“Treating our first subject in this study reflects the recent advances
in the field of gene therapy and is the culmination of years of
collaborative effort between the team at Dana-Farber/Boston Children’s
Cancer and Blood Disorders Center and our colleagues at
“Successfully initiating treatment in the Starbeam study represents an
important step towards improving outcomes for patients with CCALD and is
a major milestone for bluebird and its lentiviral gene therapy
About the Starbeam study (ALD-102)
The phase 2/3 study is designed to evaluate the safety and efficacy of Lenti-D drug product in the treatment of subjects with childhood cerebral adrenoleukodystrophy, or CCALD, a rare, hereditary neurological disorder affecting young boys that is often fatal. The trial study is planned to enroll up to 15 boys who will be followed for 24 months following a transplant with bluebird bio’s lentiviral transduced stem cells, Lenti-D. During this 24 month period, patients will be assessed for the onset of major functional disabilities, and other key assessments of disease progression.
For more information please contact firstname.lastname@example.org.
About Childhood Cerebral Adrenoleukodystrophy (CCALD)
Childhood Cerebral Adrenoleukodystrophy is an X-linked disease caused by the aberrant expression of the ABCD1 gene in boys leading to the inability of patients to metabolize very long chain fatty acids in cells of the brain. The disease is characterized by progressive destruction of myelin, leading to severe loss of neurological function and eventual death. The worldwide incidence of adrenoleukodystrophy (ALD) is approximately one in 20,000 newborns. CCALD accounts for about 30-40% of patients diagnosed with ALD.
About bluebird bio, Inc.
bluebird bio is a clinical-stage company committed to developing
potentially transformative gene therapies for severe genetic and orphan
diseases. bluebird bio has two clinical-stage programs in development.
The most advanced product candidate, Lenti-D, is in a recently-initiated
phase 2/3 study for the treatment of childhood cerebral
adrenoleukodystrophy (CCALD), a rare, hereditary neurological disorder
affecting young boys. The next most advanced product candidate,
LentiGlobin, is currently in a phase 1/2 study in
bluebird bio also has an early-stage chimeric antigen receptor-modified
T cell (CAR-T) program for oncology in partnership with
bluebird bio has operations in
This release contains “forward-looking statements” within the meaning
of the Private Securities Litigation Reform Act of 1995, including
statements regarding the advancement of the Company’s clinical studies,
the timing of data announcement and the potential safety and clinical
benefits of the Company’s product candidates. Any forward-looking
statements in this press release 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 risk of cessation or delay of any of the ongoing or
planned clinical studies and/or our development of our product
candidates, the risk of a delay in the enrollment of patients in the
Company’s clinical studies, the risk that the results of previously
conducted studies involving similar product candidates will not be
repeated or observed in ongoing or future studies involving current
product candidates, the risk that our collaboration with
Source: bluebird bio, Inc.
bluebird bio, Inc.
Richard E. T. Smith, Ph.D., 617-588-3321
Pure Communications, Inc.
Dan Budwick, 973-271-6085