Bluebird Exits Europe, Casting Clouds Over Gene Therapy Commercial Effort

bluebird bio announced two disappointing developments for its gene therapy business, casting a shadow over the planned separation of its gene therapies into a new standalone company in the fourth quarter. The company said it would wind down commercial initiatives for its gene therapy business in Europe after failing for two years to secure reimbursement for its approved gene therapy Zynteglo (betibeglogene autotemcel) for transfusion-dependent beta-thalassemia (TDT).

Bluebird also announced that the US Food and Drug Administration placed a clinical hold on its elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy for cerebral adrenoleukodystrophy (CALD) due to a report of myelodysplastic syndromes (MDS) in a patient treated with eli-cel for CALD over a year ago in the Phase III ALD-104 study