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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

Single and Combinational Multiplex Base-Edited 'Universal' CAR T Cells in a Humanised Model of Primary CD7+CD33+ AML

Arnold Kloos, Christos Georgiadis, Annie Etuk, Soragia Athina Gkazi, Farhatullah Syed, Toni Braybrook, Hong Zhan, Renuka Kadirkamanathan, Nadine Kattre, Kerstin Görlich, Thomas Fangmann, Axel Schambach, Martin G. Sauer, Michael Heuser, Waseem Qasim

Blood (2022) 140 (Supplement 1): 7425–7426.

https://doi.org/10.1182/blood-2022-168719

Published: 2022

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

Bcl11b -Edited Lymphoid Progenitors for the Generation of CAR-Engineered Natural Killer-like Cells with Potent Anti-Leukemic Activity

Franziska Baatz, Jessica Herbst, Johann Meyer, Axel Schambach, Tobias Maetzig, Michael Hust, Martin G. Sauer

Blood (2022) 140 (Supplement 1): 2862–2863.

https://doi.org/10.1182/blood-2022-157663

Published: 2022

Journal Articles

CD19-CAR-T Cells Are Effective and Safe Treatment of Post-Transplant Relapse in Pediatric and Young Adult Patients with B-Lineage ALL: Real-World Data from Germany

Blood (2022) 140 (Supplement 1): 10407–10409.

https://doi.org/10.1182/blood-2022-156775

Published: 2022

Journal Articles

Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia

Franco Locatelli, Mark C. Walters, Janet L. Kwiatkowski, Marina Cavazzana, Suradej Hongeng, John B. Porter, Adrian J. Thrasher, Andreas E. Kulozik, Isabelle Thuret, John E.J. Rasko, Evangelia Yannaki, Martin G. Sauer, Shamshad Ali, Himal Thakar, Katiana Gruppioni, Alexis A. Thompson

Blood (2022) 140 (Supplement 1): 8200–8202.

https://doi.org/10.1182/blood-2022-163178

Published: 2022

Journal Articles

Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis

Blood (2022) 139 (13): 2066–2079.

https://doi.org/10.1182/blood.2021014687

Published: 2022

Includes: Supplemental data

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

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

Journal Articles

Lentiglobin Gene Therapy for Patients with Transfusion-Dependent β-Thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies

Franco Locatelli, Mark C. Walters, MD, Janet L. Kwiatkowski, MD, John Porter, MA MDFRCP FRCPath, Martin G. Sauer, MD, Isabelle Thuret, MD, Suradej Hongeng, MD, Andreas E. Kulozik, MD PhD, Ashutosh Lal, MD, Adrian J. Thrasher, MD PhD FMedSci, Evangelia Yannaki, MD, Heidi Elliot, Ge Tao, PhD, Mohammed Asmal, MD PhD, Alexis A. Thompson, MD MPH

Blood (2018) 132 (Supplement 1): 1025.

https://doi.org/10.1182/blood-2018-99-112667

Published: 2018

Journal Articles

AML SCT-BFM 2007: Results of the Prospective Hematopoietic Stem Cell Transplantation (HCT) Trial of the Berlin-Frankfurt-MÃ¼nster (BFM) Study Group for Children with Very High Risk Acute Myeloid Leukemia

Blood (2017) 130 (Supplement 1): 599.

https://doi.org/10.1182/blood.V130.Suppl_1.599.599

Published: 2017

Journal Articles

Co-Transplantation of CAR-Gene Engineered Precursor T Cells Leads to Suppressed T Cell Development and Increased NK Cell-Based CAR-Mediated Anti-Leukemic Activity

Marcel Maluski, Jessica Herbst, Vanessa Scholl, Rolf Baumann, MD, Niels Heinz, PhD, Robert Geffers, PhD, Johann Meyer, PhD, Andreas Krueger, PhD, Axel Schambach, MD PhD, Martin G. Sauer

Blood (2017) 130 (Supplement 1): 1916.

https://doi.org/10.1182/blood.V130.Suppl_1.1916.1916

Published: 2017

Journal Articles

Outcome of Children Relapsing after First Allogeneic Hematopoietic Stem Cell Transplantation for Pediatric Acute Myeloid Leukemia: A Retrospective I-BFM Analysis of 336 Children between 2005 and 2016

Blood (2017) 130 (Supplement 1): 272.

https://doi.org/10.1182/blood.V130.Suppl_1.272.272

Published: 2017

Journal Articles

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-β 0 /β 0 Genotypes

Mark C. Walters, MD, Suradej Hongeng, MD, Janet L. Kwiatkowski, MD, Franco Locatelli, MD PhD, John B. Porter, BA MB BChir MRCP(Path) FRCP(Path) MD, Martin G. Sauer, Adrian J Thrasher, MD PhD FMedSci, Isabelle Thuret, MD, Evangelia Yannaki, MD PhD, Heidi Elliot, Marisa Gayron, Mohammed Asmal, MD PhD, Alexis A Thompson, MD MPH

Blood (2017) 130 (Supplement 1): 526.

https://doi.org/10.1182/blood.V130.Suppl_1.526.526

Published: 2017

Journal Articles

Timely Controlled T Cell Receptor Expression of Genetically Engineered Precursor T Cells Requires Early Transgene Induction for Leukemia Control after Hematopoietic Stem Cell Transplantation

Sayed Shahabuddin Hoseini, Martin Hapke, Jessica Herbst, Dirk Wedekind, PhD, Rolf Baumann, PhD, Andreas Krueger, Niels Heinz, Bernhard Schiedlmeier, PhD, Dario Vignali, PhD, Marcel R.M. van den Brink, MD PhD, Axel Schambach, MDPhD, Bruce R. Blazar, MD, Martin G. Sauer, MD

Blood (2014) 124 (21): 657.

https://doi.org/10.1182/blood.V124.21.657.657

Published: 2014

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

Single and Combinational Multiplex Base-Edited 'Universal' CAR T Cells in a Humanised Model of Primary CD7+CD33+ AML

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

Bcl11b -Edited Lymphoid Progenitors for the Generation of CAR-Engineered Natural Killer-like Cells with Potent Anti-Leukemic Activity

CD19-CAR-T Cells Are Effective and Safe Treatment of Post-Transplant Relapse in Pediatric and Young Adult Patients with B-Lineage ALL: Real-World Data from Germany

Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia

Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis

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

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

Lentiglobin Gene Therapy for Patients with Transfusion-Dependent β-Thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies

AML SCT-BFM 2007: Results of the Prospective Hematopoietic Stem Cell Transplantation (HCT) Trial of the Berlin-Frankfurt-MÃ¼nster (BFM) Study Group for Children with Very High Risk Acute Myeloid Leukemia

Co-Transplantation of CAR-Gene Engineered Precursor T Cells Leads to Suppressed T Cell Development and Increased NK Cell-Based CAR-Mediated Anti-Leukemic Activity

Outcome of Children Relapsing after First Allogeneic Hematopoietic Stem Cell Transplantation for Pediatric Acute Myeloid Leukemia: A Retrospective I-BFM Analysis of 336 Children between 2005 and 2016

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-β 0 /β 0 Genotypes

Timely Controlled T Cell Receptor Expression of Genetically Engineered Precursor T Cells Requires Early Transgene Induction for Leukemia Control after Hematopoietic Stem Cell Transplantation

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American Society of Hematology

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American Society of Hematology

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