Zynteglo (betibeglogene autotemcel) / bluebird bio 
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 1 Disease   5 Trials   5 Trials   395 News 


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  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Design of Novel Non-Viral Vectors for the Ex Vivo Gene Therapy for β-Hemoglobinopathies (Poster Board Number: Tu-206; Hall D) -  May 6, 2022 - Abstract #ASGCT2022ASGCT_1794;    
    GT for β-thalassemia provided a remarkable clinical efficacy and benefit for β-thalassemic patients with non-β0/β0 genotype, but failed to control the disease in complete absence of endogenous β-globin, due to an insufficient expression of therapeutic β-globin driven by the LentiGlobin vector...We are currently developing a second generation of β-globin expression cassettes containing different combination of small, epigenetically-identified core enhancer elements derived from the 30-kb long β-globin LCR, optimized in size and sequence to be delivered as TVs. Once identified the optimal vector for a superior production of therapeutic AS β-globin in donor-derived HSPCs, it will be further validated in vitro in patient-derived HSCs and in vivo in humanized immunodeficient mice in an efficacy/safety preclinical study.
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Trial completion, Gene therapy:  HGB-207: A Study Evaluating the Efficacy and Safety of the LentiGlobin (clinicaltrials.gov) -  Apr 27, 2022   
    P3,  N=23, Completed, 
    Once identified the optimal vector for a superior production of therapeutic AS β-globin in donor-derived HSPCs, it will be further validated in vitro in patient-derived HSCs and in vivo in humanized immunodeficient mice in an efficacy/safety preclinical study. Active, not recruiting --> Completed
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    LENTIGLOBIN FOR SCD GENE THERAPY: TREATMENT PROCESS EVOLUTION LEADS TO IMPROVED OUTCOMES () -  Apr 20, 2022 - Abstract #ASPHO2022ASPHO_256;    
    P1/2
    CD34+ cells (collected by bone marrow harvesting [Group A] and plerixafor mobilization/apheresis [Group C]) were transduced with BB305 lentiviral vector (LVV)... Alterations to the protocol and manufacturing process in HGB-206 resulted in improved cell dose, transduction efficiency, HbAT87Q expression, and clinical outcomes in Group C versus Group A.bluebird bio sponsored this study.
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    BETIBEGLOGENE AUTOTEMCEL IN PEDIATRIC PTS WITH TRANSFUSION-DEPENDENT β-THALASSEMIA IN PHASE 3 TRIALS (live stream) -  Apr 20, 2022 - Abstract #ASPHO2022ASPHO_88;    
    P3
    The safety profile of the treatment regimen was reflective of busulfan myeloablation. beti-cel is a potentially curative gene therapy for pediatric patients with TDT through the ability to achieve TI with near-normal to normal Hb levels.Sponsored by bluebird bio.
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Clinical, Clinical Trial,Phase II, Journal:  Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease. (Pubmed Central) -  Mar 3, 2022   
    P1/2
    One-time treatment with LentiGlobin resulted in sustained production of HbA in most red cells, leading to reduced hemolysis and complete resolution of severe vaso-occlusive events. (Funded by Bluebird Bio; HGB-206 ClinicalTrials.gov number, NCT02140554.).
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Clinical Trial,Phase I, Clinical Trial,Phase II, Journal:  Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial. (Pubmed Central) -  Feb 23, 2022   
    P=N/A, P1/
    Trial HGB-205 ( NCT02151526 ) aimed at evaluating gene therapy by autologous CD34 cells transduced ex vivo with lentiviral vector BB305 that encodes the anti-sickling β-globin expressed in the erythroid lineage...Four patients with TDT and three patients with SCD, ages 13-21 years, were treated after busulfan myeloablation 4.6-7.9 years ago, with a median follow-up of 4.5 years...Clinical remission and remediation of biological hallmarks of the disease have been sustained in two of the three patients with SCD, and frequency of transfusions was reduced in the third. The patients with TDT are all transfusion free with improvement of dyserythropoiesis and iron overload.
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Trial completion date, Trial primary completion date, Gene therapy:  Northstar-3: A Study Evaluating the Efficacy and Safety of the LentiGlobin (clinicaltrials.gov) -  Feb 21, 2022   
    P3,  N=18, Active, not recruiting, 
    The patients with TDT are all transfusion free with improvement of dyserythropoiesis and iron overload. Trial completion date: Jun 2022 --> Nov 2022 | Trial primary completion date: Jun 2022 --> Nov 2022
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Journal:  Betibeglogene Autotemcel Gene Therapy for Non-β/β Genotype β-Thalassemia. (Pubmed Central) -  Feb 17, 2022   
    P3
    Treatment with beti-cel resulted in a sustained HbA level and a total hemoglobin level that was high enough to enable transfusion independence in most patients with a non-β/β genotype, including those younger than 12 years of age. (Funded by Bluebird Bio; HGB-207 ClinicalTrials.gov number, NCT02906202.).
  • ||||||||||  sirolimus / Generic mfg.
    Journal:  β-Thalassemia: evolving treatment options beyond transfusion and iron chelation. (Pubmed Central) -  Feb 16, 2022   
    In addition, 2 gene editing techniques (CRISPR-Cas9 and zinc-finger nucleases) are under investigation as a means to silence BCL11A to induce HbF with agents designated CTX001 and ST-400, respectively. Results from the many clinical trials for these agents will yield results in the next few years, which may end the era of relying on transfusion alone as the mainstay of thalassemia therapy.
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Clinical, Journal:  Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease. (Pubmed Central) -  Jan 27, 2022   
    Gene therapy with LentiGlobin for sickle cell disease (bb1111, lovotibeglogene autotemcel) consists of autologous transplantation of a patient's hematopoietic stem cells transduced with the BB305 lentiviral vector that encodes the β-globin gene...Several somatic mutations predisposing to acute myeloid leukemia were present after diagnosis, which suggests that patients with sickle cell disease are at increased risk for hematologic malignant conditions after transplantation, most likely because of a combination of risks associated with underlying sickle cell disease, transplantation procedure, and inadequate disease control after treatment. (Funded by Bluebird Bio.).
  • ||||||||||  Lyfgenia (lovotibeglogene autotemcel) / bluebird bio
    Trial completion date, Trial primary completion date, Gene therapy, Viral vector:  HGB-210: A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease (clinicaltrials.gov) -  Dec 29, 2021   
    P3,  N=35, Recruiting, 
    Mean values for pts overall indicate meaningful change for all domains and Pain Intensity NRS, with the exception of Anxiety. Trial completion date: Jan 2025 --> Sep 2025 | Trial primary completion date: Jul 2024 --> Feb 2025