Almost three years after the world’s first approval of a CRISPR medicine, the gene scissors are reaching paediatrics: in July 2026, the US Food and Drug Administration (FDA) extended the use of Casgevy (exagamglogene autotemcel, exa-cel for short) to children aged two and over with sickle cell disease or transfusion-dependent beta thalassaemia.
How Casgevy works
Both diseases are caused by defects in the gene for adult haemoglobin. Casgevy uses a clever detour: blood-forming stem cells are taken from the patients. In the laboratory, CRISPR-Cas9 switches off a genetic switch (BCL11A) that normally stops the production of fetal haemoglobin after birth. After conditioning chemotherapy, the patients receive their modified cells back – and produce fetal haemoglobin again, which takes over the function of the defective adult haemoglobin.
What the studies show
In the pivotal trials, 16 of 17 evaluable people with sickle cell disease were subsequently free of painful vaso-occlusive crises. In beta thalassaemia, 25 of 27 treated patients no longer needed regular blood transfusions. According to the manufacturers CRISPR Therapeutics and Vertex, the extension to younger children could make around 5,500 additional patients eligible.
Availability and costs
- Casgevy is now approved in 39 countries in North America, Europe and the Middle East.
- In Germany, reimbursement for patients aged twelve and over was achieved in May 2026.
- Applications for approval for the 5 to 11 age group have been submitted in the UK and Saudi Arabia, among other countries.
- The list price in the USA is around 2.2 million US dollars.
- Revenue rose to 76 million US dollars in the second quarter of 2026 – a sign that the treatment is slowly reaching routine care.
The limitations
Despite its successes, the therapy remains demanding: it is only possible in specialised centres and requires stem cell collection and a gruelling chemotherapy (conditioning), which can, among other things, impair fertility. For families, this means weeks in hospital. Research is therefore working on procedures in which the gene scissors act directly in the body – without cell collection and without chemotherapy.
Conclusion
The extension to young children is an important step: the earlier the disease is stopped, the less organ damage can occur. At the same time, price and effort show that access to gene editing is still very unevenly distributed worldwide – especially in regions where sickle cell disease is most common.
Note: This article is for general information only and does not replace medical advice.
