BLA submission based on data from Phase 1/2 and Phase 3
“With this submission, we are one step closer to bringing a potentially transformative gene therapy to people living with TDT and their families,” said
The BLA submission for beti-cel is based on data from patients treated in bluebird bio studies, including the Phase 3 HGB-207 (
About transfusion-dependent β-thalassemia (TDT)
Transfusion-dependent β-thalassemia (TDT) is a severe genetic disease caused by mutations in the β-globin gene that cause reduced or significantly reduced adult hemoglobin (Hb), resulting in chronic anemia and lifelong dependence on red blood cell (RBC) transfusions; in order to survive, people with TDT typically require blood transfusions every 3-4 weeks to maintain adequate Hb levels, a process that takes 4-7 hours. While transfusions temporarily relieve symptoms of anemia, they do not address the underlying genetic cause of TDT. Transfusions also lead to unavoidable iron overload that can cause serious complications including progressive multi-organ damage and organ failure. Iron overload resulting from TDT or ongoing RBC transfusions requires chronic treatment with chelation therapy to manage the excess iron.
About betibeglogene autotemcel (beti-cel)
betibeglogene autotemcel (beti-cel) is a one-time gene therapy custom-designed to treat the underlying cause of transfusion-dependent β-thalassemia (TDT). In order to correct the deficiency of adult hemoglobin that is the hallmark of TDT, beti-cel adds functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once a patient has the βA-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived adult hemoglobin (Hb) at levels that may eliminate or significantly reduce the need for transfusions. In beti-cel studies, transfusion independence (TI) is defined as no longer needing red blood cell (RBC) transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL. Across Phase 3 studies, 89% (32/36) of evaluable patients across ages and genotypes, including pediatric patients as young as four years of age and those with the most severe β0/β0 genotypes, achieved TI.
beti-cel is manufactured using the BB305 lentiviral vector (LVV), a third-generation, self-inactivating LVV. The BB305 LVV contains a cellular (non-viral) regulatory element, known as a promoter, that drives gene expression only in the erythroid cell lineage (RBCs and their precursors).
Adverse reactions considered related to beti-cel were uncommon and consisted primarily of infusion-related reactions (abdominal pain, hot flush, dyspnea, tachycardia and non-cardiac chest pain) and cytopenias (thrombocytopenia, leukopenia and neutropenia). Pain in extremity shortly after treatment was also documented. One of these adverse events (AE) was a serious adverse event (SAE) of thrombocytopenia considered possibly related to beti-cel.
The majority of AEs and SAEs unrelated to beti-cel were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan (including several SAEs of veno-occlusive disease).
The Phase 3
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our
bluebird bio is a human company powered by human stories. We’re putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, β-thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.
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bluebird bio Cautionary Statement Regarding Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements, including statements regarding the Company’s plans and expectations of BLA submissions. Such forward-looking statements are based on historical performance and current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect bluebird bio’s business, particularly those identified in the risk factors discussion in bluebird bio’s Annual Report on Form 10-K, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the
Source: bluebird bio, Inc.