And Bluebird might be right on its heels

Speaking of sickle cell disease, Bluebird Bio is developing a gene therapy for the disorder, and it just presented some data showing that it can both boost hemoglobin and improve outcomes for patients.

As STAT's Adam Feuerstein explains, Bluebird disclosed data from four patients, all of whom have been free of blood transfusions and painful flare-ups for at least six months after treatment.

The company is extending a mid-stage trial and plotting a late-stage one for next year. If everything goes according to plan, Bluebird would be able to make its case to the FDA with full data from the former and interim results from the latter.

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