- 4-13 × 106 cells/mL dispersion for infusion
- Approved for treating transfusion-dependent beta-thalassaemia in people 12 years and over in line with NICE
- Approved (in line with NICE) for treating sickle cell disease (SCD) in people 12 years and over:
- who have:
- recurrent vaso-occlusive crises (VOCs) and
- a βS/βS, βS/β+ or βS/β0 genotype and
- when haematopoietic stem cell transplant (HSCT) is suitable, but a human leukocyte antigen-matched related haematopoietic stem cell donor is not available.
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