These slides and the accompanying oral presentation contain forward-looking statements and information. The use of words such as "may," "might," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "project," "intend," "future," "potential," or "continue," and other similar expressions are intended to identify forward-looking statements. For example, all statements we make regarding the progress and results of our ongoing investigation into the two safety events recently reported in the HGB-206 clinical study, as well as potential regulatory interactions for the HGB-206 and HGB-210 clinical studies, as well as for marketing of ZYNTEGLO®, are forward looking. All forward-looking statements are based on expectations, estimates and assumptions by our management that, although we believe to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that we expected. These statements are also subject to a number of material risks and uncertainties that are described in our most recent annual report on Form 10-K, as well as our subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.
• Insertion in VAMP4 (Vesicle-associated membrane protein 4)
• No known role in cellular proliferation or genome stability
• Totality of public/published data consistent with no role in cancer
• VAMP4 insertions seen in the majority of HGB-206 patients without sequalae
• Vector-encoded β-globin promoter / enhancer is minimally active in tumor cells (based on T87Q expression)
• No gene misregulation across a ~1MB region surrounding vector integration site
Today
Looking Ahead
• AML case: vector unlikely to have played a role
• Further detail on MDS case
• MDS case: diagnosis under evaluation; no blasts, no dysplasia, clinically stable
• Engaged with regulators to resume HGB-206 and HGB-210
• Engaged with EMA to renew ZYTENGLO cMAA
• Clarity on resumption of clinical studies and EU marketing
• Planned company split on track
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Bluebird Bio Inc. published this content on 10 March 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 10 March 2021 15:54:03 UTC.
bluebird bio, Inc. is a biotechnology company. The Company is focused on researching, developing, and commercializing potentially curative gene therapies for severe genetic diseases based on its lentiviral vector (LVV) gene addition platform. Its lead gene therapy programs for sickle cell disease, B-thalassemia, and cerebral adrenoleukodystrophy and is advancing research to apply new technologies to these and other diseases. It has two gene therapies: ZYNTEGLO (betibeglogene autotemcel) and SKYSONA (elivaldogene autotemcel). ZYNTEGLO is the first gene therapy for people with B-thalassemia who require regular red blood cell transfusions. SKYSONA (elivaldogene autotemcel), also known as eli-cel, is used to slow the progression of eurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). It is also developing (lovotibeglogene autotemcel), also known as lovo-cel, as a one-time treatment for patients with sickle cell disease (SCD).
BLUEBIRD BIO, INC. 
