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
    Journal, Real-world evidence, Gene therapy, Real-world:  Gene Therapy in Transfusion-Dependent Non-?0/?0 Genotype ?-Thalassemia: First Real-World Experience of Beti-cel. (Pubmed Central) -  Dec 26, 2024   
    Beti-cel is a potentially curative therapy for patients with TDT across genotypes and ages through achievement of durable TI and a favorable safety profile up to 10 y. These data will inform real-world treatment decisions. We report, to our knowledge, the first real-world application of betibeglogene autotemcel (beti-cel; Zynteglo) during its period of active license in Europe from January 2020 to March 2022 for patients aged ?12 years without a ?0/?0 genotype and without a HLA-matched sibling donor, before beti-cel marketing authorization was withdrawn by its holder because of nonsafety reasons...Eight patients received beti-cel after busulfan myeloablative conditioning, all achieving transfusion independence within 8 to 59
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Post-Approval, Real-World Experience with Betibeglogene Autotemcel for Transfusion-Dependent Beta Thalassemia (HCC Exhibit Hall 3; In-Person) -  Dec 19, 2024 - Abstract #TCTASTCTCIBMTR2025TCT_ASTCT_CIBMTR_1069;    
    Patients experienced prolonged platelet engraftment time and high platelet transfusion requirements, which were associated with severe bleeding in patients with VOD or HLA Class I alloimmunization. Further studies will seek to mitigate bleeding complications by reducing VOD rates and to explore the impact of HLA Class I alloimmunization in patients receiving beti-cel for TDT.
  • ||||||||||  Finding a Way for Patients to Access Gene Therapies () -  Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_2243;    
    Canada and UK mainly use financial-based schemes built on simple price discounts. Australia and the US aim at implementing schemes whereby treatment cost payers incur corresponds to expected health outcome for a particular patient.
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Review, Journal, Gene therapy:  Gene Therapy: A Revolutionary Step in Treating Thalassemia. (Pubmed Central) -  Oct 25, 2024   
    FDA-approved ZYNTEGLO is a potentially one-time curative treatment for ?-Thalassemia. Although cutting-edge, its use is limited because of the high cost-a price of USD 2.8 million per patient.
  • ||||||||||  Casgevy (exagamglogene autotemcel) / Vertex, CRISPR Therap, Molecular Templates
    Journal:  In brief: Casgevy for beta thalassemia. (Pubmed Central) -  May 2, 2024   
    No abstract available No abstract available
  • ||||||||||  Advances in Science and Challenges at HTA: The Case of CRISPR Technologies () -  Mar 8, 2024 - Abstract #ISPOR2024ISPOR_1800;    
    Exa-cel and subsequent CRISPR-based technologies are expected to face similar challenges to other cell and gene therapies, with the risk of off-target gene editing likely to be scrutinized for many years to come. HTA agencies may be more willing to accept uncertainty around clinical benefit for products demonstrating cost savings for healthcare systems.
  • ||||||||||  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) -  Feb 15, 2024   
    P3,  N=35, Recruiting, 
    Further exploration of the implications of such agreements is warranted, especially for interventions with greater uncertainty and/or safety concerns. Trial completion date: Apr 2027 --> Nov 2027 | Trial primary completion date: Oct 2026 --> May 2027
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Journal, Gene therapy, Viral vector:  Reducing the transcriptional read-through rate of a lentiviral vector for ?-thalassemia gene therapy. (Pubmed Central) -  Jan 30, 2024   
    The results of the present study show that inserting C-U+ or WPRE before the polyA sequence of the BB305 would reduce the EATRT rate at no cost of its expressing efficacy and viral preparation titers. Thus, we present an alternative improvement for a safer lentiviral vector for ?-thalassemia clinical trials.
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Journal:  Gene addition for beta thalassemia. (Pubmed Central) -  Dec 17, 2023   
    Several clinical trials have investigated the use of lentiviral vectors containing a functional beta globin gene, including Lentiglobin BB305, GLOBE, and TNS9.3.55. The efficacy and safety data from these ongoing trials are discussed in this review.
