Researchers Publish Final Results of Key Clinical Trial for Gene Therapy for Sickle Cell Disease
Children's Hospital of PhiladelphiaAn international consortium 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). The study found that 96.7% of patients in the study did not have any vaso-occlusive crises (VOCs) – a blockage that results in lack of oxygen and painful episodes – for at least one year, and 100% were able to remain hospitalization-free for the same length of time.