bluebird bio, Inc. (Nasdaq: BLUE) and Novo Nordisk A/S (NVO)
today announced that they have entered into a research
collaboration to jointly develop next-generation in vivo genome
editing treatments for genetic diseases, including haemophilia.
During the three-year research collaboration, bluebird and Novo
Nordisk will focus on identifying a development gene therapy
candidate with the ambition of offering people with haemophilia A a
lifetime free of factor replacement therapy.
The research collaboration will utilise bluebird bio’s
proprietary mRNA-based megaTAL™ technology that has the potential
to provide a highly specific and efficient way to silence, edit or
insert genetic components. Aligned with Novo Nordisk’s haemophilia
portfolio, the research collaboration will initially focus on
correcting FVIII-clotting factor deficiency, with the potential to
explore additional therapeutic targets.
“We are pleased to announce our collaboration with bluebird
whose demonstrated capabilities in gene therapy will enable the
next-generation of innovative products to make a significant impact
on patients’ lives,” said Marcus Schindler, senior vice president
for Global Drug Discovery at Novo Nordisk. “This important research
collaboration aimed at addressing genetic diseases at the DNA level
reflects Novo Nordisk’s enduring commitment and dedication to
inventing disease-modifying medicines that can truly change the
lives of people living with haemophilia and other genetic
diseases.”
“bluebird has made tremendous progress on enabling an in vivo
gene editing platform based on our megaTAL technology, including
important advances in high-quality mRNA production and
purification,” said Philip Gregory, D. Phil., chief scientific
officer, bluebird bio. “We believe this technology has the
potential to create a highly differentiated approach to the
treatment of many severe genetic diseases. Moreover, we are
thrilled to be able to combine this new platform technology with
Novo Nordisk’s deep expertise in haemophilia research and
therapeutics. We believe this collaboration will move us toward our
shared goal of recoding the treatment paradigm and substantially
reduce the burden of disease for patients with factor VIII
deficiency.”
About megaTALs MegaTALs are a single-chain fusion enzyme
that combines the natural DNA cleaving processes of Homing
Endonucleases (HEs) with the DNA binding region of transcription
activator-like (TAL) effectors. TALs are easily engineered proteins
that recognize specific DNA sequences. This protein fusion
architecture allows the generation of extremely active and highly
specific and compact nucleases that are compatible with all current
viral and non-viral cell delivery methods.
About haemophilia The incidence of haemophilia A is
approximately one in 5,000 and of haemophilia B it is approximately
one in 25,000 male births respectively1. Over 400,000 males have
haemophilia A or B and the disease is severely underdiagnosed in
developing countries2. An estimated 304,0003 people live with
haemophilia A, which is a result of decreased or defective
production of the blood clotting factor VIII. People with
haemophilia B, of which there are some 136,0004 have deficiencies
in producing clotting factor IX. For people with haemophilia,
bleeds often occur in the joints, particularly knees and ankles.
Bleeds can also occur in the muscles, soft tissues,
gastrointestinal tract or even the brain. Trauma, major surgery,
tooth extractions or other minor surgical interventions require
medical supervision to manage the associated bleeding. Without
treatment, bleeds are painful and can cause lasting damages and
lead to impaired mobility.
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
bluebirdbio.com.
Follow bluebird bio on social media: @bluebirdbio,
LinkedIn, Instagram and YouTube.
bluebird bio is a trademark of bluebird bio, Inc.
About Novo Nordisk A/S Novo Nordisk is a global
healthcare company with more than 95 years of innovation and
leadership in diabetes care. This heritage has given us experience
and capabilities that also enable us to help people defeat obesity,
haemophilia, growth disorders and other serious chronic diseases.
Headquartered in Denmark, Novo Nordisk employs approximately 41,600
people in 80 countries and markets its products in more than 170
countries. Novo Nordisk's B shares are listed on Nasdaq Copenhagen
(Novo-B). Its ADRs are listed on the New York Stock Exchange
(NVO).
For more information, visit novonordisk.com, Facebook, Twitter,
LinkedIn, YouTube.
bluebird bio 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 research and development plans
for bluebird bio’s gene therapy products and megaTAL platform. 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 bluebird bio’s megaTAL
platform will not be successful or will not be safe or effective in
clinical trials, the risks that our collaboration with Novo Nordisk
A/S will not continue or be successful, and the risk that we will
not be successful in identifying a gene therapy candidate for
development. 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.
References
- https://www.hemophilia.org
-
https://unctad.org/en/PublicationChapters/tdstat42_FS11_en.pdf
- Stonebraker JS; Study of variations in the reported haemophilia
A prevalence around the world. Haemophilia 2010; 16(1):20-32.
- Stonebraker JS, A study of variations in the reported
haemophilia B prevalence around the world. Haemophilia 2012;
18(3):e91-e94
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version on businesswire.com: https://www.businesswire.com/news/home/20191009005185/en/
For bluebird bio Investors: Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com or Media: Jennifer Snyder,
617-448-0281 jsnyder@bluebirdbio.com For Novo Nordisk A/S
Investors: Peter Hugreffe Ankersen, +45 3075 9085
phak@novonordisk.com Valdemar Borum Svarrer, +45 3079 0301
jvls@novonordisk.com Ann Søndermølle Rendbæk, +45 3075 2253
arnd@novonordisk.com Kristoffer Due Berg (US), +1 609 235
2989 krdb@novonordisk.com or Media: Martin Havtorn Petersen,
+45 3075 5246 mhpz@novonordisk.com Ken Inchausti (US), +1
609 240 9429 kiau@novonordisk.com
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