FDA Approves Second Indication for the Gene Therapy Casgevy.

The article focuses on the Food and Drug Administration's approval of Casgevy, for treating transfusion-dependent beta thalassemia (TDT) in patients aged 12 and older, following its approval for sickle cell disease in December 2023. Topics include the significant cost of Casgevy, the one-time gene therapy's mechanism utilizing CRISPR/Cas9 technology, and the provision of support through Vertex Connects, a program aiding patients and providers during the treatment journey.