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CTX001 Earns FDA’s Fast Track Status for Treating Sickle Cell Disease

The U.S. Food and Drug Administration (FDA) has awarded fast track status to investigational therapy CTX001 to treat sickle cell disease.

A Phase 1/2 clinical trial (NCT03745287) is recruiting patients.

CTX001 uses the CRISPR gene-editing technology to increase the production of fetal hemoglobin in patients’ red blood cells.

Fetal hemoglobin is a form of hemoglobin that naturally exists when one is born and later is replaced by the adult form of hemoglobin.

For the treatment, doctors collect a patient’s own hematopoietic stem cells (cells from the bone marrow) and modify them in the laboratory so they can produce high levels of fetal hemoglobin.