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Journal Articles

An Update on Lovotibeglogene Autotemcel (Lovo-cel) Clinical Trials for Sickle Cell Disease (SCD) and Analysis of Early Predictors of Response to Lovo-Cel

Stacey Rifkin-Zenenberg, Julie Kanter, Melissa A. Kinney, Janet L. Kwiatkowski, Robert S. Nickel, Mark C. Walters, Suhag Parikh, Alexis Thompson, Anil P. George, Markus Mapara, Paul L. Martin, Anjulika Chawla, Ankit Lodaya, Lin Pan, Emily Sheldon-Waniga, Francis J. Pierciey, Jr., John Tisdale, Ashish O. Gupta

Blood (2024) 144 (Supplement 1): 511.

https://doi.org/10.1182/blood-2024-199743

Published: 2024

Journal Articles

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Successful Engraftment, Increased Hemoglobin, and Reduced Transfusion Dependence in Patients with Transfusion-Dependent Beta-Thalassemia Treated in the Edithal Trial

Haydar Frangoul, Rabi Hanna, Mark C. Walters, Kai-Hsin Chang, Michael Jaskolka, Keunpyo Kim, Baisong Mei, Olubunmi Afonja, Alexis Thompson

Blood (2024) 144 (Supplement 1): 7456.

https://doi.org/10.1182/blood-2024-208623

Published: 2024

Journal Articles

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Sustained Hemoglobin Normalization and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease Treated in the RUBY Trial

Rabi Hanna, Haydar Frangoul, Christopher McKinney, Luis Pineiro, Markus Mapara, Jignesh Dalal, Hemalatha Rangarajan, Harold Atkins, Kai-Hsin Chang, Baisong Mei, Olubunmi Afonja, Mark C. Walters

Blood (2024) 144 (Supplement 1): 4955.

https://doi.org/10.1182/blood-2024-210343

Published: 2024

Journal Articles

Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease

Blood (2024) 144 (Supplement 1): 4954.

https://doi.org/10.1182/blood-2024-204001

Published: 2024

Journal Articles

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

Blood (2024) 144 (Supplement 1): 2194.

https://doi.org/10.1182/blood-2024-200152

Published: 2024

Journal Articles

Reduced Intensity Haploidentical Bone Marrow Transplantation in Adults with Severe Sickle Cell Disease: BMT CTN 1507

Blood (2023) 142 (Supplement 2): LBA-4.

https://doi.org/10.1182/blood-2023-192022

Published: 2023

Journal Articles

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Blood (2023) 142 (Supplement 1): 1052.

https://doi.org/10.1182/blood-2023-190139

Published: 2023

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

Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD) Who Have Received Lovotibeglogene Autotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up

Julie Kanter, Alexis A. Thompson, Janet L. Kwiatkowski, Suhag Parikh, Markus Mapara, Stacey Rifkin-Zenenberg, Banu Aygun, Kimberly A. Kasow, Ashish O. Gupta, Lixin Zhang, Emily Sheldon-Waniga, Meghan Gallagher, Katiana Gruppioni, Anjulika Chawla, Heidi Elliot, Francis J. Pierciey, Mark C. Walters, John F. Tisdale

Blood (2023) 142 (Supplement 1): 1051.

https://doi.org/10.1182/blood-2023-174229

Published: 2023

Journal Articles

AsCas12a Gene Editing of HBG1/2 Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia

Rabi Hanna, Haydar Frangoul, Christopher McKinney, Luis Pineiro, Markus Mapara, Jignesh Dalal, Kai-Hsin Chang, Michael Jaskolka, Keunpyo Kim, Daphne L. Farrington, Maha Wally, Baisong Mei, Adebayo Lawal, Olubunmi O. Afonja, Mark C. Walters

Blood (2023) 142 (Supplement 1): 4996.

https://doi.org/10.1182/blood-2023-187397

Published: 2023

Journal Articles

Interim Safety and Efficacy Results from a Phase 1/2 Study of Zinc Finger Nuclease-Modified Autologous Hematopoietic Stem Cells for Sickle Cell Disease (PRECIZN-1)

Asif Alavi, Mehrdad Abedi, Suhag Parikh, Richard Boismenu, Michael Chen, Ben L. Hsu, Bettina M. Cockroft, Isobelle Galeon, Pablo Rendo, Mark C. Walters

Blood (2022) 140 (Supplement 1): 4907–4909.

