Skysona (elivaldogene autotemcel), bluebird bio’s gene therapy for cerebral adrenoleukodystrophy (CALD), is set to win its first ever marketing authorization now that the European Medicines Agency has recommended that the one-time treatment for the potentially fatal neurodegenerative disease be approved throughout the EU.
Skysona will be the first one-time gene therapy approved to treat CALD, a rare disease that occurs almost exclusively in...








