bluebird bio Presents New Data from Northstar (HGB-204) and Northstar-2 (HGB-207) Studies of LentiGlobin Gene Therapy in Patients with Transfusion-Dependent ?-Thalassemia at Annual Congress of the European Hematology Association

bio, Inc. (Nasdaq: BLUE) announced that new data from the completed
Phase 1/2 Northstar (HGB-204) study in adolescents and adults with
transfusion-dependent ?-thalassemia (TDT) and any genotype, and its
ongoing, Phase 3 Northstar-2 (HGB-207) multicenter clinical study of
LentiGlobin™ investigational gene therapy in patients with TDT and non-?0/?0
genotypes, will be presented in an oral session on June 16 at the
23rd Annual Congress of the European Hematology Association
by Franco Locatelli, M.D., Ph.D., of the IRCCS Ospedale Pediatrico
Bambino Gesù of Rome, Italy.

“The maturing data from HGB-204 and HGB-207 suggest that one-time
treatment with LentiGlobin may address the underlying genetic cause
of TDT. With our refined manufacturing process, the majority of patients
with TDT and non-?0/?0 genotypes are
transfusion-free and producing total hemoglobin at normal
or near-normal levels,” said David Davidson, M.D., chief medical
officer, bluebird bio. “We are on track to submit a marketing
authorization application in the European Union later this year, and we
continue to work closely with clinical investigators and regulatory
authorities to complete our ongoing clinical trials and bring this
important treatment option to patients as soon as possible.”

People with TDT need regular blood transfusions to survive, but chronic
transfusions carry risks, including unavoidable iron overload that can
result in multi-organ damage and shortened life expectancy. Eliminating
or reducing the need for transfusions may reduce the long-term
complications associated with TDT and current standards of care.

“Consistently higher in vivo vector copy numbers and HbAT87Q
hemoglobin levels in patients indicate that LentiGlobin manufacturing
refinements have resulted in improved gene therapy characteristics and
may enable sustained transfusion independence for a great majority of
patients,” said Professor Locatelli, the lead investigator of the
Northstar-2 study. “Further, we are now seeing more than three years of
data from the Northstar study indicating that LentiGlobin therapy may
enable long-term transfusion independence in the majority of patients
with non-?0/?0 genotypes. These results hold the
promise to change the natural history of many patients with this severe
genetic disorder of hemoglobin production.”

LentiGlobin Gene Therapy for Transfusion-Dependent ?-Thalassemia
(TDT) in Patients with Non-?0/?0
Genotypes: Updated Results from Northstar-2 (Abstract #1510)

Presenter: Franco Locatelli, M.D., Ph.D., Professor of
Pediatrics, University of Pavia, Italy and Director, Department of
Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino
Gesù, Rome, ItalyOral Session: Cell and Gene Therapy:
Clinical ResultsDate & Time: Saturday, June 16, 2018,
11:45 a.m. – 12:00 p.m. CEST (5:45 a.m. EDT)Location: Room

The safety and efficacy of LentiGlobin in patients with TDT were
evaluated in the Phase 1/2 Northstar study (HGB-204). To further improve
clinical results, a refined manufacturing process was used to produce
LentiGlobin drug product (DP). The aim of the ongoing Phase 3
Northstar-2 study (HGB-207) is to evaluate the efficacy and safety of
LentiGlobin DP with manufacturing refinements in patients with TDT and
non-?0/?0 genotypes. Data from Northstar and
Northstar-2 will be included in the oral presentation.

Northstar-2 (HGB-207) results include (as of May 15, 2018):

Northstar (HGB-204) results include (as of March 7, 2018):

Conference Call & Webcast Informationbluebird bio will
host a conference call and live webcast at 8:00 a.m. EDT on Friday, June
15, 2018. To access the live webcast, please visit the “Events &
Presentations” page within the Investors and Media section of the
bluebird bio website at
Alternatively, investors may listen to the call by dialing (844)
825-4408 from locations in the United States or +1 (315) 625-3227 from
outside the United States. Please refer to conference ID number 4678706.
A replay of the webcast will be available on the bluebird bio website
for 90 days following the call.

About the Northstar (HGB-204) StudyThe completed Phase 1/2
Northstar study was an open-label, single-dose, non-randomized,
multi-center study conducted in the United States, Australia and
Thailand. It was designed to evaluate the safety and efficacy of
LentiGlobin in increasing hemoglobin production and eliminating or
reducing transfusion dependence in subjects with transfusion dependent
beta-thalassemia. The study treated 18 adults and adolescents who are
being followed to evaluate safety and efficacy post-LentiGlobin
infusion. For more information on the Northstar study, please visit or using identifier

About the Northstar-2 (HGB-207) StudyNorthstar-2 is a Phase
3 global, multi-center study being conducted in the United States,
Thailand, Germany, Italy, the United Kingdom, and France designed to
evaluate the safety and efficacy of LentiGlobin drug product in patients
with transfusion-dependent beta-thalassemia and non-?0/?0
genotypes. For this study, the manufacturing process by which the
patient’s cells are transduced with the LentiGlobin viral vector has
been improved, with the intent of increasing vector copy number and the
percentage of cells successfully transduced.

