CRISPR has been authorized for use as a gene therapy for sickle-cell disease and transfusion-dependent beta-thalassemia by the UK's Medicines and Healthcare products Regulatory Agency (MHRA). Not the FDA because this is the UK we're talking about.

"I am pleased to announce that we have authorised an innovative and first-of-its-kind gene-editing treatment called Casgevy, which in trials has been found to restore healthy haemoglobin production in the majority of participants with sickle-cell disease and transfusion-dependent beta-thalassaemia, relieving the symptoms of disease."

"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. To do this, stem cells are taken out of bone marrow, edited in a laboratory and then infused back into the patient after which the results have the potential to be life-long."

MHRA authorises world-first gene therapy that aims to cure sickle-cell disease and transfusion-dependent beta-thalassemia

#discoveries #crispr