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Journal Articles

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

Blood (2025) 146 (Supplement 1): 6089.

https://doi.org/10.1182/blood-2025-6089

Published: 2025

Journal Articles

CD19 CAR T cell therapy is an effective strategy for first CNS relapse in pediatric b ALL

Blood (2025) 146 (Supplement 1): 648.

https://doi.org/10.1182/blood-2025-648

Published: 2025

Journal Articles

Humanized CD19 chimeric antigen receptor (CAR) T-cell therapy for high-risk and post-CAR relapse of B-cell acute lymphoblastic leukemia

Blood (2025) 146 (Supplement 1): 646.

https://doi.org/10.1182/blood-2025-646

Published: 2025

Journal Articles

Toward ‘n of one’ gene therapies: Scalable ex vivo adenine base editing via lipid nanoparticles in a sickle cell disease model

Sarah Cotteta, Omar Banda, Friederike Herbst-Nowrouzi, Brooklyn McDonald, Yongping Wang, Mohamad-Gabriel Alameh, Frederic Bushman, Stephan Grupp, Eugene Khandros, Stephan Kadauke

Blood (2025) 146 (Supplement 1): 7835.

https://doi.org/10.1182/blood-2025-7835

Published: 2025

Journal Articles

CD123-directed chimeric antigen receptor T-cell (CART123) therapy for relapsed/refractory pediatric acute myeloid leukemia

Blood (2025) 146 (Supplement 1): 1643.

https://doi.org/10.1182/blood-2025-1643

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

Blood (2025) 146 (Supplement 1): 379.

https://doi.org/10.1182/blood-2025-379

Published: 2025

Journal Articles

Single-cell transcriptomics of pediatric B-ALL bone marrow to determine mechanisms of durable CD19 CAR T cell persistence

Jeremy Grenier, Katherine Mueller, Steven Pastor, Gabrielle Zuern, Susan McClory, Matthew Bode, Gbemi Olarewaju, Caroline Diorio, Prabhat Sharma, Regina Myers, Stephan Grupp, Evan Weber

Blood (2025) 146 (Supplement 1): 500.

https://doi.org/10.1182/blood-2025-500

Published: 2025

Journal Articles

Optimal therapy for pediatric B-ALL in first relapse: Comparative effectiveness of blinatumomab to hematopoietic cell transplantation versus CD19 CAR T cell therapy

Regina Myers, Yimei Li, Hongyan Liu, Alex Hoover, Laura Shinehouse, Ashley Hinson, Mira Kohorst, Anna Elias, Ella Keenan, Hannah Acres, Ibrahim Ahmed, Abdulla Al-Mulla, Laura Arthur, Jill Beck, Abigale Berry, Deepika Bhatla, Eliza Briscoe, Grace Chang, Roland Chu, Laurie Davis, J. Gregory Dolan, Lyannette Elo, Elizabeth Eom, Alejandra Escobar Vasco, Vanessa Fabrizio, Kelly Faulk, Victor Galan-Gomez, Erin Goode, Stephan Grupp, Anurekha Hall, Darcy Hamill, Smitha Vasanna, Hannah Kinoshita, Elizabeth Krieger, Cathy Lee-Miller, Katherine Lind, Jordan Milner, Giselle Moore-Higgs, Amy Moskop, Lindsey Murphy, Megan Murphy, Gabriella Nguyen, Maria Ortega, Hiren Patel, Antonio Perez, Thomas Pfeiffer, Alexandra Prosser-Dombrowski, Mahvish Rahim, April Rahrig, Shanti Ramachandran, Miza Salim Hammoud, Amanda Saraf, Jamie Shoag, Heather Symons, Nicholas Tastet, Stephanie Thomas, Molly Thornock, Keri Toner, Allison Weisnicht, Sara Zarnegar-Lumley, Nirali Shah, Shannon Maude, Jeremy Rubinstein, Troy Quigg, Deepa Bhojwani, Caitlin Elgarten

Blood (2025) 146 (Supplement 1): 3339.

https://doi.org/10.1182/blood-2025-3339

Published: 2025

Journal Articles

Improved hematopoietic stem cell mobilization for gene therapy using single agent motixafortide in sickle cell disease

Stephan Grupp, Michael Grimley, Timothy Driscoll, Kris Mahadeo, Alexis Leonard, Yongping Wang, Helen Phillips, David Justus, Leslie Lehmann, Erica Esrick, Julie Kanter, Sarah Nikiforow, Matthew Hsieh, John Tisdale, Akshay Sharma, Zachary Crees, John Manis

Blood (2025) 146 (Supplement 1): 2542.

https://doi.org/10.1182/blood-2025-2542

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

Blood (2025) 146 (Supplement 1): 2537.

https://doi.org/10.1182/blood-2025-2537

Published: 2025

Journal Articles

Outcomes and Toxicities after Allogeneic Hematopoietic Cell Transplant for Remission Consolidation or Relapse Therapy after CD19 Chimeric Antigen Receptor T Cell Therapy in Children and Young Adults with Acute Lymphoblastic Leukemia

Regina M. Myers, Sarah Mumanachit, Shannon L. Maude, Amanda M. Dinofia, Jason L. Freedman, Nancy Bunin, Lisa Wray, Allison Barz Leahy, Timothy S Olson, Stephan Grupp, Alix E. Seif, Caitlin W. Elgarten

Blood (2023) 142 (Supplement 1): 3767.

