Today, the United Kingdom became the first country to give regulatory approval to a medical procedure that uses CRISPR gene editing. The Medicines and Healthcare products Regulatory Agency (MHRA) approved Casgevy, a therapy that will be used to treat sickle cell disease and beta thalassemia (also called β -thalassaemia).
[Related: CRISPR breaks ground as a one-shot treatment for a rare disease.]
What are sickle cell disease and beta thalassemia?
Both diseases are painful, life-long genetic conditions that are caused by errors in the genes for a protein called hemoglobin. Red blood cells use hemoglobin to carry oxygen around the body. Sickle cell disease is particularly common among people with Caribbean or African ancestry. The abnormal hemoglobin makes the blood cells crescent-shaped and hard. The misshapen cells then clump together and block the flow of oxygen to the organs, which causes extreme pain. The cells can then die off early, which leads to anemia.
Beta thalassemia primarily affects patients with Mediterranean, south Asian, southeast Asian, and Middle Eastern backgrounds. It also causes anemia since the mody cannot make as much hemoglobin.
Casgevy was developed by Boston’s Vertex Pharmaceuticals and Switzerland’s Crispr Therapeutics and could be used to replace bone marrow transplants. The companies estimate that roughly 2,000 people in the UK are now eligible for the therapy.
“I am pleased to announce that we have authorized an innovative and first-of-its-kind gene-editing treatment called Casgevy, which in trials has been found to restore healthy hemoglobin production in the majority of participants with sickle-cell disease and transfusion-dependent beta thalassaemia, relieving the symptoms of disease,” interim executive director of healthcare quality and access at the MHRA Julian Beach said in a statement.
How does Casgey use CRISPR gene editing?
The new treatment uses the CRISPR-Cas9 gene…
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