UK Authorizes Revolutionary Gene Therapy, Casgevy: A Potential Cure for Sickle Cell Disease

November 16, 2023
UK Authorizes Revolutionary Gene Therapy, Casgevy: A Potential Cure for Sickle Cell Disease
  • The MHRA, the UK's medicines regulator, has authorized the use of Casgevy, a gene therapy for sickle cell disease and beta thalassemia.

  • Casgevy uses the gene-editing tool Crispr to modify the patient's own stem cells, eliminating the need for immune compatibility and immunosuppression.

  • Clinical trials have shown promising results, with patients seeing healthy hemoglobin production restored and a decrease in symptoms.

  • Regulators in other countries, including the US, are expected to approve the therapy.

  • Concerns persist about the cost and accessibility of the treatment, especially in Africa where most sickle cell disease cases are found.

  • The therapy is also currently under review in the US and the European Union.

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