HCA Healthcare Inc (HCA) announced on Monday that a gene editing therapy study it participated in has been published in the New England Journal of Medicine, showing positive results for children aged 5 to 11 with severe sickle cell disease and transfusion-dependent beta-thalassemia.

The research evaluated a CRISPR gene editing therapy named exa-cel. This is a cell therapy that modifies a patient's own hematopoietic stem cells to increase the production of fetal hemoglobin. Previously, this therapy had received FDA approval for patients aged 12 and older, and this study is the first to assess its efficacy in a younger pediatric population.

The study was based on two Phase 3 clinical trials, involving a total of 26 children aged 5 to 11. Among participants with a sufficiently long follow-up period to evaluate the primary endpoint, the results were highly positive: all 8 children with beta-thalassemia achieved transfusion independence for at least 12 months, and all 8 children with sickle cell disease were free of severe vaso-occlusive crises for at least 12 months.

Dr. Haydar Frangoul, the study's lead author and Medical Director of the Sarah Cannon Transplant and Cell Therapy Program at TriStar Centennial Children's Hospital, stated that the disease burden of sickle cell disease and beta-thalassemia begins early in life. These findings strengthen the promise of gene editing therapies and underscore the importance of continuing rigorous clinical research to evaluate new treatment options for these severe blood disorders.

HCA Healthcare Inc is working to expand access to the FDA-approved gene editing therapy through its Sarah Cannon Transplant and Cell Therapy Network. Currently, treatment is available for eligible patients at TriStar Centennial Children's Hospital in Nashville and Methodist Children's Hospital in San Antonio, with services being prepared for expansion at Medical City Children's Hospital in Dallas. The study was sponsored by Vertex Pharmaceuticals.