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SCDAA Statement About Gene Therapy Approval
On Dec. 8, 2023, the Food and Drug Administration (FDA) approved two cell-based gene therapies for sickle cell disease (SCD), Casgevy from CRISPR/Vertex and Lyfgenia from bluebird bio. These are the first treatments of their kind available to individuals with SCD in the United States. SCDAA welcomes the approval of these potentially curative therapies which mark major advances in the treatment of sickle cell disease; however, there are valid concerns about accessibility and the potential for adverse effects.
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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.