UKNoticeOfficial ✓October 30, 2026· GOV.UK
MHRA approves gene-editing treatment for children aged five and over with serious inherited blood disorders
What happened
The MHRA has extended its UK authorisation for the treatment Casgevy (exagamglogene autotemcel). This decision approves the therapy for children aged five and over with severe sickle-cell disease and transfusion-dependent β-thalassemia.
Why it matters
Children aged five and over with severe sickle-cell disease can now be treated with Casgevy in the UK. Children aged five and over with transfusion-dependent β-thalassemia can also now receive the treatment.
Receipts
The receipts are official. The summary is ours.
Read on GOV.UK ↗Summaries are generated from the official text and may simplify or omit nuance. The official document is the source of truth.
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