Business Wire·Jun 29, 2026, 8:30 AM ET

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

HCA

HCA Healthcare announced new research published in The New England Journal of Medicine evaluating the gene-editing therapy exa-cel in children ages 5-11 with sickle cell disease or transfusion-dependent beta thalassemia.
The study involved 26 participants across two phase 3 trials, including 15 with beta thalassemia and 11 with sickle cell disease.
Among participants with beta thalassemia followed long enough to assess primary endpoints, all eight achieved transfusion independence for at least 12 months.
Among participants with sickle cell disease followed long enough to assess primary endpoints, all eight remained free from severe vaso-occlusive crises for at least 12 months.
The therapy works by editing a patient's blood-forming stem cells to increase fetal hemoglobin production, allowing for earlier intervention before cumulative disease injury occurs.
Dr. Haydar Frangoul led the study as the first published data evaluation of exa-cel in this age group, building on his role in developing the first FDA-approved CRISPR therapy for patients ages 12 and older.
The research was sponsored by Vertex Pharmaceuticals Incorporated in collaboration with the Sarah Cannon Research Institute.
HCA Healthcare currently offers gene-editing therapies at TriStar Centennial Children's Hospital in Nashville and Methodist Children's Hospital in San Antonio, with Medical City Children's Hospital in Dallas preparing to expand services.
The findings suggest potential for treating these inherited blood disorders earlier in life to prevent lifelong complications such as organ damage, pain crises, and frequent hospitalizations.
HCA Healthcare's Sarah Cannon Transplant and Cellular Therapy Network performs more than 1,600 blood and marrow transplants and cellular therapies annually.