FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients With Beta-Thalassemia Who Require Regular Blood Transfusions

FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients With Beta-Thalassemia Who Require Regular Blood Transfusions

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On August 17, the US Food and Drug Administration (FDA) approved betibeglogene autotemcel, the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.