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Bluebird Bio

Known as: Blue Bird Biotec 
A clinical-stage biotechnology company using gene therapy, cancer immunotherapy, and gene editing to treat and cure genetic diseases and cancer.
National Institutes of Health

Papers overview

Semantic Scholar uses AI to extract papers important to this topic.
2018
2018
Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients
Autologous gene therapy (GT) for beta-hemoglobinopathies has demonstrated encouraging early safety and efficacy using… 
2018
2018
Outcomes for Initial Patient Cohorts with up to 33 Months of Follow-up in the Hgb-206 Phase 1 Trial
Background Sickle cell disease (SCD) is a progressively debilitating genetic disease causing significant morbidity and early… 
2018
2018
Updated Phase 1 Results of Zuma-3: Kte-C19, an Anti-CD19 Chimeric Antigen Receptor T Cell Therapy, in Adult Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia
Background: Although approximately half of adult patients with acute lymphoblastic leukemia (ALL) achieve long-term survival… 
2018
2018
Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study
Background Advances in red blood cell (RBC) transfusion and chelation have improved the prognosis of patients with transfusion… 
2017
2017
Results from the Hgb-207 (Northstar-2) Trial: A Phase 3 Study to Evaluate Safety and Efficacy of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in Patients with Non-β0/β0…
Background Gene therapy with LentiGlobin Drug Product (DP), which contains autologous CD34+ hematopoietic stem cells transduced… 
2017
2017
Longer Term Follow-up on the First Patients with Severe Hemoglobinopathies Treated with Lentiglobin Gene Therapy
Background: LentiGlobin Drug Product (DP) contains autologous CD34+ cells transduced with the betibeglogene darolentivec (BB305… 
2017
2017
Results of a Phase 3 Study of Elderly Patients with Newly Diagnosed AML Treated with Sapacitabine and Decitabine Administered in Alternating Cycles
Background: Sapacitabine is a novel oral nucleoside analogue with a unique ability to induce single-strand DNA breaks after… 
2017
2017
ROR1-Directed Chimeric Antigen Receptor T Cell Recognition of Self-Antigen Is Associated with Acute Toxicity, T Cell Dysfunction, and Poor Tumor Control.
Chimeric antigen receptor T cells (CAR T) have demonstrated robust clinical efficacy in B cell and plasma cell malignancies. In… 
2017
2017
Risk Factors and Outcomes According to Age at Transplant with an HLA Identical Sibling for Sickle Cell Disease
Background: Hematopoietic stem cell transplant (HSCT) from an HLA identical (HLAid) sibling donor is a well-established curative… 
2017
2017
Neurometabolic disease: Gene therapy success for cerebral adrenoleukodystrophy
(ALD) could be treated safely and effectively using genetically modified versions of a patient’s own haematopoietic stem cells… 
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