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Health Canada Grants Marketing Authorization of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY® (Exagamglogene Autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Depende...

Vertex Pharmaceuticals Incorporated (VRTX) 
Last vertex pharmaceuticals incorporated earnings: 4/29 04:01 pm Check Earnings Report
US:NASDAQ Investor Relations: investors.vrtx.com
Company Research Source: Yahoo! Finance
TORONTO Sept. 25, 2024 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced Health Canada has granted Marketing Authorization for Pr CASGEVY ® (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 Canada , the majority of whom are living with SCD. "Today's marketing authorization for CASGEVY as Canada's first CRISPR-based gene-editing therapy is a historic moment," said Michael Siauw , General Manager at Vertex Pharmaceuticals ( Canada ) 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 Show less Read more
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