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Haydar Frangoul
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Journal Articles

Gene therapy for HbSC disease and other compound heterozygous sickle hemoglobinopathies: a time for inclusion

Available to Purchase
Andrew Wilks, Martin H. Steinberg, Haydar Frangoul
Journal: Blood
Blood (2025) 146 (20): 2385–2391.
https://doi.org/10.1182/blood.2025029964
Published: 2025
Journal Articles

Correction of ineffective erythropoiesis and durable clinical benefit with exagamglogene autotemcel for transfusion-dependent β-thalassemia

Free
Franco Locatelli, Roland Meisel, Selim Corbacioglu, Josu de la Fuente, Mattia Algeri, Joachim Rupprecht, Kevin Kuo, Ami Shah, Peter Lang, Hayley Merkeley, Ben Carpenter, Markus Mapara, Robert Liem, Stephan Grupp, Yogi Chopra, Amanda Li, Janet Kwiatkowski, Melanie Kirby-Allen, Maria Domenica Cappellini, Antonis Kattamis, Sujit Sheth, Kevin Boerner, Sakellarios Zairis, Tina Liu, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2025) 146 (Supplement 1): 6089.
https://doi.org/10.1182/blood-2025-6089
Published: 2025
Journal Articles

Robust HbF induction and improvement of anemia and hemolysis with base editing in sickle cell disease: Safety and efficacy findings from the ongoing BEACON study

Free
Ashish Gupta, Akshay Sharma, Haydar Frangoul, Julie Kanter, Markus Mapara, Jignesh Dalal, Asif Alavi, Jennifer Jaroscak, Ernesto Ayala, John DiPersio, Edward D. Ziga, Mary Eapen, Stacey Rifkin-Zenenberg, Srikanth Ambati, Yinzhong Chen, Sarah Chesler, Marcelyne Joseney-Antoine, Leanne Ianniello, Bahru Habtemariam, Priya Chockalingam, Ling Lin, Sunita Goyal, Amy Simon, Alexis Thompson, Matthew Heeney
Journal: Blood
Blood (2025) 146 (Supplement 1): 2532.
https://doi.org/10.1182/blood-2025-2532
Published: 2025
Journal Articles

Enhanced CD34+ cell mobilizations, collections, and comparable safety profile with fixed-dose versus weight-based plerixafor dosing in patients with sickle cell disease receiving autologous CD34+ base-edited hematopoietic stem cells (BEAM-101) in the ongoing BEACON study

Free
Haydar Frangoul, Bahru Habtemariam, Arthur Lo, Srikanth Ambati, Yinzhong Chen, Sarah Chesler, Jigar Patel, Sunita Goyal, Amy Simon, Ashish Gupta
Journal: Blood
Blood (2025) 146 (Supplement 1): 1049.
https://doi.org/10.1182/blood-2025-1049
Published: 2025
Journal Articles

Long-term follow-up demonstrates durable clinical benefits of exagamglogene autotemcel for sickle cell disease with recurrent vaso-occlusive crises: Final results of climb SCD-121

Free
Stephan Grupp, Franco Locatelli, Akshay Sharma, Monica Bhatia, Markus Mapara, Robert Liem, Laurence Dedeken, Lyndsay Molinari, Michael Eckrich, Kevin Kuo, Paul Telfer, Ami Shah, Marina Cavazzana, Selim Corbacioglu, Mariane de Montalembert, Stephan Lobitz, Damiano Rondelli, Martin Steinberg, Mark Walters, Kevin Boerner, Tina Liu, Suzan Imren, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2025) 146 (Supplement 1): 2537.
https://doi.org/10.1182/blood-2025-2537
Published: 2025
Journal Articles

First in human prime edited autologous hematopoietic stem cell therapy for the treatment of p47phox CGD: Initial results of PRIME-0101

Free
Elie Haddad, Haydar Frangoul, Donald Kohn, Emma Morris, Jennifer Gori, Bradley Martin, Briana Deary, McKinley Nickerson, Alexandria Petrusich, Pierre Teira, Karine Leveille, Isabel Fernandez, Stuart Turvey, Elizabeth Kang, Suk See De Ravin, Marie Pierzynski, Tyra Estwick-McCann, Patricia Littel, Douglas Kuhns, Debra Long-Priel, Meghann McManus, Misty Evans, Ben Carpenter, Jordyn Arnold, Barrett Nehilla, Jacob Stewart-Ornstein, Tiernan O'Malley, Jack Heath, Mohammed Asmal, Harry Malech
Journal: Blood
Blood (2025) 146 (Supplement 1): 4319.
https://doi.org/10.1182/blood-2025-4319
Published: 2025
Journal Articles

CRISPR-Cas12a gene editing of the HBG1/2 promoters leads to sustained normalization of total hemoglobin and increased fetal hemoglobin in patients with severe sickle cell disease: Updated Results from the RUBY trial

Free
Rabi Hanna, Haydar Frangoul, Luis Pineiro, Christopher McKinney, Markus Mapara, Jignesh Dalal, Hemalatha Rangarajan, Harold Atkins, Monica Bhatia, Deepakbabu Chellapandian, Jennifer Jaroscak, Lakshmanan Krishnamurti, Janet Kwiatkowski, Lyndsay Molinari, Elizabeth Stenger, Michael Jaskolka, Qifeng Yu, Nnenna Badamosi, Brieana Cox-Buckley, Baisong Mei, Olubunmi Afonja, Mark Walters
Journal: Blood
Blood (2025) 146 (Supplement 1): 4314.
https://doi.org/10.1182/blood-2025-4314
Published: 2025
Journal Articles

First results of exagamglogene autotemcel in pediatric patients aged 5-11 years with transfusion-dependent β-thalassemia or sickle cell disease with recurrent severe vaso-occlusive crises

Free
Haydar Frangoul, Josu de la Fuente, Mattia Algeri, Yogi Chopra, Persis Amrolia, Akshay Sharma, Roland Meisel, Maria Domenica Cappellini, Selim Corbacioglu, Antonis Kattamis, Stephan Lobitz, Mariane de Montalembert, Damiano Rondelli, Sujit Sheth, Martin Steinberg, Mark Walters, Kevin Boerner, Katie Ender, Tina Liu, William Hobbs, Stephan Grupp, Franco Locatelli
Journal: Blood
Blood (2025) 146 (Supplement 1): 379.
https://doi.org/10.1182/blood-2025-379
Published: 2025
Journal Articles

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

Free
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

Free
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

Free
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

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

Free
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

Free
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

Free
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

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

Free
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

Free
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

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

Free
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

Free
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

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Free
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

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

Free
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