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Clinical Trials associated with γ-globin gene therapy(CSL Behring LLC)A Phase 1 Pilot Study to Evaluate the Safety and Feasibility of Gene Therapy With CSL200 (Autologous Enriched CD34+ Cell Fraction That Contains CD34+ Cells Transduced With Lentiviral Vector Encoding Human γ-GlobinG16D and Short-Hairpin RNA734) in Adult Subjects With Severe Sickle Cell Disease
This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor cells by apheresis after mobilization with plerixafor, reduced intensity conditioning with melphalan, and administration of CSL200.
100 Clinical Results associated with γ-globin gene therapy(CSL Behring LLC)
100 Translational Medicine associated with γ-globin gene therapy(CSL Behring LLC)
100 Patents (Medical) associated with γ-globin gene therapy(CSL Behring LLC)
100 Deals associated with γ-globin gene therapy(CSL Behring LLC)