Children as young as 2 can now receive an FDA-approved gene-editing therapy for sickle cell disease, extending a treatment presented as a potential cure beyond older patients.
TriStar Centennial in Nashville said pediatric hematologist-oncologist Dr. Haydar Frangoul helped develop the therapy, linking the approval to work done at the hospital.
Sickle cell disease has long meant painful complications, repeated hospital stays and limited treatment options, making the expanded approval a major shift for families.
A Nashville mother said her son was among the first teenagers in the U.S. to receive the therapy, underscoring how quickly the treatment is moving into patient care.
A revolutionary gene-editing therapy promises to cure sickle cell in toddlers, but will massive costs keep this miracle out of reach for most families?
While doctors celebrate a sickle cell cure for two-year-olds, what hidden, lifelong side effects lurk within the severe chemotherapy required beforehand?
Casgevy for Children as Young as 2: The $2.2 Million Gene Therapy Revolution and Its Ethical, Clinical, and Access Challenges
Overview
In July 2026, the FDA made history by expanding Casgevy, a CRISPR-based gene therapy, to children as young as 2 with sickle cell disease or transfusion-dependent β-thalassemia. This rapid approval was enabled by the National Priority Voucher program, which cut review times to just 53 days. Casgevy works by editing the BCL11A gene in a patient’s stem cells, allowing the body to produce healthy fetal hemoglobin and eliminating painful crises or transfusion needs. However, the treatment requires intense chemotherapy that can cause permanent infertility, especially challenging for young children whose fertility preservation options are limited and often unaffordable. High costs, insurance hurdles, and limited treatment centers further complicate access, while long-term safety—especially the risk of unintended genetic changes—remains under close surveillance through a 15-year registry.