Another significant milestone in gene therapy occurred earlier this month: the FDA expanded the approval for Casgevy (exagamglogene autotemcel), the world’s first CRISPR-based gene therapy. Previously restricted to patients aged 12 and older, the updated label now allows its use in children as young as 2 years old. biopharminternational.com +2
This expansion applies to two conditions: severe sickle cell disease (with recurrent vaso-occlusive crises) and transfusion-dependent beta-thalassemia. By using CRISPR/Cas9 technology, the therapy edits the patient’s own hematopoietic stem cells to reactivate fetal hemoglobin (HbF), which helps prevent red blood cells from deforming into sickle shapes. For patients with transfusion-dependent beta-thalassemia, it can eliminate the need for regular blood transfusions. biopharminternational.com +1
The move significantly broadens the potential patient population for this one-time therapy, offering the prospect of a functional cure for diseases that cause lifelong suffering beginning in infancy. Vertex Pharmaceuticals and CRISPR Therapeutics have reported that more than 75 authorized treatment centers in the U.S. are now activated for Casgevy.
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