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Monday, March 15, 2021

bluebird bio Has Long-Term Data for elivaldogene autotemcel (eli-cel, Lenti-D™) Gene Therapy

 

90% of evaluable patients (27/30) alive and free of major functional disabilities (MFDs) at two years follow-up in Phase 2/3 Starbeam study (ALD-102)

Patients in long-term follow-up study (LTF-304) continue to remain alive and MFD-free through up to nearly seven years of follow-up, suggesting eli-cel stabilizes the progression of disease

No reports of graft failure, graft rejection, graft-versus-host disease, replication competent lentivirus or insertional oncogenesis in the 51 patients treated with eli-cel in clinical studies (ALD-102/LTF-304 and ALD-104)

Data presented in oral session during Presidential Symposium at the 47th Annual Meeting of the EBMT

bluebird bio, Inc. (Nasdaq: BLUE) announced new data from the clinical development program for its investigational elivaldogene autotemcel (eli-cel, Lenti-D™) gene therapy in patients with cerebral adrenoleukodystrophy (CALD), including updated results from the pivotal Phase 2/3 Starbeam study (ALD-102) and the long-term follow-up study LTF-304, as well as safety outcomes from the Phase 3 ALD-104 study. Data were presented today in an oral presentation during the Presidential Symposium at the 47th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2021), taking place virtually from March 14 - 17, 2021.

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