Breaking News: UK Approves First Gene Therapy Treatment for Sickle Cell Disease
In a medical breakthrough, the United Kingdom’s Medicines and Healthcare Regulatory Agency (MHRA) has approved Casgevy as the first gene therapy treatment for sickle cell disease and thalassemia. This revolutionary treatment developed by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics uses the cutting-edge CRISPR gene-editing tool, previously awarded the Nobel Prize in 2020 according to Time. The approval by the MHRA has been hailed by experts as a “positive moment in history” and offers new hope to thousands of people affected by these devastating conditions.
The therapy is intended to benefit patients 12 and older by offering an alternative to the traditional bone marrow transplants which, while effective, are often associated with complex procedures and unpleasant side effects. Casgevy aims to relieve patients from the pain episodes of sickle cell disease and the need for regular blood transfusions for thalassemia patients, offering the potential for a cure.
The treatment utilizes the CRISPR-Cas9 gene-editing technique to modify a gene in bone marrow stem cells in a lab. However, patients must first undergo a “conditioning treatment,” which may include immunosuppressants, radiation, or chemotherapy, to prepare their bone marrow for receiving the modified cells. Following this, patients may spend at least one month in a hospital facility while the treated cells settle in the bone marrow.
While the UK has made history by approving Casgevy, Europe and the United States are expected to follow suit. The US Food and Drug Administration is set to make a decision by December 8 after an advisory group certified the treatment as safe. Moving forward, Vertex and CRISPR Therapeutics will need to provide additional safety and effectiveness data to maintain the MHRA approval. It’s estimated that around 2,000 individuals in the UK could benefit from this ground-breaking gene therapy.
Sickle cell disease mainly affects African and Caribbean populations, while beta-thalassemia primarily impacts Mediterranean, South Asian, Southeast Asian, and Middle Eastern individuals. The approval of Casgevy has the potential to significantly improve the lives of patients affected by these lifelong and in some cases fatal conditions.
As the UK leads the way in groundbreaking medical advancements, the momentum for improving patient care and treatment options is expected to grow across the globe.
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