Latest news

• bluebird bio sells second FDA priority review voucher for $95m

  Skysona (elivaldogene autotemcel) for the treatment of early, active cerebral adrenoleukodystrophy last year.

• FDA lifts partial clinical hold on bluebird bio’s sickle cell gene therapy studies

  The company’s Skysona (elivaldogene autotemcel) gene therapy was granted accelerated approval by the FDA in September this year to slow the progression of neurologic dysfunction in boys four to 17

• bluebird bio sells Rare Pediatric Disease Priority Review Voucher

  The company was granted two PRVs upon the FDA approvals of Zynteglo and Skysona. ... Prior to the approval of Skysona, effective options were limited to allogeneic haematopoietic stem cell transplant.

• bluebird bio’s Skysona approved by FDA to treat cerebral adrenoleukodystrophy

  Prior to the approval of Skysona, effective options were limited to allogeneic hematopoietic stem cell transplant (allo-HSCT). ... Altogether, three patients in Skysona’s clinical programme showed MDS, which is believed to be related to the lentiviral

• EU approves bluebird bio’s CALD gene therapy Skysona

  The European Commission (EC) has approved bluebird bio’s gene therapy Skysona for the rare inherited neurological disease cerebral adrenoleukodystrophy (CALD). ... Skysona is designed to add functional copies of the ABCD1 gene into a patient’s