Casgevy, a CRISPR-based gene therapy, was one of the first cell-based gene therapies for the treatment of sickle cell disease in patients 12 years and older to be granted approval by the US Food and Drug Administration (FDA) (see BioNews 1220).
Now, the FDA has approved the same drug for the treatment of patients 12 years and older with the most severe form of beta-thalassaemia, transfusion-dependent thalassaemia, which is a rare blood disorder requiring blood transfusions.
Read more in Reuters.