https://doi.org/10.1182/blood-2022-163725

Published: 2022

Journal Articles

Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Severe Sickle Cell Disease

Haydar Frangoul, Franco Locatelli, Monica Bhatia, Markus Y. Mapara, Lyndsay Molinari, Akshay Sharma, Stephan Lobitz, Mariane de Montalembert, Damiano Rondelli, Martin Steinberg, Mark C. Walters, Suzan Imren, Lanju Zhang, Anjali Sharma, Yang Song, Christopher Simard, William Hobbs, Stephen Grupp

Blood (2022) 140 (Supplement 1): 29–31.

https://doi.org/10.1182/blood-2022-162353

Published: 2022

Journal Articles

Lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease: Updated Clinical Results and Investigations into Two Cases of Anemia from Group C of the Phase 1/2 HGB-206 Study

Mark C. Walters, Alexis A. Thompson, Janet L. Kwiatkowski, Suhag Parikh, Markus Y. Mapara, Stacey Rifkin-Zenenberg, Banu Aygun, Kimberly A. Kasow, Alex Miller, Lixin Zhang, Anjulika Chawla, Elizabeth R. Macari, Francis J. Pierciey, Jr., John F. Tisdale, Julie Kanter

Blood (2022) 140 (Supplement 1): 26–28.

https://doi.org/10.1182/blood-2022-162288

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

Access to CAR-T Cell Therapy in Underrepresented Populations: A Multicenter Cohort Study of Pediatric and Young Adult ALL Patients

Anurekha G. Hall, Lena E. Winestone, Erin M. Sullivan, Qian Vicky Wu, Adam J. Lamble, Mark C. Walters, Paibel Aguayo-Hiraldo, Lourdes Baez Conde, Tumaini R. Coker, Hema Dave, Dana Dornsife, Amy K. Keating, Diana M. Merino, Bonnie Ramsey, Julie R. Park, Anurag K. Agrawal

Blood (2021) 138 (Supplement 1): 339.

https://doi.org/10.1182/blood-2021-148256

Published: 2021

Journal Articles

Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

Blood (2021) 138 (Supplement 1): 561.

https://doi.org/10.1182/blood-2021-147760

Published: 2021

Journal Articles

Updated Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Mark C. Walters, Angela R. Smith, Gary J. Schiller, Erica B. Esrick, David A. Williams, Tatiana Gogoleva, Didier Rouy, Bettina M. Cockroft, Gregory M. Vercellotti

Blood (2021) 138 (Supplement 1): 3974.

https://doi.org/10.1182/blood-2021-147907

Published: 2021

Journal Articles

Preliminary Safety and Efficacy Results from Precizn-1: An Ongoing Phase 1/2 Study on Zinc Finger Nuclease-Modified Autologous CD34+ HSPCs for Sickle Cell Disease (SCD)

Asif Alavi, Lakshmanan Krishnamurti, Mehrdad Abedi, Isobelle Galeon, David Reiner, Sharon E Smith, Lin Wang, Anne Ramezi, Pablo Rendo, Mark C. Walters

Blood (2021) 138 (Supplement 1): 2930.

https://doi.org/10.1182/blood-2021-151650

Published: 2021

NARROW

An Update on Lovotibeglogene Autotemcel (Lovo-cel) Clinical Trials for Sickle Cell Disease (SCD) and Analysis of Early Predictors of Response to Lovo-Cel

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Successful Engraftment, Increased Hemoglobin, and Reduced Transfusion Dependence in Patients with Transfusion-Dependent Beta-Thalassemia Treated in the Edithal Trial

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Sustained Hemoglobin Normalization and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease Treated in the RUBY Trial

Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease

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

Reduced Intensity Haploidentical Bone Marrow Transplantation in Adults with Severe Sickle Cell Disease: BMT CTN 1507

Exagamglogene Autotemcel for Severe Sickle Cell Disease

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

Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD) Who Have Received Lovotibeglogene Autotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up

AsCas12a Gene Editing of HBG1/2 Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia

Interim Safety and Efficacy Results from a Phase 1/2 Study of Zinc Finger Nuclease-Modified Autologous Hematopoietic Stem Cells for Sickle Cell Disease (PRECIZN-1)

Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Severe Sickle Cell Disease

Lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease: Updated Clinical Results and Investigations into Two Cases of Anemia from Group C of the Phase 1/2 HGB-206 Study

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

Access to CAR-T Cell Therapy in Underrepresented Populations: A Multicenter Cohort Study of Pediatric and Young Adult ALL Patients

Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

Updated Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Preliminary Safety and Efficacy Results from Precizn-1: An Ongoing Phase 1/2 Study on Zinc Finger Nuclease-Modified Autologous CD34+ HSPCs for Sickle Cell Disease (SCD)

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American Society of Hematology

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American Society of Hematology

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