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Could This Move Make Vertex's Next Blockbuster Even Bigger?


Vertex Pharmaceuticals (NASDAQ: VRTX) recently reported great news. It completed the submission of exa-cel, its treatment candidate for blood disorders, to the U.S. Food and Drug Administration (FDA). The company and partner CRISPR Therapeutics (NASDAQ: CRSP) wrapped up submissions to Europe and the U.K. late last year.

The companies aim for approval in adults suffering from beta thalassemia or sickle cell disease. Regulatory decisions could come as soon as this year. Vertex has already prepared the infrastructure to launch the treatment -- and it could become the company's next blockbuster. But things may get even better. Could a recent move by Vertex make the therapy bigger down the road?

First, a bit of detail about exa-cel. It's a gene-editing treatment using CRISPR Therapeutics' cutting-edge CRISPR/Cas9 technology. This involves cutting DNA in a particular spot and letting the natural repair process take over.

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Source Fool.com

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