Bluebird Bio gene therapy becomes first FDA-approved med for ultra-rare neuro disorder
Bluebird Bio gene therapy Skysona has won FDA approval, making it the first FDA-approved therapy for cerebral adrenoleukodystrophy, an ultra-rare and fatal neurological disorder. Skysona’s regulatory green light follows the August approval of Zynteglo, a Bluebird gene therapy for a rare blood disorder.