Why in News

  • The U.S. Food and Drug Administration (FDA) approved a pair of gene therapies for sickle cell disease, including the first treatment based on the breakthrough CRISPR gene editing technology.

  • The agency approved Lyfgenia from bluebird bio, and a separate treatment called Casgevy by partners Vertex Pharmaceuticals and CRISPR Therapeutics for the illness.

What is Casgevy 

  • Casgevy AKA exagamglogene autotemcel.

  • It is a groundbreaking gene therapy treatment for sickle cell disease (SCD) and beta thalassemia.

  • These are two serious inherited blood disorders.

  • It utilizes the revolutionary CRISPR-Cas9 gene-editing technology.

Here's how Casgevy works:

  1. Stem cell extraction: The patient's bone marrow is extracted to collect CD34+ hematopoietic stem cells.

  2. Gene editingCRISPR-Cas9 technology is used to edit the defective gene in the stem cells.

3. Stem cell reintroduction: The modified stem cells are then infused back into the patient's bloodstream.

4. Production of healthy red blood cells: The edited stem cells begin producing healthy red blood cells, which alleviate the symptoms of SCD and beta thalassemia.

Benefits of Casgevy:

  • Potentially curative: Casgevy is considered a one-time treatment with the potential to cure these previously incurable diseases.

  • Improved quality of life: By correcting the underlying genetic defect, Casgevy can significantly improve the quality of life for patients by reducing pain, fatigue, and other symptoms.

  • Reduced transfusion dependence: Patients with beta thalassemia often require regular blood transfusions, which can be a burden. Casgevy can reduce or even eliminate the need for transfusions.



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Learnerz IAS | Concept oriented UPSC Classes in Malayalam: Casgevy UPSC NOTE
Learnerz IAS | Concept oriented UPSC Classes in Malayalam
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