Researchers publish final results of key clinical trial for gene therapy for sickle cell disease

Researchers publish final results of key clinical trial for gene therapy for sickle cell disease

In a landmark study, an international consortium led by researchers at Children’s Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).

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