CTX001™ granted Priority Medicines (PRIME) designation by the European Medicines Agency for the Treatment of Sickle Cell Disease (SCD)

CTX001 is an investigational gene-edited therapy that is being evaluated for patients suffering from transfusion-dependent beta thalassemia or severe SCD, in which a patient’s hematopoietic stem cells are engineered to produce high levels of foetal haemoglobin in red blood cells.

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Biospace Inc.