LentiGlobin BB305 Drug Product

GENETIC5 trials

Conditions

Beta-ThalassemiaBeta-Thalassemia MajorBeta-thalassaemiaSickle Cell Diseaseβ-thalassemia Major

Phase 1

A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants

Genetix Biotherapeutics Inc.β-thalassemia Major

Start: 2013-08-31End: 2018-02-21Updated: 2019-05-08

A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease

CompletedNCT02151526

Genetix Biotherapeutics Inc.Beta-Thalassemia Major, Sickle Cell Disease

Start: 2013-06-07End: 2019-02-26Updated: 2022-04-26

Phase 3

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

CompletedNCT02906202

Genetix Biotherapeutics Inc.Beta-Thalassemia

Start: 2016-08-08End: 2022-03-31Updated: 2023-06-18

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

CompletedNCT03207009

Genetix Biotherapeutics Inc.Beta-Thalassemia

Start: 2017-06-08End: 2022-11-15Updated: 2024-03-07

Long-Term Follow-up of Subjects With Transfusion-Dependant β-Thalassemia Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector

Active, not recruitingCTIS2024-513905-29-00

Genetix Biotherapeutics Inc.Beta-thalassaemia

Start: 2008-10-16Target: 24Updated: 2025-12-18

Nature Communications2025-04-019 citations

Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial.

2024-11-0822 citations

Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy Results in Durable Hemoglobin a Production with up to 10 Years of Follow-up with Transfusion-Dependent β-Thalassemia

Blood2024-11-051 citations

Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States.

2024-04-2913 citations

Sustained Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel)

Blood2023-11-025 citations

Improvement in Iron Burden in Patients with Transfusion-Dependent β-Thalassemia (TDT) Treated with Betibeglogene Autotemcel (Beti-cel) Gene Therapy: Up to 9 Years of Follow-up

Blood2023-11-021 citations

Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials

Blood2022-11-154 citations

Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial.

2022-01-2470 citations

Betibeglogene Autotemcel Gene Therapy for Non–β 0 /β 0 Genotype β-Thalassemia

New England Journal of Medicine2021-12-11235 citations

Improvement in Health-Related Quality of Life Following Treatment with Betibeglogene Autotemcel in Patients with Transfusion-Dependent β-Thalassemia Enrolled in Phase 3 Studies

Blood2021-11-053 citations

Wild-type HIV infection after treatment with lentiviral gene therapy for β-thalassemia

Blood Advances2021-07-018 citations

Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

Blood2020-11-051 citations

Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

Blood2020-11-0410 citations

Interim Results from the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy (LentiGlobin) for the Treatment of Transfusion-Dependent β-Thalassemia

Biology of Blood and Marrow Transplantation2020-01-239 citations

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Blood2019-11-1325 citations

Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy

Blood2019-11-1318 citations

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Blood2019-11-1314 citations

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Blood2019-11-1311 citations

S1632 SAFETY AND EFFICACY OF LENTIGLOBIN GENE THERAPY IN PATIENTS WITH TRANSFUSION‐DEPENDENT B‐THALASSAEMIA AND NON‐B0/B0 GENOTYPES IN THE PHASE 3 NORTHSTAR‐2 STUDY

HemaSphere2019-06-011 citations

S140 RESULTS FROM THE PHASE 3 NORTHSTAR‐3 STUDY EVALUATING LENTIGLOBIN GENE THERAPY IN PATIENTS WITH TRANSFUSION‐DEPENDENT β‐THALASSAEMIA AND A β0 OR IVS‐I‐110 MUTATION AT BOTH ALLELES OF THE HBB GENE

HemaSphere2019-06-011 citations

9 more papers not shown

LentiGlobin BB305 Drug Product

Sponsors

Conditions

Phase 1

Phase 3

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