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The NHS in England has launched a groundbreaking gene therapy for sickle cell disease, offering hope for a potential cure for patients with this life-threatening condition. The treatment, known as Casgevy or Exa-cel, costs £1.65 million and will initially be available to around 50 patients annually. The therapy uses CRISPR gene-editing to modify blood stem cells, enabling the body to produce healthy red blood cells. It specifically enhances the production of fetal haemoglobin, which is unaffected by sickle cell disease, thus alleviating symptoms and preventing hospitalizations.
NHS chief Amanda Pritchard called the treatment "transformative," offering patients the possibility of living free from the debilitating pain crises associated with sickle cell. The treatment will be available in specialist centres for patients aged 12 and above who experience recurrent sickle cell crises and do not have access to a stem cell transplant. While this advancement is hailed as a milestone by campaigners and patients alike, there is still a need to ensure that all those with sickle cell have access to the care they deserve.
This therapy, which has already been used to treat another blood disorder, transfusion-dependent beta thalassemia, is a significant step forward in gene therapy and its potential to cure genetic diseases.
