HCA Healthcare Announces Gene Editing Therapy Research Results
HCA Healthcare announced new research published in The New England Journal of Medicine, NEJM, demonstrating promising results from a gene-editing therapy being investigated in children ages 5-11 with severe sickle cell disease and transfusion-dependent beta thalassemia. The study evaluated exa-cel, a CRISPR-based cell therapy, which is currently approved by the U.S. Food and Drug Administration for eligible patients ages 12 and older with sickle cell disease and transfusion-dependent beta thalassemia. The therapy is designed to work by editing a patient's own blood-forming stem cells to increase production of fetal hemoglobin, which can help prevent or reduce disease complications. The findings were based on two phase 3 studies that enrolled 26 children ages 5-11, including 15 with transfusion-dependent beta thalassemia and 11 with sickle cell disease. Among participants who had been followed long enough to evaluate the study's primary endpoints, all eight children with beta thalassemia achieved transfusion independence for at least 12 months, while all eight children with sickle cell disease remained free from severe vaso-occlusive crises for at least 12 months.