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Improvement in Iron Burden in Patients with Transfusion-Dependent  (SDCC - Halls G-H) -  Nov 3, 2023 - Abstract #ASH2023ASH_3408;    
    P=N/A, P1/
    In this analysis with up to 9 years of follow up, patients treated with beti-cel who achieved TI also demonstrated sustained improvements in iron burden, and the majority of patients were able to stop chelation. Collectively, these results demonstrate the long-term durability and stability of response after beti-cel gene therapy in patients with TDT.
  • ||||||||||  Review, Journal, Gene therapy:  Clinical applications of gene therapy for rare diseases: A review. (Pubmed Central) -  Jul 17, 2023   
    An initial comparison of the effectiveness of AAV and oligonucleotide therapies in SMA is possible with Zolgensma, an AAV serotype 9 vector, and Spinraza. Through these examples of marketed gene therapies and gene cell therapies, we will discuss the expanding applications of such novel technologies to previously intractable rare diseases.
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Enrollment closed, Trial completion date, Trial primary completion date, Gene therapy, Viral vector:  LTF-303: Long-term Follow-up of Subjects With Transfusion-Dependent ?-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy (clinicaltrials.gov) -  May 6, 2023   
    P=N/A,  N=66, Active, not recruiting, 
    Through these examples of marketed gene therapies and gene cell therapies, we will discuss the expanding applications of such novel technologies to previously intractable rare diseases. Enrolling by invitation --> Active, not recruiting | Trial completion date: Mar 2031 --> Nov 2035 | Trial primary completion date: Mar 2031 --> Nov 2035
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Can PK/PD Modeling and Simulation Bring a Gene Therapy (for ?-thalassemia) to Patients-in-Need Faster? (Board No. 1593) -  Apr 21, 2023 - Abstract #ASGCT2023ASGCT_2029;    
    Enrolling by invitation --> Active, not recruiting | Trial completion date: Mar 2031 --> Nov 2035 | Trial primary completion date: Mar 2031 --> Nov 2035 Recently the FDA approved an autologous transplant/ex-vivo gene therapy beti-cel (betibeglogene autotemcel; commercial brand name: Zynteglo
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Improving Lentiviral Gene Therapy by Inhibiting the Innate Immune Response (Board No. 732) -  Apr 6, 2023 - Abstract #ASGCT2023ASGCT_559;    
    P1
    With the advent of CAR-T therapy and other in vivo gene therapies like Zynteglo, lentiviral gene delivery has become a successful way to address severe chronic disorders...Analysis of the mRNA-seq data and extensive literature review yielded an ordered list of AVGs that are the most potent against lentiviral transgenes, and the top 4 were selected from this list for Cas9 KO and inhibition with siRNAs and/or small molecule inhibitors (SMIs), to determine if inhibiting these targets can improve transduction efficiency or transgene expression. (Table 1)
  • ||||||||||  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) -  Mar 24, 2023   
    P3,  N=35, Recruiting, 
    Active, not recruiting --> Completed Trial completion date: Sep 2025 --> Apr 2027 | Trial primary completion date: Feb 2025 --> Oct 2026
  • ||||||||||  Zynteglo (betibeglogene autotemcel) / bluebird bio
    Journal:  lovo-cel gene therapy for Sickle Cell Disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study. (Pubmed Central) -  Sep 29, 2022   
    P1/2
    Lovo-cel (bb1111; LentiGlobin for sickle cell disease [SCD]) gene therapy (GT) comprises autologous transplantation of hematopoietic stem and progenitor cells transduced with the BB305 lentiviral vector encoding a modified β-globin gene (β ) to produce anti-sickling hemoglobin (HbA )...The safety of the lovo-cel for SCD treatment regimen largely reflected the known side effects of HSPC collection, busulfan conditioning regimen, and underlying SCD; acute myeloid leukemia was observed in 2 patients in Group A and deemed unlikely related to insertional oncogenesis...Changes made during development of the lovo-cel treatment process were associated with improved outcomes and provide lessons for future SCD GT studies. (ClinicalTrials.gov: NCT02140554; Trial: HGB-206.