STAT+: Bluebird submits sickle cell gene therapy to FDA for approval

Gene therapy developer Bluebio 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-cell, 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.

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