July 2, 2026- HCA Healthcare, Inc. 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. Building on this research, HCA Healthcare’s Sarah Cannon Transplant and Cellular Therapy Network is expanding access to FDA-approved gene-editing therapies through specialized transplant and cellular therapy programs.
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.