CRISPR therapy shows promise in children as young as 5 with sickle cell disease

HCA Healthcare announced a study showing CRISPR gene-editing therapy was effective in 26 children ages 5-11 with severe blood disorders.stocktitan
All eight evaluable sickle cell patients remained crisis-free for 12 months, and all eight beta thalassemia patients achieved transfusion independence.businesswire
The therapy, sponsored by Vertex Pharmaceuticals, is currently FDA-approved only for patients ages 12 and older.stocktitan+1

HCA Healthcare announced on Monday that research published in The New England Journal of Medicine demonstrates promising results from a CRISPR gene-editing therapy tested in children ages 5 to 11 with severe sickle cell disease and transfusion-dependent beta thalassemia, marking the first published data evaluating the treatment in this younger age group.stocktitan+1

Study Results

The findings are based on two phase 3 studies that enrolled 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 evaluate primary endpoints, all eight children with beta thalassemia achieved transfusion independence for at least 12 months, while all eight children with sickle cell disease remained free from severe vaso-occlusive crises for at least 12 months.businesswire+1

The therapy evaluated is exagamglogene autotemcel (exa-cel), a CRISPR-based cell therapy currently approved by the FDA for eligible patients ages 12 and older. It works by editing a patient's own blood-forming stem cells to increase production of fetal hemoglobin, which can help prevent or reduce disease complications. The study was sponsored by Vertex Pharmaceuticals .stocktitan+1

Significance for Younger Patients

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 importance of continuing rigorous clinical research to evaluate new treatment options for children and individuals affected by these serious blood disorders."stocktitan

Sickle cell disease affects approximately 100,000 people in the United States and is the nation's most common inherited blood disorder. Children with the condition can experience severe pain crises, organ damage, and frequent hospitalizations. A therapy that works in children ages 5 to 11 could enable earlier intervention before years of cumulative organ injury occur.stocktitan

Expanding Access

HCA Healthcare said it is expanding access to FDA-approved gene-editing therapies through its Sarah Cannon Transplant and Cellular Therapy Network, which performs more than 1,600 blood and marrow transplants and cellular therapies annually. 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.stocktitan

Dr. Frangoul was previously 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 for patients ages 12 and older. In 2026 alone, he has authored five gene-editing studies published in the New England Journal of Medicine.stocktitan

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