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Haydar Frangoul
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Journal Articles

Single Cell Hemoglobin Quantification Reveals HbF and HbS Increases in Sickle Erythrocytes from Patients Treated with Hydroxyurea

Laura Bencheikh, Laura Matabishi-Bibi, Gabriela Luszczewska, Meghan Perkins, Nicolas Hebert, Nagamy Rose Balli, Yosr Zaouali, Vincent Malcor Deydier De Pierrefeu, Fatima Bensiradj, Caroline Barau, France Pirenne, Carlo Brugnara, Martin H. Steinberg, Haydar Frangoul, Marie Cambot, Pablo Bartolucci
Journal: Blood
Blood (2024) 144 (Supplement 1): 1105.
https://doi.org/10.1182/blood-2024-209868
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
Journal: Blood
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

Haydar Frangoul, Franco Locatelli, Akshay Sharma, Monica Bhatia, Markus Y Mapara, Robert I. Liem, Donna Wall, Lyndsay Molinari, Laurence Dedeken, Kevin H.M. Kuo, Paul Telfer, Ami J. Shah, Marina Cavazzana, Selim Corbacioglu, Michael J. Eckrich, Damiano Rondelli, Roland Meisel, Stephan Lobitz, Mariane De Montalembert, Martin H. Steinberg, Mark C. Walters, Laura Bower, Suzan Imren, Tina Liu, Weiyu Zhou, William Hobbs, Stephan A. Grupp
Journal: Blood
Blood (2024) 144 (Supplement 1): 4954.
https://doi.org/10.1182/blood-2024-204001
Published: 2024
Journal Articles

Initial Results from the BEACON Clinical Study: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease with Severe Vaso-Occlusive Crises

Ashish O. Gupta, Akshay Sharma, Haydar Frangoul, Jignesh Dalal, Julie Kanter, Asif Alavi, John DiPersio, Mary Eapen, Jennifer J. Jaroscak, Ernesto Ayala, Edward D. Ziga, Stacey Rifkin-Zenenberg, Alex C. Minella, Guo Chen, Yinzhong Chen, Priya S. Chockalingam, Ling Lin, Marcelyne Joseney-Antoine, Leanne Ianniello, Beth Gardner, Adam J. Hartigan, Giuseppe Ciaramella, Sunita Goyal, Amy Simon, Alexis Thompson, Matthew M. Heeney
Journal: Blood
Blood (2024) 144 (Supplement 1): 513.
https://doi.org/10.1182/blood-2024-204888
Published: 2024
Journal Articles

Durable Clinical Benefits with Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Franco Locatelli, Peter Lang, Roland Meisel, Donna Wall, Selim Corbacioglu, Amanda M Li, Josu de la Fuente, Ami J. Shah, Ben Carpenter, Janet L. Kwiatkowski, Markus Y Mapara, Robert I. Liem, Maria Domenica Cappellini, Mattia Algeri, Antonis Kattamis, Sujit Sheth, Stephan A. Grupp, Hayley Lynne Merkeley, Kevin H.M. Kuo, Joachim Rupprecht, Puja Kohli, Gang Xu, Leorah Ross, Yael Bobruff, Bo Tong, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2024) 144 (Supplement 1): 512.
https://doi.org/10.1182/blood-2024-205779
Published: 2024
Journal Articles

Health-Related Quality-of-Life Improvements after Exagamglogene Autotemcel in Patients with Severe Sickle Cell Disease

Akshay Sharma, Haydar Frangoul, Franco Locatelli, Kevin H.M. Kuo, Monica Bhatia, Markus Y Mapara, Michael J. Eckrich, Suzan Imren, Nanxin Li, Jaime Rubin, Siyu Zhang, Tina Liu, William Hobbs, Stephan A. Grupp
Journal: Blood
Blood (2024) 144 (Supplement 1): 7453.
https://doi.org/10.1182/blood-2024-204112
Published: 2024
Journal Articles

Health-Related Quality-of-Life Improvements after Exagamglogene Autotemcel in Patients with Transfusion-Dependent Beta-Thalassemia

