The FDA approved a CRISPR-based therapy for beta thalassemia, an inherited blood disorder. The treatment, called Casgevy and developed by Vertex Pharmaceuticals and CRISPR Therapeutics, was expected to be approved and was ahead of the FDA’s deadline. This approval expands the use of the same therapy that was approved for sickle cell disease in December. The CEO of Vertex Pharmaceuticals, Reshma Kewalramani, expressed excitement over securing the approval for thalassemia, stating that patients with this condition deserve new therapy options. The therapy is expected to benefit eligible patients waiting for a potentially curative treatment.
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CRISPR-based therapy receives expanded approval for beta thalassemia
