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FDA Approves Casgevy, the First CRISPR-Based Gene Editing Therapy for β-Thalassemia
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Casgevy (exagamglogene autotemcel), the pioneering gene editing therapy based on CRISPR technology, has received approval from the US Food and Drug Administration (FDA) for the treatment of patients aged 12 and above with transfusion-dependent β-Thalassemia (TDT). This marks a significant milestone as Casgevy becomes the world’s first CRISPR-based gene editing…
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Vertex Pharmaceuticals and CRISPR Therapeutics Receive Conditional Approval for CRISPR Gene-Editing Therapy
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Vertex Pharmaceuticals (NASDAQ: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP) have received conditional approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for their jointly developed therapy, Casgevy (exagamglogene autotemcel), a treatment for sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and for transfusion-dependent beta thalassemia (TDT) in…
