Medical CRISPR treatment has been greenlit in UK in global first (for sickle cell disease and beta thalassemia)

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The UK has made a significant advancement in medical treatment by becoming the first country to approve a CRISPR gene editing therapy called Casgevy for sickle cell disease and beta thalassemia. The Medicines and Healthcare products Regulatory Agency (MHRA) announced this approval, highlighting that both conditions are caused by genetic errors affecting hemoglobin production, leading to severe health issues. Currently, bone marrow transplants are the only permanent treatment option, which comes with risks. Casgevy has shown promise in clinical trials, successfully restoring healthy hemoglobin levels in many patients, thereby alleviating symptoms associated with these lifelong conditions. This development marks a pivotal moment in gene therapy and its potential to transform treatment for genetic disorders.
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Interesting developments; I'm not sure how controversial this is...

https://www.cnn.com/2023/11/16/heal...rispr-gene-editing-sickle-cell-scn/index.html

CNN —

The United Kingdom has become the first country to give regulatory approval to a medical treatment involving the revolutionary CRISPR gene editing tool.

The country’s Medicines and Healthcare products Regulatory Agency said Thursday it had given a greenlight to a treatment known as Casgevy, which will be used to treat sickle cell disease and beta thalassemia. Both genetic conditions are caused by errors in the genes for hemoglobin, which is used by red blood cells to carry oxygen around the body. There is no known universally successful treatment for either disorder.

Sickle cell disease, which can result in attacks of debilitating pain, is more common in people with an African or Caribbean family background. Beta thalassemia mainly affects people of Mediterranean, South Asian, Southeast Asian and Middle Eastern origin, the statement said.
A worker from the Community Blood Center hangs a bag of blood during a transfusion for Kevin Wake at the Sickle Cell Center at University Health on March 7, 2023. (Tammy Ljungblad/The Kansas City Star/Tribune News Service via Getty Images)

FDA considers first CRISPR gene editing treatment that may cure sickle cell

“Both sickle cell disease and β-thalassemia are painful, life-long conditions that in some cases can be fatal. To date, a bone marrow transplant — which must come from a closely matched donor and carries a risk of rejection — has been the only permanent treatment option,” said Julian Beach, the interim executive director of healthcare quality and access at the MHRA, in a statement.

“I am pleased to announce that we have authorised an innovative and first-of-its-kind gene-editing treatment called Casgevy, which in trials has been found to restore healthy haemoglobin production in the majority of participants with sickle-cell disease and transfusion-dependent β-thalassaemia, relieving the symptoms of disease.”
 
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