CSL200

**CSL200** is an investigational lentiviral stem cell gene therapy developed by CSL Behring for the long-term management of sickle cell disease (SCD). It involves autologous CD34-positive hematopoietic stem cells transduced with a lentiviral vector encoding a γ-globinG16D/shRNA734 construct, which induces expression of a modified gamma-globin (G16D variant) and shRNA targeting the BCL11A gene to suppress beta-globin-like chains, thereby increasing fetal hemoglobin (HbF) levels and counteracting sickle hemoglobin polymerization. This approach aims to reduce vaso-occlusive crises and other SCD complications by genetically modifying patient stem cells ex vivo for reinfusion. CSL Behring is advancing it alongside other SCD programs like CSL889 (plasma-derived hemopexin).[1][5][11][13][15]

Other names

Gamma globin gene therapyγ-Globin gene therapy