UK MHRA approves Casgevy for sickle cell disease and beta thalassemia, the first approval of a CRISPR-based medicine

CRISPR Therapeutics/Casgevy (exa-cel)

The UK medicines regulator authorised Casgevy for patients aged 12+ with sickle cell disease or transfusion-dependent beta thalassemia on 17 November 2023. It edits blood stem cells to restore fetal hemoglobin and was the world's first authorisation of a CRISPR-based medicine.

• Clinical evidence: 28 of 29 SCD patients (97%) were free of severe pain crises for ≥12 months post-treatment.
• For TDT: 39 of 42 evaluated patients (93%) did not require red-blood-cell transfusions for ≥12 months.
• Treatment requires stem-cell collection, ex vivo CRISPR editing, and reinfusion after bone-marrow conditioning.
• World-first CRISPR approval positioned UK as leader in advanced gene-therapy regulation and access.