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bluebird bio to Present Data from Clinical Development Program of Lenti-D™ Gene Therapy for Cerebral Adrenoleukodystrophy (CALD) at the 13th European Pediatric Neurology Society (EPNS) Congress

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

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep. 3, 2019-- bluebird bio, Inc. (Nasdaq: BLUE) announced that updated data from the clinical development program for its investigational Lenti-D™ gene therapy in patients with cerebral adrenoleukodystrophy (CALD) will be presented at the 13th European Pediatric Neurology Society (EPNS) Congress, to be held September 17 – 21, 2019 in Athens, Greece.

Data will include updates from the ongoing Phase 2/3 Starbeam study (ALD-102) which assesses the efficacy and safety of Lenti-D gene therapy in boys 17 years of age and under with CALD. The company will also present updated 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.

Oral Presentations at EPNS

Phase 2/3 Trial to Assess the Safety and Efficacy of Lenti-D Hematopoietic Stem Cell Gene Therapy for Cerebral Adrenoleukodystrophy

Presenter: Dr. Caroline Sevin, Hôpital Universitaire Bicêtre-Paris Sud, Paris, France
Date & Time (Parallel Session 2D:Neurometabolic Disorders I): Wednesday, September 18, 2019, 4:30 – 6:15 p.m. GMT+3 (9:30 – 11:15 a.m. EDT)

An observational study of outcomes of Allogeneic Hematopoietic Stem Cell Transplant in patients with Cerebral Adrenoleukodystrophy (CALD)

Presenter: Dr. Florian Eichler,Center for Rare Neurological Diseases, Associate Professor of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, USA
Date & Time (Parallel Session 2D:Neurometabolic Disorders I): Wednesday, September 18, 2019, 4:30 – 6:15 p.m. GMT+3 (9:30 – 11:15 a.m. EDT)

Clinical Development Program for Lenti-D

The Lenti-D clinical development program is assessing the efficacy and safety of an investigational gene therapy in boys up to 17 years of age with CALD. Gene therapy for CALD consists of the patient’s own stem cells transduced with Lenti-D lentiviral vector that encodes ABCD1 cDNA for human adrenoleukodystrophy protein (ALDP). This allows for the production of functional ALDP that enables the local degradation of very long chain fatty acids (VLCFAs) in the brain. Buildup of VLCFAs in the central nervous system contributes to neurodegeneration in CALD.

The Phase 2/3 Starbeam study has completed enrollment. Visit for more information.

bluebird bio is currently enrolling a Phase 3 study (ALD-104) designed to assess the efficacy and safety of Lenti-D in boys 17 years of age or younger with CALD after myeloablative conditioning with busulfan and fludarabine. Visit for more information and a list of study sites.

bluebird bio is also conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have completed two years of follow-up in bluebird bio-sponsored studies of Lenti-D for CALD (ALD-102 or ALD-104). Visit for more information.

In addition, the company continues to conduct ongoing observational study (ALD-103) of allo‑HSCT in boys 17 years of age or younger with CALD. Visit for more information.

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

The United States Food and Drug Administration granted Lenti-D Orphan Drug status for the treatment of ALD, and Rare Pediatric Disease designation and Breakthrough Therapy designation for the treatment of CALD.

About Cerebral 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 ALDP and subsequently cause toxic accumulation of VLCFAs, primarily in the adrenal cortex and white matter of the brain and spinal cord.

Approximately 35-40 percent of boys with ALD will develop 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 allo-HSCT. Beneficial effects have been reported if allo-HSCT is performed early in the course of cerebral progression. Potential complications of allo-HSCT, which can be fatal, include graft failure, GvHD, and opportunistic infections, particularly in patients who do not receive transplant using cells from a matched sibling donor.

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 U.S., 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. Outside the U.S., the Minister of Health in the Netherlands has approved the addition of adrenoleukodystrophy to the newborn screening program, and a pilot will start in 2019.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, we’re developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, we’re working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. We’re putting our care and expertise to work across a spectrum of disorders by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent β-thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

Lenti-D and bluebird bio are trademarks 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 development and commercial 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 efficacy and safety results for our Lenti-D product candidate from the Starbeam Study seen to date 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 U.S. 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.

Elizabeth Pingpank, 617-914-8736


Victoria von Rinteln, 617-914-8774