Sickle cell disease and beta-thalassaemia are caused by different mutations in a single gene that codes for haemoglobin, an essential blood molecule that helps red blood cells carry oxygen around the body.
The UK Parliament has published a research briefing detailing how these conditions are treated, and what challenges patients face.
The report also discusses the use of CRISPR-based genome editing drugs, such as Casgevy, also called exa-cel, which was approved by the Medicines and Healthcare products Regulatory Agency for the treatment of sickle cell disease and transfusion-dependent beta-thalassaemia in patients 12 years of age and older (see BioNews 1216). The National Institute for Health and Care Excellence's independent appraisal committee has since approved the use of Casgevy on the NHS in England (see BioNews 1275).
Read the full report here.