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Journal Articles

Interleukin-1 blockade in patients with Wiskott-Aldrich syndrome: a retrospective multinational case series

Blood (2024) 144 (16): 1699–1704.

https://doi.org/10.1182/blood.2024024524

Published: 2024

Includes: Supplemental data

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

Development of Anti-CD45 Antibody Drug Conjugates As Targeted Conditioning Agents for Transplantation/Gene Therapy with Potent Anti-Leukemic Properties

Jenny Yeung, Aiyin Liao, Matthew Shaw, Soraia Caetano-Silva, Winston Vetharoy, Ian Kirby, Francesca Zammarchi, Karin Havenith, Patrick H van Berkel, Claire Booth, H Bobby Gaspar, Adrian J. Thrasher, Kerry Chester, Persis J Amrolia

Blood (2022) 140 (Supplement 1): 10211–10212.

https://doi.org/10.1182/blood-2022-162390

Published: 2022

Journal Articles

Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)

Blood (2022) 140 (Supplement 1): 7774–7775.

https://doi.org/10.1182/blood-2022-158578

Published: 2022

Journal Articles

Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion

Blood (2022) 140 (Supplement 1): 7770–7771.

https://doi.org/10.1182/blood-2022-159979

Published: 2022

Journal Articles

Lentiviral-mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials

Blood (2022) 140 (Supplement 1): 10646–10647.

https://doi.org/10.1182/blood-2022-168342

Published: 2022

Journal Articles

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

Blood (2022) 140 (Supplement 1): 10643–10645.

https://doi.org/10.1182/blood-2022-159768

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

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

Biochemical Engraftment and Clinical Outcomes Following Ex-Vivo Autologous Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIA

Robert F Wynn, Jane L Kinsella, Rebecca J Holley, Jane Potter, Adrian J. Thrasher, Claire Booth, Karen Buckland, Natalia Izotova, Stewart Rust, Daniel Weisberg, Heather J Church, Karen L Tylee, Kathryn L Brammeier, Helena Lee, Laura Ford, Atusa Sadegholnejat, Simon A Jones, Stuart Ellison, Brian Bigger

Blood (2022) 140 (Supplement 1): 1897–1898.

https://doi.org/10.1182/blood-2022-168672

Published: 2022

Journal Articles

Gene Therapy for Fanconi Anemia [Group A]: Interim Results of RP-L102 Clinical Trials

Blood (2021) 138 (Supplement 1): 3968.

https://doi.org/10.1182/blood-2021-147071

Published: 2021

Journal Articles

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

Donald B. Kohn, Claire Booth, Julián Sevilla, Gayatri R Rao, Elena Almarza, Dayna Terrazas, Eileen Nicoletti, Augustine Fernandes, Caroline Kuo, Satiro de Oliveira, Theodore B. Moore, Kenneth M Law, Brian C Beard, Grace Choi, Miriam Zeini, Cristina Mesa-Núñez, Adrian J. Thrasher, Juan A Bueren, Jonathan D Schwartz

Blood (2021) 138 (Supplement 1): 2932.

https://doi.org/10.1182/blood-2021-151898

Published: 2021

Journal Articles

Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome

Blood (2021) 138 (Supplement 1): 2934.

https://doi.org/10.1182/blood-2021-147697

Published: 2021

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

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

Bipotent Lymphoid Progenitors Independently Maintain Long-Term Genetically Engineered T and NK Cell Production in Humans

Natalia Izotova, Christine Rivat, Cristina Baricordi, PhD, Elena Blanco-Alvarez, Danilo Pellin, Eleanor Watt, Soragia Athina Gkazi, PhD, Stuart Adams, PhD, Kimberly Gilmour, Jinhua Xu Bayford, Claire Booth, MBBS, PhD MSc, Bobby H Gaspar, MBBS, PhD BS, Adrian J. Thrasher, MBBS, PhD FMedSci, Luca Biasco, PhD

Blood (2020) 136 (Supplement 1): 50.

https://doi.org/10.1182/blood-2020-137001

Published: 2020

Journal Articles

Ex-Vivo Autologous Stem Cell Gene Therapy Clinical Trial for Mucopolysaccharidosis Type IIIA: Trial in Progress - NCT04201405

Jane L Kinsella, MBChB, Robert F Wynn, MRCP,MDFRCPath, Brian Bigger, PhD, Adrian J. Thrasher, MBBS, PhD FMedSci, Claire Booth, MBBS, PhD MSc, Karen Buckland, PhD, Natalia Izotova, Stewart Rust, B.Sc. (Hons), D.Clin.Psych, Pg.Dip., C.Psychol., AfBPS., Daniel Weisberg, BSc, PhD PGCert, ClinPsyD, Heather J Church, PhD, Kathryn L Brammeier, Karen L Tylee, Ceri Jones, BSc, Helena Lee, Laura Ford, Atusa Sadegholnejat, Stuart Ellison, Simon A Jones, MBChB, BSc, MRCPCH

Blood (2020) 136 (Supplement 1): 15–16.

https://doi.org/10.1182/blood-2020-141762

Published: 2020

Journal Articles

Editing the LRF Repressor Binding Site in the γ-Globin Promoters Induces Therapeutically Relevant Fetal Hemoglobin Levels for the Treatment of β-Hemoglobinopathies

Giacomo Frati, PhD, Panagiotis Antoniou, MSc, Giulia Hardouin, Bochra Mlaya, MSc, Leslie Weber, Tristan Felix, Antonio Casini, Marion Rosello, Anne Chalumeau, Mario Amendola, Adrian J. Thrasher, MBBS, PhD FMedSci, Giandomenico Turchiano, PhD, Filippo Del Bene, Wassim El Nemer, PhD, Anna Cereseto, Jean-Paul Concordet, Carine Giovannangeli, Marina Cavazzana, MD PhD, Annarita Miccio, PhD

Blood (2020) 136 (Supplement 1): 33.

https://doi.org/10.1182/blood-2020-139107

Published: 2020

NARROW

Interleukin-1 blockade in patients with Wiskott-Aldrich syndrome: a retrospective multinational case series

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

Development of Anti-CD45 Antibody Drug Conjugates As Targeted Conditioning Agents for Transplantation/Gene Therapy with Potent Anti-Leukemic Properties

Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)

Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion

Lentiviral-mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

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

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

Biochemical Engraftment and Clinical Outcomes Following Ex-Vivo Autologous Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIA

Gene Therapy for Fanconi Anemia [Group A]: Interim Results of RP-L102 Clinical Trials

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome

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

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

Bipotent Lymphoid Progenitors Independently Maintain Long-Term Genetically Engineered T and NK Cell Production in Humans

Ex-Vivo Autologous Stem Cell Gene Therapy Clinical Trial for Mucopolysaccharidosis Type IIIA: Trial in Progress - NCT04201405

Editing the LRF Repressor Binding Site in the γ-Globin Promoters Induces Therapeutically Relevant Fetal Hemoglobin Levels for the Treatment of β-Hemoglobinopathies

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American Society of Hematology

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