Recent advances in CRISPR-based gene-editing therapy may prompt new conversations between physicians and families about treatment options for children with inherited blood disorders. These include sickle cell disease, in which abnormal hemoglobin causes sickle-shaped red blood cells, and beta thalassemia, in which low levels of hemoglobin reduce oxygen in the body and cause a shortage of red blood cells. Both disorders can cause severe symptoms, including anemia, jaundice and growth delays.
Gene editing is one of several approaches physicians and families may consider when managing these conditions. Whether it is appropriate depends on the patient’s disease, treatment history, risks and preferences
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Complications from sickle cell disease can accumulate as children become adolescents and adults. For children with transfusion-dependent beta thalassemia, the need for ongoing red blood cell transfusions can also lead to cumulative harm through iron overload. Those complications can disrupt school, work and daily activities
To address the challenge of cumulative harm from these blood disorders, several studies have investigated the potential of using approved gene-editing therapies in children at a younger age.
A recent <a href="https://hcahealthcaretoday.com/2026/06/29/hca-healthcare-announces-new-england-journal-of-medicine-study-highlighting-advances-in-crispr-based-therapy-for-children/” rel=”nofollow noopener” target=”_blank”>study led by Haydar Frangoul, MD, MS, medical director of HCA Healthcare’s Sarah Cannon Transplant and Cellular Therapy Program at TriStar Centennial Children’s Hospital, evaluated a CRISPR-based gene-editing therapy in children 5–11 years old with sickle cell disease and transfusion-dependent beta thalassemia. The study was conducted in collaboration with Sarah Cannon Research Institute.
On July 1, 2026, the Food and Drug Administration expanded approval of a CRISPR-based gene editing therapy, to eligible patients 2 years or older. The therapy, called CASGEVY or exagamglogene autotemcel, was previously approved only for patients 12 years or older. The expanded indication follows years of clinical research in gene-editing therapies, including studies involving HCA Healthcare investigators.
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Weigh benefits of earlier treatment
Gene-editing therapy may prevent future complications from an inherited blood disorder. But it cannot always reverse damage that has already occurred
That potential benefit is only one part of the treatment decision. Physicians and families must also consider the risks of therapy, how well the disease is controlled with other treatments and the patient’s individual circumstances
For a patient who has already developed irreversible damage to a joint, for example, treatment may prevent the same complication from affecting another joint. It cannot restore the joint that has already been damaged
“It’s like you have a hammer. It’s hitting a wall, and you can take the hammer away when you do curative therapy, but you can’t fix the damage in the wall,” said Dr. Frangoul, who also served as an investigator in the first U.S. clinical trial evaluating CRISPR gene-editing for sickle cell disease, research that contributed to the development of the first FDA-approved CRISPR-based therapy
That is why the growing use of gene editing in younger children is changing more than treatment eligibility. It is also changing when physicians may need to begin conversations about curative therapy
“Doing these therapies earlier in life may prevent complications from accumulating over time,” he said
But Dr. Frangoul is not arguing that every eligible child should undergo gene editing or that it should replace other therapies. His larger message is that families should have enough information to consider it alongside other options before disease-related complications narrow their choices
“Now it might not be for everybody,” he said. But patients and families should understand how gene editing works, its potential benefits and its risks so they can decide whether it belongs among the options they want to consider.
The outcomes seen across different age groups illustrate why the timing of treatment may matter, he noted
Adults treated after years of disease may continue to experience chronic pain related to established organ damage. Meanwhile, adolescents have generally had fewer such complications, while the younger children in his team’s more recent research had not developed the same pattern of chronic pain
“Doing these therapies early in life can potentially offer patients an option to be cured from a disease that currently has very limited cure options,” Dr. Frangoul said
Advancing gene-editing therapy through clinical trials
Dr. Frangoul traces the progress in gene editing to patients who agreed to enter early clinical trials despite considerable uncertainty, noting that “the heroes in all of this are the patients who agreed to participate in these trials.”
He pointed to Victoria Gray, the first patient his team treated with CRISPR-based gene editing for sickle cell disease, and to the children who later participated in pediatric research
“These are the pioneers,” Dr. Frangoul said. “Those children who participated in the trial … are the people who opened the door and made this therapy available.”
