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The first CRISPR treatment for sickle cell disease has been submitted to the Food and Drug Administration, beating a rival gene therapy that’s been delayed due to a manufacturing issue.

On Monday, Vertex Pharmaceuticals and its partner CRISPR Therapeutics said they had met their previously announced goal of filing by the end of March a marketing application for their therapy known as exa-cel. In addition to sickle cell disease, the two companies are seeking approval to treat beta-thalassemia, another type of inherited blood disease, but one that’s rarer in the U.S.


The submission gives Vertex and CRISPR Therapeutics an early lead over Bluebird Bio, which missed a first-quarter deadline to file FDA paperwork for its sickle cell gene therapy, called lovo-cel. Bluebird said last week that its application is complete but can’t be formally filed until the FDA provides the company with additional clearance for a step in its manufacturing process. The agency could take a few weeks to respond, Bluebird said, although there’s no firm timeline.

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