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

First published data evaluating gene-editing treatment in children ages 5-11 with inherited blood disorders highlights the potential for earlier intervention before cumulative injury

NASHVILLE, Tenn.--(BUSINESS WIRE)--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.

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 new treatment options for children and individuals affected by these serious blood disorders."

According to the Centers for Disease Control and Prevention, sickle cell disease affects approximately 100,000 people in the United States and is the nation's most common inherited blood disorder. Children with sickle cell disease can experience severe pain crises, organ damage and frequent hospitalizations. Severe beta thalassemia affects thousands of people in the United States, and children with the condition often require lifelong blood transfusions that can lead to significant health complications.

The study evaluated exa-cel, a CRISPR-based cell therapy, which is currently approved by the U.S. Food and Drug Administration for eligible patients ages 12 and older with sickle cell disease and transfusion-dependent beta thalassemia. The therapy is designed to work by editing a patient's own blood-forming stem cells to increase production of fetal hemoglobin, which can help prevent or reduce disease complications. Participants underwent myeloablative conditioning, a conditioning chemotherapy treatment used to prepare the bone marrow to receive modified stem cells.

The findings were based on two phase 3 studies that enrolled 26 children ages 5-11, including 15 with transfusion-dependent beta thalassemia and 11 with sickle cell disease. Among participants who had been followed long enough to evaluate the study's 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.

"Studies like this demonstrate the important role research plays in advancing medicine and expanding treatment possibilities for patients," said Dr. Michael Cuffe, executive vice president and chief clinical officer of HCA Healthcare. “Through the HCA Healthcare Research Institute and in collaboration with the Sarah Cannon Research Institute, we are helping develop new therapies and treatment possibilities for patients facing serious and complex diseases."

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 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 in the U.S. for patients ages 12 and older. Like the previous CRISPR gene-editing clinical trial, the current NEJM study is sponsored by Vertex Pharmaceuticals Incorporated. In 2026 alone, Dr. Frangoul has authored five gene-editing studies published in NEJM.

Building on this research, HCA Healthcare’s Sarah Cannon Transplant and Cellular Therapy Network 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.

The full study is available on The New England Journal of Medicine website.

About HCA Healthcare

Nashville-based HCA Healthcare is one of the nation’s leading providers of healthcare services comprising 189 hospitals and approximately 2,600 ambulatory sites of care, including surgery centers, freestanding ERs, urgent care centers, and physician clinics, in 19 states and the United Kingdom. With its founding in 1968, HCA Healthcare created a new model for hospital care in the United States, using combined resources to strengthen hospitals, deliver patient-focused care and improve the practice of medicine. HCA Healthcare has conducted a number of clinical studies, including one that demonstrated that full-term delivery is healthier than early elective delivery of babies and another that identified a clinical protocol that can reduce bloodstream infections in ICU patients by 44%. HCA Healthcare is a learning health system that uses its approximately 47 million annual patient encounters to advance science, improve patient care and save lives.

All references to “Company,” “HCA” and “HCA Healthcare” as used throughout this document refer to HCA Healthcare, Inc. and its affiliates.