NEJM study shows CRISPR therapy results in children ages 5-11

HCA Healthcare (NYSE: HCA) announced the publication of a study in The New England Journal of Medicine reporting results from a gene-editing therapy tested in children ages 5 to 11 with severe sickle cell disease and transfusion-dependent beta thalassemia.

The study evaluated exagamglogene autotemcel (exa-cel), a CRISPR-based therapy currently approved by the U.S. Food and Drug Administration for patients ages 12 and older with those conditions. The research was sponsored by Vertex Pharmaceuticals and conducted in collaboration with the Sarah Cannon Research Institute.

The findings drew from two phase 3 studies enrolling 26 children ages 5 to 11, including 15 with transfusion-dependent beta thalassemia and 11 with sickle cell disease. Among participants followed long enough to assess primary endpoints, all eight children with beta thalassemia achieved transfusion independence for at least 12 months, and all eight children with sickle cell disease remained free from severe vaso-occlusive crises for at least 12 months.

The therapy works by editing a patient's own blood-forming stem cells to increase production of fetal hemoglobin, which can help reduce disease complications. Participants also underwent myeloablative conditioning chemotherapy to prepare the bone marrow.

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, said the findings "reinforce the promise of gene-editing therapy" and underscore the need for continued clinical research in younger patients.

According to the Centers for Disease Control and Prevention, sickle cell disease affects approximately 100,000 people in the United States and is the most common inherited blood disorder in the country.

HCA Healthcare stated that TriStar Centennial Children's Hospital in Nashville and Methodist Children's Hospital in San Antonio currently offer FDA-approved gene-editing therapies for eligible patients, with Medical City Children's Hospital in Dallas preparing to expand services.

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