When Gray was treated in 2019, gene editing in humans was still largely uncharted territory
“If you looked in ClinicalTrials.gov, you could not find any gene-editing clinical trials for any disease,” Dr. Frangoul said
The field has since expanded to include research on gene editing for multiple inherited conditions
That rapid progress means physicians who do not perform gene-editing procedures may still face more questions from patients and families. They will need enough familiarity to explain why referral may be appropriate and what the treatment process entails
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CRISPR-based cell therapy is a type of gene-editing therapy. 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. Because the treatment uses the patient’s own cells, it does not require a matched donor
“You don’t have to be on immune suppression afterward to prevent rejection or graft-versus-host disease,” Dr. Frangoul said
That may allow patients to return to school or work sooner than they would after a traditional bone marrow transplant using donor cells. While it is a one-time treatment, the treatment journey consists of multiple stages before, during and after treatment.
As with many advanced therapies, patients still undergo conditioning chemotherapy with this therapy as part of the treatment process
One of the most difficult issues for families is the potential effect of chemotherapy on fertility
Determining whether gene-editing therapy is the right option is an individualized process involving the patient, family and physician. The goal is not to minimize the risks or portray treatment as inevitable. It is to give families enough information to weigh those risks against the child’s current disease burden and the possible consequences of waiting
Dr. Frangoul believes hematologists should encourage families to meet with physicians who specialize in transplantation and curative therapy
“This is not for everybody,” he said. But patients and families should have an opportunity to learn about gene editing and determine with their doctor and care team whether it warrants further consideration.
A specialist consultation can help families understand whether treatment may be appropriate, what timing could make sense and what risks they would need to consider
“They need to at least talk to somebody who knows how to deliver this therapy and understand: Is it right for them? Is it right for their child? What is the right timing to do it?” Dr. Frangoul said
Importantly, a referral is not the same as recommending treatment
There may also be some families who learn about gene-editing therapy and decide to wait. They may feel their child is doing well with current therapy or want more time before taking on the risks of chemotherapy
“Some people might meet with me and say, ‘Hey, look, this is not for me. We don’t want to do it right now. We want to wait. My child is doing OK for now,’” he said
A consultation can help a family leave with information they can revisit later
Coordinating care across specialties
Gene-editing therapies are delivered at specialized centers. The physicians performing those procedures are not always the same hematologists who provide a patient’s ongoing care. That creates a need for collaboration
“The people performing this therapy—the transplant physicians, like me—are not necessarily the same people caring for the people with sickle cell disease and thalassemia,” Dr. Frangoul said
For transfusion-dependent beta thalassemia, he favors early referral to specialists so families can learn about potentially curative approaches and determine whether they may be appropriate
Meanwhile, for sickle cell disease, referral decisions may depend on symptoms, pain crises, the patient’s treatment goals and how well the disease is controlled with other therapies. Gene editing is not the only option, and some patients may continue to do well with their existing treatment
Even for patients doing well on hydroxyurea, Dr. Frangoul believes a discussion about gene editing may be appropriate so families understand the range of options available
Primary care physicians also have a role. They may not determine eligibility, but they can encourage patients to remain connected with hematology care and ask whether a consultation about curative therapy is appropriate
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While gene editing has advanced, Dr. Frangoul sees the current treatment process as only one stage in the field’s development
Researchers are exploring ways to edit cells inside the body, rather than collecting and returning them. They are also investigating approaches that could require less chemotherapy or no chemotherapy
The current approach used established conditioning chemotherapy so researchers could determine whether the gene-editing process itself worked
“The next step in the field is: How can we, now that we have proven we can gene edit safely and we can gene edit the cells to effectively eliminate the signs and symptoms of disease, deliver this therapy with fewer side effects?” he said
Discussing potentially curative therapy with families
Physicians caring for children with sickle cell disease or transfusion-dependent beta thalassemia should not assume that families already know about gene editing or other potentially curative approaches
They can begin by explaining that:
- Gene editing is one of several treatment approaches that may be considered.
- Gene-editing therapy may offer a potentially curative option for some patients.
- Earlier treatment may prevent some complications from accumulating.
- The process still requires stem-cell collection and chemotherapy.
- Fertility and other treatment risks require careful discussion.
- Referral to a specialized center provides information, not an obligation to proceed.
The shift, Dr. Frangoul said, is in when those conversations can begin. Gene editing may not be the right choice for every child with sickle cell disease or beta thalassemia, but discussing it alongside other treatment options can give families time to weigh potential benefits, risks and alternatives with their doctor and care team.
Learn more about sickle cell disease and the treatment options available
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