Exagamglogene autotemcel (Exa-cel) (aka CTX001™) is a genetically modified cell therapy that is used to increase fetal hemoglobin (HbF) in patients with sickle-cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).  Exagamglogene autotemcel is an investigational therapy administered via a stem cell transplant procedure as a single dose through a central venous catheter. This is potentially a one-time functional cure for patients with transfusion-dependent beta-thalassemia or severe sickle cell disease!  WOW

First, the patient’s hematopoietic stem cells are harvested from the blood. Exa-cel is then used to edit the stem cells to produce high levels of HbF in the red blood cells. The edited cells are transfused back into the patient via an autologous stem cell transplant through a central venous catheter. Exa-cel reduces the β globin damage that occurs in TDT and SCD. The need for red blood cell transfusions may be reduced or eliminated in patients with a reduction in severe vasocclusive episodes due to the increase in total hemoglobin levels.

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