01/11/2026
Casgevy works by editing a faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin. The stem cells are collected from the patient, edited in a laboratory and then infused back into the body following conditioning treatment.
🇬🇧 : UK approval now includes eligible children aged 5+
Younger with transfusion-dependent β- or severe disease are now included in Casgevy’s authorisation.
MHRA, the UK regulator for medicines and medical devices, granted the extension on 29 October and announced it today, lowering the approved minimum age from twelve to five years of age.
Casgevy is a gene-editing treatment using the patient’s own stem cells. Treatment involves conditioning therapy, specialist hospital care and continued safety monitoring.
The approved minimum age is two in the and remains 12 in the . Clinical eligibility criteria apply in each jurisdiction.
🔗 Read the full MHRA announcement:
https://www.gov.uk/government/news/mhra-approves-gene-editing-treatment-for-children-aged-five-and-over-with-serious-inherited-blood-disorders