In a world first, Vertex, CRISPR win UK approval for CRISPR-edited therapy to treat sickle cell disease, beta-thalassemia
Vertex and CRISPR Therapeutics have won the first-ever approval for a therapy created with CRISPR gene editing, as the UK drug regulator MHRA greenlights their treatment for two blood disorders.
The therapy previously known as exa-cel will now be branded Casgevy. It is authorized to treat sickle cell disease and transfusion-dependent beta-thalassemia.
“Today is a historic day in science and medicine: this authorization of Casgevy in Great Britain is the first regulatory authorization of a CRISPR-based therapy in the world,” said Vertex CEO Reshma Kewalramani in a Tuesday statement.
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