bluebird bio, Inc. (Nasdaq: BLUE) and Forty Seven, Inc.
(Nasdaq:FTSV) announced today that they have entered into a
research collaboration to pursue clinical proof-of-concept for
Forty Seven’s novel antibody-based conditioning regimen, FSI-174
(anti-cKIT antibody) plus magrolimab (anti-CD47 antibody), with
bluebird’s ex vivo lentiviral vector hematopoietic stem cell (LVV
HSC) gene therapy platform. This collaboration will focus on a
conditioning approach aimed to deliver reduced toxicity and will
initially target diseases that have the potential to be corrected
with transplantation of autologous gene-modified blood-forming stem
cells. If successful, the new conditioning regimen could allow for
more patients to undergo gene therapy.
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Autologous hematopoietic stem cell transplantation (HSCT) and
most ex vivo LVV HSC gene therapies require that a patient’s own
stem cells first be depleted from the bone marrow to facilitate the
engraftment of the new (or gene-modified) HSCs through a process
called conditioning. Conditioning is performed using chemotherapy
or radiation, which can place patients at risk for infection and
require hospitalization until bone marrow cells have recovered. In
addition, conventional conditioning can place patients at risk for
secondary malignancy and infertility. As a result, the overall
toxicity profile of current conditioning regimens limits the types
of patients who are eligible for gene therapy. It is hoped that
novel antibody based conditioning regimens could avoid these
toxicities.
“We are excited about this collaboration, combining our
industry-leading LVV HSC gene therapy platform with Forty Seven’s
novel antibody-based conditioning regimen,” said Philip Gregory,
chief scientific officer, bluebird bio. “We believe that, if
successful, this novel conditioning modality could not only
increase the number of patients and physicians who may consider
gene therapy but also improve the overall risk benefit profile for
stem cell-based gene therapy, as well as potentially reduce time
and costs associated with hospital visits.”
“Forty Seven is advancing the pioneering work on CD47 and cKIT
from our scientific founder, Irv Weissman’s lab. We have shown that
antibody blockade of CD47 can synergize with other antibodies
targeting cancer to promote tumor engulfment. Based on this
experience, coupled with the results of preclinical studies, we are
eager to explore this dual-antibody approach for the potential
treatment of non-malignant diseases,” says Jens Peter Volkmer,
M.D., Founder and Vice President of Research and Development at
Forty Seven.
Forty Seven’s President and Chief Executive Officer, Mark
McCamish, M.D., Ph.D., commented, “bluebird is a leading gene
therapy company and we are excited to collaborate with them. Stem
cell transplantation is potentially curative for a variety of blood
diseases, including genetic blood disorders like sickle cell
disease and beta-thalassemia. If successful, we believe our chemo-
and radiation-free, all-antibody approach could expand
transplantation beyond genetic blood disorders to a range of
indications for which current transplantation approaches are
suboptimal. In 2020, we plan to evaluate FSI-174 in healthy
volunteers, before initiating a combination study of Forty Seven’s
novel all-antibody conditioning regimen and bluebird’s gene therapy
product.”
Under the terms of the agreement, bluebird bio will provide its
ex vivo LVV HSC gene therapy platform and Forty Seven will
contribute its innovative antibody-based conditioning regimen for
the collaboration.
About FSI-174 and Magrolimab FSI-174 is a humanized
monoclonal antibody targeting cKIT, which is a receptor that is
highly expressed on hematopoietic stem cells. Magrolimab is a
humanized monoclonal antibody targeting CD47, which is a “don’t eat
me” signal to macrophages and is expressed on all cells. Magrolimab
is currently being investigated in Phase 2 clinical trials to treat
cancer and has established clinical efficacy in four indications,
including myelodysplastic syndrome, acute myeloid leukemia, diffuse
large B cell lymphoma and follicular lymphoma, with a favorable
safety profile in over 350 patients treated, including some
patients treated continuously for over two years. When combined,
FSI-174 sends a positive signal to macrophages to target blood
forming stem cells for removal and magrolimab disengages inhibitory
signals that block phagocytosis. Combination of these antibodies
has shown efficient removal of blood forming stem cells, allowing
for transplantation in pre-clinical models.
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 Forty Seven Inc. Forty Seven, Inc. is a
clinical-stage immuno-oncology company that is developing therapies
targeting cancer immune evasion pathways based on technology
licensed from Stanford University. Forty Seven’s lead program,
magrolimab, is a monoclonal antibody against the CD47 receptor, a
“don’t eat me” signal that cancer cells commandeer to avoid being
ingested by macrophages. This antibody is currently being evaluated
in multiple clinical studies in patients with myelodysplastic
syndrome, acute myeloid leukemia, non-Hodgkin’s lymphoma, ovarian
cancer and colorectal carcinoma.
For more information, please visit www.fortyseveninc.com
or contact info@fortyseveninc.com.
Follow Forty Seven on social media: @FortySevenInc,
LinkedIn
Forward-Looking Statements This release contains
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995. Words such as "may,"
"will," “potentially,” and similar expressions (as well as other
words or expressions referencing future events, conditions, or
circumstances) are intended to identify forward-looking statements.
These statements include those related to the research and
development plans for bluebird bio’s and Forty Seven’s respective
platforms and product candidates, the timing and success of Forty
Seven’s collaboration with bluebird bio, Forty Seven’s plans to
pursue clinical proof-of-concept for FSI-174 plus magrolimab with
the LVV HSC gene therapy platform, the focus on diseases that have
the potential to be corrected with transplantation of autologous
gene-modified blood-forming stem cells, the tolerability and
efficacy of FSI-174 and magrolimab, Forty Seven’s plans to continue
development of FSI-174 plus magrolimab, as well as related timing
for clinical trials of the same.
Any forward-looking statements are based on the companies’
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
exploratory antibody-based conditioning platform will not be
successful or will not be safe or effective in clinical trials, the
risks that the collaboration between bluebird bio and Forty Seven
will not continue or be successful, and the risk that the parties
will not be successful in advancing the collaboration in
development, the risk that potential product candidates that
bluebird bio and Forty Seven develop may not progress through
clinical development or receive required regulatory approvals
within expected timelines or at all, the risk that clinical trials
may not confirm any safety, potency or other product
characteristics described or assumed in this press release and the
risk that such product candidates may not be beneficial to patients
or successfully commercialized. For a discussion of other risks and
uncertainties, and other important factors, any of which could
cause the companies’ actual results to differ from those contained
in the forward-looking statements, see the section entitled “Risk
Factors” in each company’s most recent Form 10-K as well as
discussions of potential risks, uncertainties and other important
factors in subsequent filings with the Securities and Exchange
Commission at www.sec.gov. All information contained in this press
release are not guarantees of future performance and speak only as
of the date hereof, and each of bluebird bio and Forty Seven
disclaims any obligation to update this information to reflect
future events or circumstances unless required by law.
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version on businesswire.com: https://www.businesswire.com/news/home/20191112005324/en/
bluebird bio Investors: Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com or Media: Jennifer Snyder,
617-448-0281 jsnyder@bluebirdbio.com Forty Seven Investors:
Hannah Deresiewicz, (212) 362-1200
hannah.deresiewicz@sternir.com or Media: Sarah Plumridge,
(312) 506-5218 fortyseven@hdmz.com
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