The United Kingdom has granted regulatory approval for Casgevy, the first CRISPR gene editing treatment aimed at sickle cell disease and beta thalassemia. This groundbreaking decision by the Medicines and Healthcare products Regulatory Agency (MHRA) marks a significant advancement in genetic medicine, as both conditions are traditionally managed through bone marrow transplants, which carry risks and require closely matched donors. Casgevy has demonstrated the ability to restore healthy hemoglobin production in clinical trials, providing a potential cure for patients suffering from these debilitating genetic disorders.
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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.”