HCA Healthcare Announces New England Journal of Medicine Study Highlighting Advances in CRISPR-based Therapy for Children

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HCA Healthcare, Inc. (NYSE:HCA), one of the nation's leading healthcare providers, today 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. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260629348492/en/The current NEJM study builds on HCA Healthcare's leadership in cell and gene therapy research and the pioneering work conducted at TriStar Centennial Medical Center, a part of the Sarah Cannon Transplant and Cellular Therapy Network. Across the network, more than 1,600 blood and marrow transplants and cellular therapies are performed annually. Dr. Frangoul was an investigator in the first U.S. clinical trial to use gene-editing to treat sickle cell disease, contributing to the development of the first FDA-approved CRISPR-based therapy in the U.S. for patients ages 12 and older. HCA Healthcare is expanding access to FDA-approved gene-editing therapies through specialized transplant and cellular therapy programs. TriStar Centennial Children's Hospital in Nashville and Methodist Children's Hospital in San Antonio currently offer gene-editing therapies for eligible patients, with Medical City Children's Hospital in Dallas preparing to expand services. Sickle cell disease and beta thalassemia are inherited blood disorders that can cause serious, lifelong complications beginning in childhood. A therapy that works in children ages 5-11 could make earlier intervention possible, potentially treating these diseases before years of cumulative injury and treatment burden occur. The study's lead author, Dr. Haydar Frangoul, medical director of HCA Healthcare’s Sarah Cannon Transplant and Cellular Therapy Program at TriStar Centennial Children's Hospital, presented the first published data evaluating exagamglogene autotemcel (exa-cel) in children ages 5-11. The study was conducted in collaboration with Sarah Cannon Research Institute. "For many patients with sickle cell disease and beta thalassemia, the burden of disease begins early in life," said Dr. Frangoul. “These findings reinforce the promise of gene-editing therapy and underscore the importance of continuing rigorous clinical research to evaluate ne...

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