Skysona® (elivaldogene autotemcel) is the first FDA approved therapy to slow the progression of neurologic dysfunction with early, active Cerebral Adrenoleukodystrophy (CALD) in boys 4- 17 years of age. Skysona, manufactured by bluebird bio, Inc., is a one-time gene therapy used to treat the underlying cause of CALD.

• It is anticipated that Skysona will be available by the end of 2022 and is a single-dose treatment.
• bluebird bio, has set the wholesale acquisition cost of Skysona in the U.S. at $3.0M per single- dose treatment.