Additional follow-up from the completed Phase 1/2
New data from ongoing Phase 3 Northstar-2 (HGB-207) study of
LentiGlobin for TDT in patients who do not have β0/β0
genotype and Phase 3 Northstar-3 (HGB-212) of patients with β0/β0
genotype or an IVS-
New data from ongoing Phase 1/2 HGB-206 study of investigational LentiGlobin for patients with SCD
Company to hold conference call and webcast, Friday, June 14 at
bluebird bio will present data from its clinical studies of LentiGlobin™
gene therapy for TDT including updated results up to 54 months from the
long-term follow-up period of the completed Phase 1/2
New data from the company’s Phase 1/2 HGB-206 study of LentiGlobin gene therapy for SCD will include additional patients treated in the study and updated data for those previously reported.
Oral Presentations: Transfusion-Dependent β-Thalassemia
Results from the Phase 3 Northstar-3 Study Evaluating LentiGlobin
Gene Therapy in Patients with Transfusion-Dependent β-Thalassaemia and a
β0 or IVS-
Presenting Author: Andreas Kulozik, M.D., Ph.D., University Hospital Heidelberg, Heidelberg,
Date & Time:
Clinical Outcomes of LentiGlobin Gene Therapy for
Transfusion-Dependent β-Thalassaemia (TDT) Following Completion of the
Date & Time:
Safety and Efficacy of LentiGlobin Gene Therapy in Patients with
Transfusion-Dependent β-Thalassaemia and Non-β0/β0
Genotypes in the Phase 3 Northstar-2 Study (HGB-207)
Date & Time:
Oral Presentation: Sickle Cell Disease
Updated Results from the HGB-206 Study in Patients with Severe Sickle
Cell Disease Treated Under a Revised Protocol with LentiGlobin Gene
Therapy Using Plerixafor-Mobilised Haematopoietic Stem Cells (HGB-206)
Date & Time:
Abstracts outlining bluebird bio’s accepted data at EHA have been made available on the EHA conference website.
Investor Event & Webcast Information
bluebird bio will host a conference call and live webcast at
About LentiGlobin for Transfusion-Dependent β-Thalassemia
Non-serious adverse events (AEs) observed during clinical trials that were attributed to ZYNTEGLO were hot flush, dyspnoea, abdominal pain, pain in extremities and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to ZYNTEGLO.
Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan including SAEs of veno-occlusive disease.
ZYNTEGLO continues to be evaluated in the ongoing Phase 3 Northstar-2
About LentiGlobin for Sickle Cell Disease
LentiGlobin for sickle cell disease is an investigational gene therapy being studied as a potential treatment to address the underlying genetic cause of SCD. bluebird bio’s clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study.
bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for TDT and LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/medical-professionals/our-clinical-trials/.
The European Medicines Association (EMA) granted Priority Medicines (PRIME) eligibility and Orphan Medicinal Product designation to LentiGlobin for the treatment of TDT. LentiGlobin for TDT was also part of the EMA’s Adaptive Pathways pilot program, which is part of the EMA’s effort to improve timely access for patients to new medicines.
The U.S. Food and Drug Administration granted Orphan Drug status and Breakthrough Therapy designation to LentiGlobin for TDT; as well as Orphan Drug status and Regenerative Medicine Advanced Therapy designation for LentiGlobin for the treatment of SCD.
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.
ZYNTEGLO and LentiGlobin are trademarks of bluebird bio.
The full common name for ZYNTEGLO: A genetically modified autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiviral vector encoding the βA-T87Q-globin gene.
This release contains “forward-looking statements” within the meaning
of the Private Securities Litigation Reform Act of 1995. 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 risk that our MAA submitted for LentiGlobin for TDT may not be
approved by the European Commission when expected, or at all; the risk
that the efficacy and safety results from our prior and ongoing clinical
trials of LentiGlobin for TDT will not continue or be repeated in our
ongoing or planned clinical trials of LentiGlobin for TDT; the risk that
the current or planned clinical trials of LentiGlobin for TDT will be
insufficient to support regulatory submissions or marketing approval in
the US and EU; the risk that the production of HbA-T87Q may
not be sustained over extended periods of time; and the risk that we may
not secure adequate pricing or reimbursement to support continued
development or commercialization of LentiGlobin for TDT following
regulatory approval. 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 with the
Source: bluebird bio, Inc.