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bluebird bio to Present Updated Data for Lenti-D™ Gene Therapy for Cerebral Adrenoleukodystrophy at SSIEM 2018 Symposium

− Data Include Updates from Phase 2/3 Starbeam (ALD-102) Study of Lenti-D for Cerebral Adrenoleukodystrophy (CALD) and Initial Data from Ongoing Observational Study (ALD-103) of Allogeneic Hematopoietic Stem Cell Transplant for CALD –

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Aug. 3, 2018-- bluebird bio, Inc. (Nasdaq: BLUE) announced that new data from its clinical development program for its investigational Lenti-D™ gene therapy in pediatric patients with cerebral adrenoleukodystrophy (CALD) will be presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2018 Symposium held September 4 - 7, 2018 in Athens, Greece.

Updated data from the ongoing Phase 2/3 Starbeam study (ALD-102) assessing the efficacy and safety of Lenti-D gene therapy in boys 17 years of age and under with CALD will be presented. The company will also present initial data from its ongoing observational study (ALD-103) of allogeneic hematopoietic stem cell transplant (allo-HSCT) in boys 17 years of age and under with CALD.

“We continue to advance our Lenti-D gene therapy development program for CALD and are pleased by the opportunity to share new data from two of our studies at SSIEM,” said David Davidson, M.D., chief medical officer, bluebird bio. “In addition to updated Lenti-D clinical results from the ALD-102 treatment study, we will for the first time present interim data from our ALD-103 observational study of patients treated with allogeneic hematopoietic stem cell transplant for CALD. We hope that improving the understanding of outcomes with allo-HSCT will help patients, families and healthcare providers when making treatment choices.”

Oral Presentations

Lenti-D hematopoietic stem cell gene therapy for cerebral adrenoleukodystrophy: safety and efficacy outcomes from an ongoing Ph 2/3 trial

Presenter: Paul Gissen, MBChB, Ph.D., FRCPCH, Pediatric Metabolic Diseases at Great Ormond Street Hospital, London, United Kingdom
Date & Time:Wednesday, September 5, 2018, 9:00 – 10:30 a.m. EEST (2:00 – 3:30 a.m. EDT)

An observational study of patients with cerebral adrenoleukodystrophy (CALD) treated with allogeneic hematopoietic stem cell transplant

Presenter: Robert Chiesa, M.D., Pediatric Bone Marrow Transplantation at Great Ormond Street Hospital, London, United Kingdom
Date & Time:Wednesday, September 5, 2018, 9:00 – 10:30 a.m. EEST (2:00 – 3:30 a.m. EDT)

Clinical Development Program for Lenti-D

bluebird bio’s clinical development program for Lenti-D gene therapy includes the ongoing Starbeam ALD-102 study with sites in France, the United Kingdom and the United States. The study is designed to assess the safety and efficacy of investigational Lenti-D gene therapy in boys 17 years of age and under with cerebral adrenoleukodystrophy (CALD). For more information visit: or using identifier NCT01896102.

In addition, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-304) for boys who have participated in ALD-102.

The European Medicines Agency (EMA) accepted Lenti-D gene therapy for the treatment of CALD into its Priorities Medicines scheme (PRIME) in July 2018, and previously granted Orphan Medicinal Product designation to Lenti-D.

The U.S. Food and Drug Administration (FDA) also granted Lenti-D Orphan Drug status, Rare Pediatric Disease designation and Breakthrough Therapy designation for the treatment of CALD.

About Cerebral Adrenoleukodystrophy

Adrenoleukodystrophy (ALD) is a rare, X-linked metabolic disorder that is estimated to affect one in 21,000 male newborns worldwide. ALD is caused by mutations in the ABCD1 gene that affect the production of adrenoleukodystrophy protein (ALDP) and subsequently cause toxic accumulation of very long chain fatty acids (VLCFAs) in the adrenal cortex and white matter of the brain and spinal cord.

Approximately 35-40 percent of boys with ALD will develop cerebral ALD (CALD), the most severe form of ALD. CALD is a progressive neurogenerative disease that involves breakdown of myelin, the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control. Symptoms of CALD usually occur in early childhood and progress rapidly, if untreated, leading to severe loss of neurologic function, and eventual death, in most patients.

Currently, the only therapeutic option for patients with CALD is allogeneic hematopoietic stem cell transplant. Beneficial effects have been reported if allogeneic hematopoietic stem cell transplant is performed early in the course of CALD progression. Potential complications of allogeneic hematopoietic stem cell transplant, which can be fatal, include graft failure, graft-versus-host disease, and opportunistic infections, particularly in patients who undergo transplant with non-sibling matched donor cells.

Early diagnosis of CALD is important, as the outcome of treatment varies with the clinical stage of the disease at the time of transplant. Newborn screening for ALD is a critical enabler of early diagnosis and successful treatment of ALD. In the United States, newborn screening for ALD was added to the Recommended Universal Screening Panel in February 2016 but is currently active in only a limited number of states.

About bluebird bio, Inc.

With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio (Nasdaq: BLUE) has built a pipeline with broad potential application in severe genetic diseases and cancer.

bluebird bio's gene therapy clinical programs include investigational treatments for cerebral adrenoleukodystrophy, transfusion-dependent β-thalassemia, also known as β-thalassemia major, and severe sickle cell disease.

bluebird bio's oncology pipeline is built upon the company's lentiviral gene delivery and T cell engineering, with a focus on developing novel T cell-based immunotherapies, including chimeric antigen receptor (CAR T) and T cell receptor (TCR) therapies. The company’s lead oncology programs are anti-BCMA CAR T programs partnered with Celgene.

bluebird bio’s discovery research programs include utilizing megaTAL/homing endonuclease gene editing technologies with the potential for use across the company's pipeline.

bluebird bio has operations in Cambridge, Massachusetts; Seattle, Washington; Durham, North Carolina and Zug, Switzerland.

Lenti-D is a trademark of bluebird bio, Inc.

Forward-Looking Statements

This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the clinical, regulatory approval, and market potential of the Company’s Lenti-D product candidate to treat cerebral adrenoleukodystrophy. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, risks that the preliminary efficacy and safety data for our Lenti-D product candidate from the Starbeam Study will not continue or persist, the risk of cessation or delay of any of the ongoing clinical studies and/or our development of Lenti-D, the risks regarding future potential regulatory approvals of Lenti-D, including the risk that the Starbeam Study will be insufficient to support regulatory submissions or marketing approval in the US and EU, and the risk that any one or more of our product candidates will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

Source: bluebird bio, Inc.

bluebird bio
Elizabeth Pingpank, 617-914-8736
Catherine Falcetti, 339-499-9436