Vertex’s First Crispr Gene Editing Therapy Gets EU Backing
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Europe’s health regulator followed the US and UK in backing the first gene-editing therapy to use Crispr technology, a Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG treatment for sickle cell disease.
The European Medicines Agency’s expert panel recommended on Friday authorizing the Vertex and Crispr drug, Casgevy, for people with severe sickle cell disease and another serious hereditary blood disorder, beta-thalassemia, which is traditionally treated with repeated transfusions. Vertex said before the ruling that it had yet to establish a European list price for the one-time therapy, which costs $2.2 million in the US.