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Sickle Cell Disease News
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Gene-editing therapy to now be available to patients at low or no cost
Casgevy (exagamglogene autotemcel) will be reimbursed by Germany’s public health system, enabling people with severe sickle cell disease (SCD) in the country, ages 12 and older, to access the one-time gene-editing therapy at low or no cost.
The reimbursement agreement, established with Vertex Pharmaceuticals — which developed the therapy alongside CRISPR Therapeutics — covers both eligible SCD patients and eligible people with transfusion-dependent beta thalassemia (TDT), a related blood condition. This matches the approved indications of Casgevy in the European Union, where the therapy was cleared for use in 2024.