The US Food and Drug Administration (FDA) on Friday approved two milestone treatments, Casgevy (exagamglogene autotemcel [exa-cel]), and Lyfgenia (lovotibeglogene autotemcel [lovo-cel)], representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD).
In what was seen as a land-mark decision, the FDA granted approval of Casgevy to Vertex Pharmaceuticals (Nasdaq: VRTX) and of Lyfgenia to bluebird bio (Nasdaq: BLUE).
Additionally, the agency pointed out, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy.
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