Market Overview

bluebird bio's LentiGlobin™ Gene Therapy Granted Accelerated Assessment by European Medicines Agency for the Treatment of Transfusion-Dependent β-Thalassemia


− Company on Track to Submit Marketing Authorization Application
(MAA) in European Union in 2018 –

bio, Inc
. (NASDAQ:BLUE) today announced that its investigational
LentiGlobin™ gene therapy for the treatment of adolescent and adult
patients with transfusion-dependent β-thalassemia (TDT) and a non-β00 genotype,
was granted an accelerated assessment by the Committee for Medicinal
Products for Human Use (CHMP) of the European Medicines Agency (EMA),
for its upcoming marketing authorization application (MAA). LentiGlobin
is a potential one-time gene therapy that may address the underlying
genetic cause of TDT.

"Transfusion-dependent β-thalassemia is a severe genetic disease that
requires a lifetime of chronic blood transfusions for survival, and
while these transfusions are life-saving, they are also associated with
serious medical complications such as organ failure from iron overload,"
said David Davidson, M.D., chief medical officer, bluebird bio.
"Receiving accelerated assessment for LentiGlobin helps support our goal
of delivering the first gene therapy to patients with TDT. We look
forward to working in collaboration with the regulatory authorities on
this potentially transformative treatment option."

bluebird bio intends to file an MAA for LentiGlobin in TDT with the EMA
in 2018. Accelerated assessments can reduce the active review time of an
MAA from 210 days to 150 days once it has been validated by the EMA. An
accelerated assessment is granted to products deemed by the CHMP to be
of major interest for public health and represent therapeutic innovation.

The accelerated assessment for LentiGlobin is supported by data from
clinical studies, including the completed Phase 1/2 Northstar (HGB-204)
study, the ongoing Phase 1/2 HGB-205 study as well as available data
from the Phase 3 Northstar-2 (HGB-207) study and the long-term follow-up
study LTF-303.

The EMA previously granted Priority Medicines (PRIME) eligibility and
Orphan Medicinal Product designation to LentiGlobin for the treatment of
TDT. LentiGlobin is also part of 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 U.S. Food and Drug Administration (FDA) also granted LentiGlobin
Orphan Drug status and Breakthrough Therapy designation for the
treatment of TDT.

About Transfusion-Dependent β-Thalassemia

Transfusion-dependent β-thalassemia (TDT) is an inherited blood
disorder caused by a mutation in the β-globin gene, which causes
ineffective red blood cell production leading to severe anemia. TDT is
the most severe clinical presentation of β-thalassemia and includes
patients who receive blood transfusions as frequently as every two to
four weeks.

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.

By eliminating or reducing the need for blood transfusions, the
long-term complications associated with TDT may be reduced.

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.

Clinical Development Program for LentiGlobin

bluebird bio's clinical development program for LentiGlobin includes
ongoing studies around the world with sites in Australia, Germany,
Greece, France, Italy, Thailand, the United Kingdom and the United
States. For more information visit: or using identifier NCT01745120.

In addition, bluebird is conducting a long-term safety and efficacy
follow-up study (LTF-303) for people who have participated in bluebird
bio-sponsored clinical studies of LentiGlobin for treatment-dependent
β-thalassemia and sickle cell disease.

About bluebird bio, Inc.

With its lentiviral-based gene therapies, T cell immunotherapy expertise
and gene editing capabilities, bluebird bio 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

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.

LentiGlobin 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 Company's development and regulatory approval plans for its
LentiGlobin product candidate to treat transfusion-dependent
ß-thalassemia. 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-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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