Casgevy, a CRISPR-based gene therapy, was one of the first cell-based gene therapies granted approval by the the Medicines and Healthcare products Regulatory Agency in the UK for the treatment of sickle cell disease and transfusion-dependent beta-thalassemia in patients 12 years and older (see BioNews 1216)
However, the gene therapy has not been approved for widespread NHS use for the treatment of sickle cell in draft guidance. The National Institute for Health and Care Excellence said it needed more information before a decision could be made.
The Independent has more.