Josu de la Fuente, Franco Locatelli, Peter Lang, Roland Meisel, Donna Wall, Selim Corbacioglu, Amanda M Li, Ami J Shah, Markus Y Mapara, Robert I. Liem, Hayley Lynne Merkeley, Kevin H.M. Kuo, Joachim Rupprecht, Puja Kohli, Nanxin Li, Jaime Rubin, Siyu Zhang, Tina Liu, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2024) 144 (Supplement 1): 7454.
https://doi.org/10.1182/blood-2024-204115
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
Journal: Blood
Blood (2024) 144 (Supplement 1): 7456.
https://doi.org/10.1182/blood-2024-208623
Published: 2024
Journal Articles

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Franco Locatelli, Peter Lang, Selim Corbacioglu, Donna Wall, Roland Meisel, Amanda M Li, Josu de La Fuente, Ami J. Shah, Ben Carpenter, Janet L. Kwiatkowski, Markus Mapara, Robert I. Liem, Maria Domenica Cappellini, Mattia Algeri, Antonis Kattamis, Sujit Sheth, Stephan Grupp, Puja Kohli, Daoyuan Shi, Leorah Ross, Yael Bobruff, Christopher Simard, Lanju Zhang, Phuong Khanh Morrow, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2023) 142 (Supplement 1): 1053.
https://doi.org/10.1182/blood-2023-190534
Published: 2023
Journal Articles

Improvements in Health-Related Quality of Life after Exagamglogene Autotemcel in Patients with Transfusion-Dependent Beta-Thalassemia

Franco Locatelli, Peter Lang, Selim Corbacioglu, Donna Wall, Amanda M Li, Josu de La Fuente, Puja Kohli, Nanxin Li, Jaime Rubin, Siyu Zhang, Lanju Zhang, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2023) 142 (Supplement 1): 4997.
https://doi.org/10.1182/blood-2023-179916
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
Journal: Blood
Blood (2023) 142 (Supplement 1): 4996.
https://doi.org/10.1182/blood-2023-187397
Published: 2023
Journal Articles

Improvements in Health-Related Quality of Life after Exagamglogene Autotemcel in Patients with Severe Sickle Cell Disease

Akshay Sharma, Haydar Frangoul, Markus Mapara, Suzan Imren, Nanxin Li, Tina Liu, Jaime Rubin, Daoyuan Shi, William Hobbs, Stephan Grupp
Journal: Blood
Blood (2023) 142 (Supplement 1): 4999.
https://doi.org/10.1182/blood-2023-190203
Published: 2023
Journal Articles

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Haydar Frangoul, Franco Locatelli, Akshay Sharma, Monica Bhatia, Markus Mapara, Lyndsay Molinari, Donna Wall, Robert I. Liem, Paul Telfer, Ami J. Shah, Marina Cavazzana, Selim Corbacioglu, Damiano Rondelli, Roland Meisel, Laurence Dedeken, Stephan Lobitz, Mariane de Montalembert, Martin H. Steinberg, Mark C. Walters, Laura Bower, Suzan Imren, Christopher Simard, Fengjuan Xuan, Weiyu Zhou, Phuong Khanh Morrow, William Hobbs, Stephan Grupp
Journal: Blood
Blood (2023) 142 (Supplement 1): 1052.
https://doi.org/10.1182/blood-2023-190139
Published: 2023
Journal Articles

Differing Definitions of Vaso-Occlusion in Clinical Studies of Sickle Cell Disease Can Result in Differing Outcomes

Haydar Frangoul, Suzan Imren, Fengjuan Xuan, Nanxin Li, Jaime Rubin, William Hobbs, Franco Locatelli
Journal: Blood
Blood (2023) 142 (Supplement 1): 7107.
https://doi.org/10.1182/blood-2023-189246
Published: 2023
Journal Articles

Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Franco Locatelli, Peter Lang, Amanda Li, Selim Corbacioglu, Josu de la Fuente, Donna A. Wall, Robert Liem, Roland Meisel, Markus Y. Mapara, Ami J. Shah, Maria Domenica Domenica Cappellini, Antonis Kattamis, Sujit Sheth, Yael Bobruff, Laura Bower, Lanju Zhang, Anjali Sharma, Yang Song, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2022) 140 (Supplement 1): 4899–4901.
https://doi.org/10.1182/blood-2022-166881
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
Journal: Blood
Blood (2022) 140 (Supplement 1): 29–31.
https://doi.org/10.1182/blood-2022-162353
Published: 2022
Journal Articles

Reduction in Transplantation Activity without Impairment in Outcomes in the Covid-19 Era- Data from the Sarah Cannon Blood Cancer Network (SCBCN)

Minoo Battiwalla, MD, Carlos Bachier, MD, Vikas Bhushan, MD, Michael J. Eckrich, MD MPH, Alireza Eghtedar, MD, Haydar Frangoul, MD, Suman Kambhampati, MD, Shahbaz Malik, MD, Peter A. McSweeney, MD, Jeremy Pantin, MBBS, Aravind Ramakrishnan, MD, Hana Safah, MD, Paul J Shaughnessy, MD, Betsy Blunk, BSN, RN, BMTCN, Tonya Cox, BSN, RN, Rocky Billups, MS, Charles F. Lemaistre, MD
Journal: Blood
Blood (2020) 136 (Supplement 1): 26–27.
https://doi.org/10.1182/blood-2020-139121
Published: 2020
Journal Articles

Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9-Modified CD34+ Hematopoietic Stem and Progenitor Cells

Haydar Frangoul, MD, Yael Bobruff, PhD, Maria Domenica Cappellini, Selim Corbacioglu, MD, Christine Marie Fernandez, RN,MSN, Josu de la Fuente, Stephan A Grupp, MD PhD, Rupert Handgretinger, MD, Tony W. Ho, MD, Suzan Imren, MD, Antonis Kattamis, Julie Lekstrom-Himes, MD, Franco Locatelli, MD PhD, Yimeng Lu, PhD, Markus Y Mapara, MD PhD, Mariane de Montalembert, MD PhD, Sarah D. Mulcahey, MS, Damiano Rondelli, MD, Niraj Shanbhag, MD PhD, Sujit Sheth, MD, Sandeep Soni, MBBS, Martin H. Steinberg, MD, Michael J. Weinstein, John K. Wu, MBBS, FRCPC, MS, Donna Wall, MDCCPE
Journal: Blood
Blood (2020) 136 (Supplement 1): 3–4.
https://doi.org/10.1182/blood-2020-139575
Published: 2020
Journal Articles

Comparison of Outcomes for Myeloablative Conditioning Regimens Combining Busulfan with Either Cyclophosphamide or Fludarabine in Children

Andrew C. Harris, MD, Jaap Jan Boelens, MDPhD, Kwang Woo Ahn, PhD, Mingwei Fei, MS, Allistair Abraham, MD, Andrew Artz, MD, Christopher C. Dvorak, MD, Haydar Frangoul, MD, Cesar O Freytes, MD, Robert Peter Gale, MD PhD, Sanghee Hong, MD, Hillard M. Lazarus, MD, Shin Mineishi, MD, Taiga Nishihori, MD, Tracey O'Brien, MD, Kirsten M. Williams, MD, Marcelo C. Pasquini, MD, John E Levine, MD MS
Journal: Blood
Blood (2016) 128 (22): 664.
https://doi.org/10.1182/blood.V128.22.664.664
Published: 2016
Journal Articles

Incidence and Risk Factors for Hypogammaglobulinemia in Pediatric Allogeneic Stem Cell Transplant (SCT) Patients

Haydar Frangoul, MD, Elliot Min, BS, Zengqi Lu, Mario Mario, PhD, Richard Ho, MD, Valerie Brown, MD, PhD, Jennifer Domm, MD
Journal: Blood
Blood (2012) 120 (21): 4492.
https://doi.org/10.1182/blood.V120.21.4492.4492
Published: 2012