Crispr Therapeutics (CRSP) and its partner, Vertex Pharmaceuticals (VRTX), announced receipt of the first-ever approval of a CRISPR-based gene-editing therapy in the United States. The Food and Drug Administration approved Casgevy, an autologous genome edited hematopoietic stem cell-based gene therapy indicated for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises. The FDA’s approval of Casgevy triggered Vertex’s obligation to make a $200M milestone payment to Crispr Therapeutics.
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