Why in news: The United Kingdom’s drug regulator approves the world’s first gene therapy treatment for sickle cell disease and thalassemia.

About Casgevy

  • Casgevy by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics gets approval for patients 12 years old and over with sickle cell disease and thalassemia.

    • Both sickle cell disease and thalassemia stem from gene errors affecting haemoglobin, the oxygen-carrying protein in red blood cells.
    • Sickle cell disease is prevalent in regions with a history of malaria, like Africa and India, and is more common in certain ethnic groups, such as people of African, Middle Eastern, and Indian descent.
  • Previously, bone marrow transplants were the primary long-term treatment, despite their harsh side effects. But with this approval, Casgevy offers hope to thousands affected by these diseases in the UK.
  • The decision to greenlight this gene therapy was based on studies involving 29 sickle cell patients, where 28 reported no severe pain problems for at least a year post-treatment. In thalassemia, among 42 patients who received the therapy, 39 did not require a red blood cell transfusion for a year after treatment.
  • Casgevy functions by targeting the defective gene in a patient’s bone marrow stem cells, allowing the body to produce properly functioning haemoglobin.

For more information: How gene therapy will revolutionise treatment of sickle cell disease | Explained News – The Indian Express