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