The first medical treatment using Crispr gene-editing technology, called Casgevy, has been approved for sickle cell disease and beta thalassemia in several countries. This breakthrough treatment works by modifying a person’s cells to produce healthy hemoglobin, offering hope to approximately 35,000 patients across the US and Europe.
Overview of Casgevy and its Approval
Casgevy is the first medical treatment that uses the gene-editing technology Crispr, which was approved for sickle cell disease and a related blood disorder called beta thalassemia. UK regulators approved the treatment in November 2023, followed by the US and Europe in December.
Key Points about Casgevy
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The treatment works by using Crispr to modify a person’s own cells so that they produce a healthy type of hemoglobin.
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In clinical trials, Casgevy greatly reduced or eliminated debilitating pain crises in people with sickle cell disease, and it allowed most people with beta thalassemia to end blood transfusions.
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Vertex estimates that around 35,000 sickle cell and beta thalassemia patients across the US and Europe could benefit from Casgevy.
Challenges for Patients Interested in Getting Casgevy
Some patients interested in getting Casgevy face challenges such as:
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The need to undergo chemotherapy, which can cause infertility.
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The long and arduous process of cell collection and staying in the hospital for weeks after receiving the edited cells.
Progress of Casgevy Distribution
According to Vertex, 45 treatment centers are now authorized to administer Casgevy, and the company expects this number to grow to approximately 75 around the world.
