Business
Health Canada Grants Marketing Authorization of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY® (Exagamglogene Autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
Health Canada Grants Marketing Authorization of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY&#x...
About this update from Vortex Energy Corp.
[{"type":"text","content":"\n\n\n\n Health Canada Grants Marketing Authorization of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY® (Exagamglogene Autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia\n \n\n /* Style Definitions */\nspan.prnews_span\n{\nfont-size:8pt;\nfont-family:\"Arial\";\ncolor:black;\n}\na.prnews_a\n{\ncolor:blue;\n}\nli.prnews_li\n{\nfont-size:8pt;\nfont-family:\"Arial\";\ncolor:black;\n}\np.prnews_p\n{\nfont-size:0.62em;\nfont-family:\"Arial\";\ncolor:black;\nmargin:0in;\n}\n.prntac{\nTEXT-ALIGN: CENTER\n}\n \n\n\n\n\n\n Canada NewsWire\n \n\n\n\n\n – First regulatory authorization of a CRISPR-based gene-editing therapy in\n \n Canada\n \n –\n \n\n\n\n\n TORONTO\n \n\n ,\n \n\n Sept. 25, 2024\n \n\n /CNW/ -\n \n Vertex Pharmaceuticals Incorporated\n \n (Nasdaq: VRTX) today announced Health Canada has granted Marketing Authorization for\n \n Pr\n \n CASGEVY\n \n ®\n \n (exagamglogene autotemcel), an autologous genome edited hematopoietic stem cell-based therapy, for the treatment of patients 12 years of age and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT). There are an estimated 2,000 patients eligible for CASGEVY in\n \n Canada\n \n , the majority of whom are living with SCD.\n \n\n\n\n\n\n\n\n\n \"Today's marketing authorization for CASGEVY as\n \n Canada's\n \n first CRISPR-based gene-editing therapy is a historic moment,\" said\n \n Michael Siauw\n \n , General Manager at Vertex Pharmaceuticals (\n \n Canada\n \n ) Incorporated. \"We are excited for the potential of this one-time therapy for eligible sickle cell disease and transfusion-dependent beta thalassemia patients and are working closely with provincial, territorial and federal governments to facilitate access as rapidly as possible.\"\n \n\n This approval is based on the positive interim results from two global and ongoing clinical trials, CLIMB-121 in SCD and CLIMB-111 in TDT. The primary endpoint of being free from severe VOCs for at least 12 consecutive months or transfusion independence for at least 12 consecutive months was met in both trials. The safety profile is generally consistent with myeloablative conditioning with busulfan and hematopoietic stem cell transplant.\n \n\n \"CASGEVY's approval is an exci...