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BioWorld - Wednesday, December 17, 2025
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Home » Bluebird wins FDA nod for one-time gene therapy for CALD, sets $3M price tag
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Bluebird wins FDA nod for one-time gene therapy for CALD, sets $3M price tag

Sep. 19, 2022
By Jennifer Boggs
Bluebird Bio Inc.’s elivaldogene autotemcel (eli-cel) gained U.S. approval late Sept. 16 for use in early active cerebral adrenoleukodystrophy (CALD), making it the firm’s second gene therapy to clear the FDA in as many months. Branded Skysona, eli-cel is expected to be available commercially by the end of 2022 and its launch will require only “incremental” company resources on top of those required for the ongoing launch of beta-thalassemia gene therapy Zynteglo (betibeglogene autotemcel), Bluebird said.
BioWorld Regulatory Neurology/psychiatric Gene therapy U.S. FDA

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