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Innovation Observatory > Reports > Drugs > CTX001 for transfusion-dependent beta (β)-thalassaemia

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CTX001 for transfusion-dependent beta (β)-thalassaemia


Haematology and Blood Products

September 2021

CTX001 is under clinical development for the treatment of patients with beta-thalassaemia who require blood transfusions. Thalassaemia is a commonly inherited blood disorder resulting from an abnormality in one of the genes that affects the production of haemoglobin, a protein in red blood cells that carries oxygen throughout the body. Beta-thalassaemia is a subtype caused by a specific gene mutation. People with thalassaemia produce either little or no normal haemoglobin. Current treatment options for beta-thalassaemia are limited to blood transfusions with its associated risks and complications.
CTX001 is an ex vivo CRISPR investigational gene-edited therapy that is administered intraveneously. The patient’s haematopoietic stem cells are genetically engineered to produce high levels of foetal haemoglobin (HbF; haemoglobin F) in red blood cells. The elevation of HbF by CTX001 has the potential to improve transfusion requirements for beta-thalassaemia patients. If licensed CTX001 will offer a treatment for patients with transfusion-dependent beta-thalassaemia.

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