Casgevy expanded to age 2 and older, opening CRISPR therapy to younger patients

The FDA issued a supplemental approval on 1 July 2026 for Casgevy (exagamglogene autotemcel) in certain patients aged 2 years and older with sickle cell disease or transfusion-dependent beta thalassemia. The key shift is a much younger US indication for a CRISPR-based gene therapy, not a simple treatment that every patient can immediately receive.

Who Is Affected

The expanded approval covers patients aged 2 years and older with sickle cell disease and recurrent vaso-occlusive crises, or transfusion-dependent beta thalassemia. FDA says Casgevy had previously been approved for patients aged 12 years and older with SCD or TDT. The change matters for families with younger children, but eligibility still depends on disease criteria, treatment history, transplant risk, and access to specialized centers.

What Changes

FDA issued the supplemental approval on 1 July 2026. FDA’s Casgevy product page lists STN 125787 for patients aged 2 years and older with SCD with recurrent VOCs or TDT. FDA calls it the first gene therapy approved for patients aged 2 years and older with SCD. Vertex describes it as the first approved genetic therapy indicated for children as young as 2 for both SCD and TDT.

What The Therapy Is

Casgevy uses a patient’s own hematopoietic stem cells, edits them with CRISPR/Cas9, and returns them as a one-time intravenous infusion. FDA says the edited cells engraft in the bone marrow. That means this is not a pharmacy pill or routine outpatient injection; it involves cell collection, conditioning, intensive care planning, and long-term follow-up.

Limits And Cautions

The approval is a US decision, and age or disease indications can differ by country. It also does not mean every child with SCD or TDT is eligible. Families need specialist review of disease severity, prior therapies, infection and transplant risks, fertility preservation counseling, and follow-up plans. FDA product documents and approval letters are the starting point.

Official Checks

Use the FDA announcement for the date, age expansion, and first pediatric SCD gene-therapy scope. Use FDA’s Casgevy product page for STN 125787 indication, package insert, and approval letter. Use Vertex’s release to cross-check manufacturer framing and SCD/TDT wording.

Official sources

FDA release: approval date, age expansion, and first pediatric SCD gene-therapy scope

FDA Casgevy page: STN 125787 indication and approval documents

Vertex release: manufacturer explanation of expanded indication