergo hematopoietic stem cell procurement by bone marrow harvest or apheresis after mobilization with plerixafor
* Reduced intensity chemotherapy conditioning with single dose melphalan will be used to facilitate engraftment of ex-vivo transduced cells.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | United States |