– Patient treated with LentiGlobinTM drug product demonstrates high levels (~50% of total hemoglobin) of anti-sickling hemoglobin (HbAT87Q) 15 months after treatment –
“We have managed this patient at Necker for more than 10 years, and
standard treatments were not able to control his SCD symptoms. He had to
receive blood transfusions every month to prevent severe pain crises,”
said Professor
“Since our initial publication of this therapeutic approach in mouse
models in 2001, we are delighted to obtain such a clear
proof-of-principle of its efficacy in a patient,” said
“We are pleased to see this case study published in NEJM and
shared with the broader research community. The successful outcome in
Patient 1204 demonstrates the promise of treatment with LentiGlobin gene
therapy in patients with severe SCD and serves as a guide for our
efforts to optimize outcomes in future patients,” said
Clinical and Biological Outcomes for the First Patient with Sickle
Cell Disease Treated with Gene Therapy
Patient 1204, a male
patient with βS/βS genotype, was enrolled in
Neutrophil and platelet engraftment were achieved on Day +38 and Day +91
post-transplantation, respectively. HbAT87Q levels increased
steadily and
Adverse events (AEs) were consistent with busulfan conditioning, and no AEs related to LentiGlobin drug product have been observed to date.
Over the 15 months since transplantation, no SCD-related clinical events or hospitalizations have occurred, contrasting favorably with the period before the patient began regular transfusions. All medications have been discontinued, including pain medication. The patient has resumed regular school attendance and reports full participation in normal physical activities.
About SCD
Sickle cell disease (SCD) is an inherited disease
caused by a mutation in the β-globin gene that results in sickle-shaped
red blood cells. The disease is characterized by anemia, vaso-occlusive
crisis, infections, stroke, overall poor quality of life and, sometimes,
early death.
Where adequate medical care is available, common treatments for patients with SCD largely revolve around management and prevention of acute sickling episodes. Chronic management may include hydroxyurea and, in certain cases, chronic transfusions. Given the limitations of these treatments, there is no effective long-term treatment. The only advanced therapy for SCD is allogeneic hematopoietic stem cell transplantation (HSCT). Complications of allogeneic HSCT include a significant risk of treatment-related mortality, graft failure, graft-versus-host disease, 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 its Lenti-D™ product
candidate, currently in a Phase 2/3 study, called the Starbeam Study,
for the treatment of cerebral adrenoleukodystrophy, and its
LentiGlobin™ BB305 product candidate, currently in four clinical studies
for the treatment of transfusion-dependent β-thalassemia 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) and T cell receptor (TCR) therapies.
bluebird bio’s lead oncology program, bb2121, is an anti-BCMA CAR T
program partnered with Celgene. bb2121 is currently being studied in a
Phase 1 trial for the treatment of relapsed/refractory multiple myeloma.
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,
About AP-HP
AP-HP -
About the Imagine Institute
As the leading European
center for research, care and teaching in genetic diseases, the
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 research and development plans for its LentiGlobin product
candidate to treat severe sickle cell disease, including statements
whether the manufacturing process changes for LentiGlobin will improve
outcomes of patients with severe sickle cell disease and whether the
planned changes to the HGB-206 clinical trial protocol will improve
outcomes in patients with 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, 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, planned or expanded clinical trials of
LentiGlobin, the risks that the changes we have made in the LentiGlobin
manufacturing process or the HGB-206 clinical trial protocol 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, the risk
of a delay in the enrollment of patients in our clinical studies, 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 quarterly report on Form 10-K, as well as discussions of
potential risks, uncertainties, and other important factors in our
subsequent filings with the
View source version on businesswire.com: http://www.businesswire.com/news/home/20170301006561/en/
Source: bluebird bio, Inc.
Investors:
bluebird bio, Inc.
Manisha Pai, 617-245-2107
mpai@bluebirdbio.com
or
Media:
bluebird
bio, Inc.
Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com
or
Pure
Communications, Inc.
Dan Budwick, 973-271-6085
or
AP-HP:
Anne-Cécile
Bard and Marine Leroy, +33 (0)1 40 27 37 22
service.presse@aphp.fr
or
Imagine
Institute, Paris, France:
Béatrice Parrinello-Froment, +33
(0)6 63 72 16 06
beatriceparrinello@bpfconseil.com
or
Pauline
Rodrigue-Moriconi, +33 (0)6 77 23 71 19
pauline.rodrigue@institutimagine.org