The target enrollment of the study is 15 adult and adolescent patients
and 8 pediatric patients. The study’s primary endpoint is the proportion
of treated subjects who meet the definition of “transfusion
independence,” defined as total hemoglobin levels of at least 9 g/dL
without any red blood cell transfusions for a continuous period of at
least 12 months at any time during the study. For more information on
the Northstar-2 study, please visit or using identifier NCT02906202.

About LentiGlobinbluebird bio is developing LentiGlobin
with a goal of filing for regulatory approval in the United States and
the EU for TDT and for severe sickle cell disease (SCD). The company is
currently conducting four ongoing clinical studies of LentiGlobin with a
fifth that has recently completed. Studies currently ongoing include
HGB-205, a single center Phase 1/2 study in both TDT and SCD;
Northstar-2 (HGB-207) and Northstar-3 (HGB-212), both multi-center,
international Phase 3 studies for the treatment of patients with both
non-?0/?0 and ?0/?0 TDT
genotypes, respectively; and HGB-206, a multicenter Phase 1 study in the
United States for the treatment of patients with severe SCD. In
addition, bluebird is conducting a long-term safety and efficacy
follow-up study (LTF-303) for subjects with hemoglobinopathies (TDT or
severe SCD) who have been treated with LentiGlobin in bluebird
bio-sponsored clinical studies.

LentiGlobin was granted Orphan Drug status by the U.S. Food and Drug
Administration (FDA) and the European Medicines Agency (EMA) for the
treatment of ?-thalassemia and SCD. The FDA granted Breakthrough Therapy
designation to LentiGlobin for the treatment of transfusion-dependent
patients with ?-thalassemia major and Fast-Track Designation for the
treatment of beta-thalassemia major and for the treatment of certain
patients with severe SCD. bluebird bio is participating in the EMA’s
Adaptive Pathways pilot program, which is part of the EMA’s effort to
improve timely access for patients to new medicines. The EMA granted
Priority Medicines (PRIME) eligibility to LentiGlobin for the treatment
of TDT.

About TDTTransfusion-dependent ?-thalassemia (TDT) is a
severe genetic disease characterized by reduced or absent hemoglobin
levels that results in severe anemia and ineffective red blood cell
production. Supportive care for people with TDT consists of a lifelong
regimen of chronic blood transfusions to enable survival and suppress
symptoms of the disease, and iron chelation therapy to manage iron
overload that results from the transfusions. Despite the availability of
supportive care, many people with TDT experience serious complications
and organ damage due to underlying disease and iron overload.

Allogeneic hematopoietic stem cell transplantation (HSCT) is currently
the only available option with the potential to correct the genetic
deficiency in TDT. Complications of allogeneic HSCT include a risk of
treatment-related mortality, graft failure, graft-versus-host disease
(GvHD) and opportunistic infections, particularly in patients who
undergo non-sibling matched allogeneic HSCT.

About bluebird bio, Inc.With its lentiviral-based gene
therapies, T cell immunotherapy expertise and gene editing capabilities,
bluebird bio has built an integrated product platform with broad
potential application to severe genetic diseases and cancer. bluebird
bio’s gene therapy clinical programs include Lenti-D™ for the treatment
of cerebral adrenoleukodystrophy, and LentiGlobin™ for the treatment of
transfusion-dependent ?-thalassemia, also known as ?-thalassemia major,
and severe sickle cell disease. bluebird bio’s oncology pipeline is
built upon the company’s leadership in 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. bluebird bio’s lead oncology programs, bb2121
and bb21217, are anti-BCMA CAR T programs partnered with Celgene.
bluebird bio also has discovery research programs 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.

LentiGlobin and Lenti-D are trademarks of bluebird bio, Inc.

Forward-Looking StatementsThis release contains
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995, including statements regarding
the Company’s research, development, manufacturing and regulatory
approval plans for its LentiGlobin product candidate to treat
transfusion-dependent ß-thalassemia and severe sickle cell disease. 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, the risks that the preliminary positive efficacy and safety results
from our prior and ongoing clinical trials of LentiGlobin will not
continue or be repeated in our ongoing or planned clinical trials of
LentiGlobin, the risks that the changes we have made in the LentiGlobin
manufacturing will not result in improved patient outcomes, risks that
the current or planned clinical trials of LentiGlobin 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-K, 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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