bluebird bio Receives US and European Orphan Drug Designation …

CAMBRIDGE, Mass., Jun 19, 2012 (BUSINESS WIRE) –
bluebird bio, a leader in the development of innovative gene therapies
for severe genetic disorders, announced today that both the U.S. Food
and Drug Administration (FDA) and the European Medicines Agency (EMA)
have granted an orphan drug designation to its investigational gene
therapy product for the treatment of adrenoleukodystrophy (ALD). The
product consists of the patient’s own CD34+ hematopoietic stem cells
transduced with bluebird bio’s lentiviral vector, Lenti-D, encoding the
human ABCD1 cDNA. Based on promising early clinical proof of concept
results, bluebird bio plans to initiate a Phase 2/3 clinical study in
childhood cerebral ALD in both the United States and Europe in 2013.

“Receiving orphan drug designation is a positive step forward in our
efforts to bring hope to ALD patients and their families,” said David
Davidson, M.D., chief medical officer of bluebird bio. “We believe our
lentiviral technology has the potential to be a one-time transformative
therapy for patients suffering from rare genetic disorders like ALD for
whom there are limited treatment options. bluebird is committed to
advancing the clinical and commercial development of our gene therapy
platform because of the dramatic benefit it may have on the lives of
patients.”

Orphan drug designation, which is intended to facilitate drug
development for rare diseases, provides substantial benefits to the
sponsor, including the potential for funding for certain clinical
studies, study-design assistance, and several years of market
exclusivity for the product upon regulatory approval.

About ALD

Adrenoleukodystrophy (ALD) is a rare X-linked, inherited neurological
disorder that, in its most severe form, causes damage to the myelin
sheath (an insulating layer of membranes that surrounds nerve cells in
the brain) and progressive dysfunction of the adrenal glands. Also known
as Lorenzo’s Oil disease, ALD is estimated to affect one in every 21,000
boys worldwide. In the childhood cerebral form (CCALD), symptoms usually
occur between the ages of 4 and 10. Boys afflicted with this form of ALD
develop normally until the onset of symptoms. The symptoms of this
disorder often progress rapidly and, in a matter of years, can lead to a
vegetative state and, ultimately, death. Current treatment options are
limited to allogeneic stem cell transplantation when there is an
appropriate donor. Allogeneic transplants carry a significant risk of
serious morbidity and death.

About bluebird bio’s CCALD Product Development

bluebird bio’s CCALD product program has the potential to halt the
progression of CCALD by providing a functional ABCD1 gene to the
patient’s own stem cells. These stem cells proliferate, and some of the
progeny cells travel to the brain where they become microglial cells
incorporating the corrective gene. Data from the first clinical study
treating X-linked CCALD patients with the company’s lentiviral gene
therapy product demonstrated continued stable expression of the
transgene and the corresponding ABCD-1 protein for over four years in
two CCALD patients, resulting in prolonged disease stabilization.
bluebird bio plans to initiate a Phase 2/3 clinical study in CCALD in
both the United States and Europe in 2013.

About bluebird bio

bluebird bio is developing innovative gene therapies for severe genetic
disorders. At the heart of bluebird bio’s product creation efforts is
its broadly applicable gene therapy platform for the development of
novel treatments for diseases with few or no clinical options. The
company’s novel approach uses stem cells harvested from the patient’s
own bone marrow into which a healthy version of the disease causing gene
is inserted. bluebird bio’s approach represents a true paradigm shift in
the treatment of severe genetic diseases by eliminating the potential
complications associated with donor cell transplantation and presenting
a one-time potentially transformative therapy using a patient’s own stem
cells. bluebird bio has two later stage clinical products in development
for childhood cerebral adrenoleukodystrophy (CCALD) and
beta-thalassemia/sickle cell anemia. Led by a world-class team, bluebird
bio is privately held and backed by top-tier life sciences investors,
including Third Rock Ventures, TVM Capital, ARCH Venture Partners,
Forbion Capital Partners, Easton Capital and Genzyme Ventures. Its
operations are located in Cambridge, Mass. and Paris, France. For more
information, please visit
www.bluebirdbio.com .

SOURCE: bluebird bio

        
        Pure Communications, Inc. 
        Dan Budwick, 973-271-6085
        

Copyright Business Wire 2012