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Journal Articles

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)

Blood (2023) 142 (Supplement 1): 1102.

https://doi.org/10.1182/blood-2023-174068

Published: 2023

Journal Articles

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

Blood (2023) 142 (Supplement 1): 2480.

https://doi.org/10.1182/blood-2023-173869

Published: 2023

Journal Articles

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

Blood (2022) 140 (Supplement 1): 5355–5357.

https://doi.org/10.1182/blood-2022-162400

Published: 2022

Journal Articles

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

Janet L. Kwiatkowski, Franco Locatelli, Mark C. Walters, John B. Porter, Suradej Hongeng, Evangelia Yannaki, Andreas E. Kulozik, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, Ruiting Guo, Richard A. Colvin, Katiana Gruppioni, Alexis A. Thompson

Blood (2021) 138 (Supplement 1): 3085.

https://doi.org/10.1182/blood-2021-150392

Published: 2021

Journal Articles

Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up

Alexis A. Thompson, Franco Locatelli, Evangelia Yannaki, Mark C. Walters, John B. Porter, Suradej Hongeng, Andreas E. Kulozik, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, Marina Cavazzana, John E.J. Rasko, Lin Du, Richard A. Colvin, Janet L. Kwiatkowski

Blood (2021) 138 (Supplement 1): 573.

https://doi.org/10.1182/blood-2021-148177

Published: 2021

Journal Articles

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

Mark C. Walters, MD, David H.K. Chui, MD FRCPC, John J Farrell, PhD, Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Martin G. Sauer, MD, Isabelle Thuret, MD, Suradej Hongeng, MD, Andreas E. Kulozik, MD PhD, Adrian J. Thrasher, MBBS, PhD FMedSci, Evangelia Yannaki, MD, Julia Yang, Dustin Whitney, PhD, Alexandria Petrusich, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD

Blood (2020) 136 (Supplement 1): 1–3.

https://doi.org/10.1182/blood-2020-137642

Published: 2020

Journal Articles

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia: Results in Patients with up to 6 Years of Follow-up

Janet L. Kwiatkowski, MD MSCE, Mark C. Walters, MD, Suradej Hongeng, MD, Franco Locatelli, MD PhD, John E.J. Rasko, BSc, MBBS, PhD, Marina Cavazzana, MD PhD, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD

Blood (2020) 136 (Supplement 1): 51–52.

https://doi.org/10.1182/blood-2020-135850

Published: 2020

Journal Articles

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

Alexis A. Thompson, MD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Suradej Hongeng, MD, Evangelia Yannaki, MD, Andreas E. Kulozik, MD PhD, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Ashutosh Lal, MD, Ruiting Guo, MS, Weijian Liu, PhD, Richard A. Colvin, MD PhD, Mark C. Walters, MD, Franco Locatelli, MD PhD

Blood (2020) 136 (Supplement 1): 52–54.

https://doi.org/10.1182/blood-2020-135857

Published: 2020

Journal Articles

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

Alexis A. Thompson, MD MPH, Mark C. Walters, MD, Janet L. Kwiatkowski, MD MSCE, Suradej Hongeng, MD, John B. Porter, MA MDFRCP FRCPath, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Heidi Elliot, Ge Tao, PhD, Richard A. Colvin, MD PhD, Franco Locatelli, MD PhD

Blood (2019) 134 (Supplement_1): 3543.

https://doi.org/10.1182/blood-2019-126046

Published: 2019

Journal Articles

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

Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, Andreas E. Kulozik, MD PhD, Evangelia Yannaki, MD, John B. Porter, MA MDFRCP FRCPath, Isabelle Thuret, MD, Martin G. Sauer, MD, Heidi Elliot, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 815.

https://doi.org/10.1182/blood-2019-128482

Published: 2019

NARROW

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)

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

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

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

Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up

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

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia: Results in Patients with up to 6 Years of Follow-up

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

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

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

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American Society of Hematology

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American Society of Hematology

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