Bluebird lands another rare disease gene therapy OK — but there are a few caveats to note
Relative to the protracted journey of developing a gene therapy delivered by a lentiviral vector, the speed at which bluebird bio moved from having one approved product to two was extremely fast.
Exactly a month after the FDA gave its green light to bluebird’s Zynteglo — to treat beta thalassemia — the agency came through with an approval for a second gene therapy for early, active cerebral adrenoleukodystrophy (CALD). The drug, previously known as eli-cel and now branded Skysona, will have a wholesale acquisition price of $3 million and should become available by the end of this year.
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