U.K. Health Authorities Grant Approval for a New Medicine using CRISPR Gene Editing to Treat Beta thalassemia and Sickle Cell
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U.K. Health Authorities Grant Approval for a New Medicine using CRISPR Gene Editing to Treat Beta thalassemia and Sickle Cell

CRISPR was first described ten years ago as a viral mechanism for defense, taken from bacteria, which could be transformed into genetic scissors and accurately alter DNA. About Casgevy CRISPR…

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A Preview of Potentially Historic FDA Approvals for 2023
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A Preview of Potentially Historic FDA Approvals for 2023

  The later part of 2023 will see decisions on drugs in four areas such as advanced melanoma, sickle cell disease, Alzheimer’s disease, and depression. BioSpace shines the spotlight on…

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Gene Therapy for Severe Beta Thalassemia Earns Conditional Marketing Approval in the EU
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Gene Therapy for Severe Beta Thalassemia Earns Conditional Marketing Approval in the EU

According to a story from BioPortfolio, the gene therapy company bluebird bio, Inc. recently announced that its gene therapy ZYNTEGLO™ has earned conditional marketing authorization in the European Union from…

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Clinical Trial Results Look Good for Possible Treatment for Transfusion Dependent Beta Thalassemia
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Clinical Trial Results Look Good for Possible Treatment for Transfusion Dependent Beta Thalassemia

According to a story from Market Screener, the biopharmaceutical company Orchard Therapeutics recently announced that the results of a recent clinical trial testing the company' investigational product OTL-300 were encouraging.…

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Experimental Treatment for Anemia Associated With Beta Thalassemia Gets Fast Track Designation
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Experimental Treatment for Anemia Associated With Beta Thalassemia Gets Fast Track Designation

According to a story from PR Newswire, the drug developer Protagonist Therapeutics recently announced that its investigational therapy PTG-300 has earned Fast Track designation from the US Food and Drug…

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Gene Therapy Could Put an End to Blood Transfusions for Beta Thalessemia Patients
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Gene Therapy Could Put an End to Blood Transfusions for Beta Thalessemia Patients

According to a story from empr.com, a recent study revealed that gene therapy could allow patients with beta thalessemia to stop having to get regular blood transfusions. The gene therapy…

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