Gene therapy developer Bluebird Bio said Monday that it has submitted a long-awaited application asking the Food and Drug Administration to approve its drug for sickle cell disease, setting up competition with Vertex Pharmaceuticals and CRISPR Therapeutics.
Bluebird’s treatment, called lovo-cel, is a gene therapy that uses an engineered virus to insert a modified gene into the DNA of a patient’s stem cells. The gene produces normally functioning hemoglobin, which patients with sickle cell disease lack. Instead, their oxygen-carrying hemoglobin is rigid, sticky, and misshapen, and can block blood vessels, inhibiting the flow of oxygen through the body.
The company had originally planned to file for FDA approval by the end of the first quarter, but reported in March that it was delayed until the FDA provided the company with additional clearance for a step in its manufacturing process.
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