Market Overview

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 –

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
& 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
& 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

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

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.

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