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FDA grants CRISPR gene therapy fast track designation for sickle cell disease
The FDA granted fast track designation to CTX001, a gene-edited hematopoietic stem cell therapy, for the treatment of sickle cell disease.
CTX001 (CRISPR Therapeutics, Vertex Pharmaceuticals Inc.) is an investigational, autologous gene therapy under evaluation for patients with severe hemoglobinopathies. The ex-vivo therapy involves engineering a patient’s hematopoietic stem cells to produce high levels of fetal hemoglobin in red blood cells, with the goal of alleviating transfusion requirements for patients with beta-thalassemia and sickle crises for patients with sickle cell disease.
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.