− Data Include Updates from Phase 2/3 Starbeam (ALD-102) Study of Lenti-D for Cerebral Adrenoleukodystrophy (CALD) and Initial Data from Ongoing Observational Study (ALD-103) of Allogeneic Hematopoietic Stem Cell Transplant for CALD –
Updated data from the ongoing Phase 2/3 Starbeam study (ALD-102) assessing the efficacy and safety of Lenti-D gene therapy in boys 17 years of age and under with CALD will be presented. The company will also present initial data from its ongoing observational study (ALD-103) of allogeneic hematopoietic stem cell transplant (allo-HSCT) in boys 17 years of age and under with CALD.
“We continue to advance our Lenti-D gene therapy development program for
CALD and are pleased by the opportunity to share new data from two of
our studies at SSIEM,” said
Lenti-D hematopoietic stem cell gene therapy for cerebral adrenoleukodystrophy: safety and efficacy outcomes from an ongoing Ph 2/3 trial
Date & Time:
An observational study of patients with cerebral adrenoleukodystrophy (CALD) treated with allogeneic hematopoietic stem cell transplant
Date & Time:
bluebird bio’s clinical development program for Lenti-D gene therapy
includes the ongoing Starbeam ALD-102 study with sites in
In addition, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-304) for boys who have participated in ALD-102.
The U.S. Food and Drug Administration (
About Cerebral Adrenoleukodystrophy
Adrenoleukodystrophy (ALD) is a rare, X-linked metabolic disorder that is estimated to affect one in 21,000 male newborns worldwide. ALD is caused by mutations in the ABCD1 gene that affect the production of adrenoleukodystrophy protein (ALDP) and subsequently cause toxic accumulation of very long chain fatty acids (VLCFAs) in the adrenal cortex and white matter of the brain and spinal cord.
Approximately 35-40 percent of boys with ALD will develop cerebral ALD (CALD), the most severe form of ALD. CALD is a progressive neurogenerative disease that involves breakdown of myelin, the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control. Symptoms of CALD usually occur in early childhood and progress rapidly, if untreated, leading to severe loss of neurologic function, and eventual death, in most patients.
Currently, the only therapeutic option for patients with CALD is allogeneic hematopoietic stem cell transplant. Beneficial effects have been reported if allogeneic hematopoietic stem cell transplant is performed early in the course of CALD progression. Potential complications of allogeneic hematopoietic stem cell transplant, which can be fatal, include graft failure, graft-versus-host disease, and opportunistic infections, particularly in patients who undergo transplant with non-sibling matched donor cells.
Early diagnosis of CALD is important, as the outcome of treatment varies
with the clinical stage of the disease at the time of transplant.
Newborn screening for ALD is a critical enabler of early diagnosis and
successful treatment of ALD. In the
About bluebird bio, Inc.
With its lentiviral-based gene therapies, T cell immunotherapy expertise
and gene editing capabilities,
bluebird bio's gene therapy clinical programs include investigational treatments for cerebral adrenoleukodystrophy, transfusion-dependent β-thalassemia, also known as β-thalassemia major, and severe sickle cell disease.
bluebird bio's oncology pipeline is built upon the company's lentiviral
gene delivery and T cell engineering, with a focus on developing novel T
cell-based immunotherapies, including chimeric antigen receptor (CAR T)
and T cell receptor (TCR) therapies. The company’s lead oncology
programs are anti-BCMA CAR T programs partnered with
bluebird bio’s discovery research programs include utilizing megaTAL/homing endonuclease gene editing technologies with the potential for use across the company's pipeline.
bluebird bio has operations in
Lenti-D is a trademark of bluebird bio, Inc.
This release contains “forward-looking statements” within the meaning
of the Private Securities Litigation Reform Act of 1995, including
statements regarding the clinical, regulatory approval, and market
potential of the Company’s Lenti-D product candidate to treat cerebral
adrenoleukodystrophy. 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 efficacy and safety
data for our Lenti-D product candidate from the Starbeam Study will not
continue or persist, the risk of cessation or delay of any of the
ongoing clinical studies and/or our development of Lenti-D, the risks
regarding future potential regulatory approvals of Lenti-D, including
the risk that the Starbeam Study will be insufficient to support
regulatory submissions or marketing approval in the US and EU, 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 Form 10-Q, as well as discussions of potential risks,
uncertainties, and other important factors in our subsequent filings
Source: bluebird bio, Inc.