MHRA authorises gene therapy to cure sickle-cell disease and thalassemia

Article summary

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised a new treatment for sickle-cell disease and transfusion-dependent β-thalassemia for patients aged 12 and above. Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin. It is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory. Patients must then undergo conditioning treatment to prepare the bone marrow before the modified cells are infused back into the patient. Clinical trials are ongoing and further results will be made available in due course. The decision to authorise Casgevy has been endorsed by the Commission on Human Medicines, after a robust review of the available evidence.