https://doi.org/10.1182/blood-2023-188047

Published: 2023

Journal Articles

Significant Cytokine Release Syndrome Risk Model with T-Cell Engaging Therapies

Penelope Lafeuille, William Blumentals, Claire Brulle Wohlhueter, Weixi Chen, Chao Sang, Sydney Manning, Silvy Saltzman, Jan Canvin, Susan Richard, Cris Kamperschroer, Giovanni Abbadessa, Aniketh Talwai, Caroline Der-Nigoghossian, Yahav Itzkovich, Vibhu Agarwal, Rahul Jain, Tanmay Jain, Jacob Aptekar, Stephan Grupp

Blood (2023) 142 (Supplement 1): 3629.

https://doi.org/10.1182/blood-2023-181985

Published: 2023

Journal Articles

IL-10 Levels Differentially Regulate IFNg Signaling of CART19 and CART22

Caroline Diorio, Regina M. Myers, Anastasia Frank-Kamenetski, Rawan Shraim, Amanda M. Dinofia, Zachary Martinez, Ohaila Mustafa, Joseph A Fraietta, Vanessa Gonzalez, Vivian Yi, Lahari Uppuluri, Haley Newman, Edward Behrens, Carl June, Scott Canna, Stephan Grupp, David T. Teachey

Blood (2023) 142 (Supplement 1): 3431.

https://doi.org/10.1182/blood-2023-182377

Published: 2023

Journal Articles

Updated Results of the Phase I BALLI-01 Trial of UCART22 Process 2 (P2), an Anti-CD22 Allogeneic CAR-T Cell Product Manufactured By Cellectis Biologics, in Patients with Relapsed or Refractory (R/R) CD22+ B-Cell Acute Lymphoblastic Leukemia (B-ALL)

Nitin Jain, Patrice Chevallier, Hongtao Liu, Gary J. Schiller, Jean-Baptiste Méar, Daniel J. DeAngelo, Kevin J Curran, Stephan Grupp, Andre Baruchel, Marie Balsat, Alexandra LaCroce, Caroline Roudet, Ana B. Korngold, Kathryn J. Newhall, Eric Laille, Daniel J. Lee, Mark G. Frattini, Richard A. Larson, Nicolas Boissel

Blood (2023) 142 (Supplement 1): 4847.

https://doi.org/10.1182/blood-2023-187252

Published: 2023

Journal Articles

Long Term Durable Responses in Relapsed/Refractory (r/r) ALL, DLBCL, and FL Patients Treated with Tisagenlecleucel and Its Association with Persistence of CAR T-Cells

Rakesh Awasthi, Edward Waldron, Stephan Grupp, Michael A. Pulsipher, Peter Bader, Stephen J Schuster, Richard T Maziarz, Edmund K. Waller, Ulrich Jaeger, Catherine Thieblemont, Martin Dreyling, Nathan Fowler, Harald Jakob Maier, Jennifer Willert

Blood (2023) 142 (Supplement 1): 4872.

https://doi.org/10.1182/blood-2023-181663

Published: 2023

Journal Articles

Single-Cell Multi-Omics Reveals Type-2 Functionality in Maintaining CAR T Cell Longevity Associated with 8-Year Leukemia Remission

Zhiliang Bai, Bing Feng, Susan E. Mcclory, Caroline Diorio, Ziran Zhao, Li Tang, J. Joseph Melenhorst, Carl June, Stephan Grupp, Rong Fan

Blood (2023) 142 (Supplement 1): 352.

https://doi.org/10.1182/blood-2023-179480

Published: 2023

Journal Articles

The MAGIC Algorithm Probability Predicts Long-Term Outcomes and Treatment Response to Second-Line Therapy for Acute Gvhd

Blood (2023) 142 (Supplement 1): 3576.

https://doi.org/10.1182/blood-2023-182300

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

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

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

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

Published: 2023

Journal Articles

Flares of Acute Graft-Versus-Host Disease: Mount Sinai Acute Gvhd International Consortium (MAGIC) Study

Blood (2023) 142 (Supplement 1): 776.

https://doi.org/10.1182/blood-2023-181900

Published: 2023

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

CD19 CAR T cell therapy is an effective strategy for first CNS relapse in pediatric b ALL

Humanized CD19 chimeric antigen receptor (CAR) T-cell therapy for high-risk and post-CAR relapse of B-cell acute lymphoblastic leukemia

Toward ‘n of one’ gene therapies: Scalable ex vivo adenine base editing via lipid nanoparticles in a sickle cell disease model

CD123-directed chimeric antigen receptor T-cell (CART123) therapy for relapsed/refractory pediatric acute myeloid leukemia

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

Single-cell transcriptomics of pediatric B-ALL bone marrow to determine mechanisms of durable CD19 CAR T cell persistence

Optimal therapy for pediatric B-ALL in first relapse: Comparative effectiveness of blinatumomab to hematopoietic cell transplantation versus CD19 CAR T cell therapy

Improved hematopoietic stem cell mobilization for gene therapy using single agent motixafortide in sickle cell disease

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

Outcomes and Toxicities after Allogeneic Hematopoietic Cell Transplant for Remission Consolidation or Relapse Therapy after CD19 Chimeric Antigen Receptor T Cell Therapy in Children and Young Adults with Acute Lymphoblastic Leukemia

Significant Cytokine Release Syndrome Risk Model with T-Cell Engaging Therapies

IL-10 Levels Differentially Regulate IFNg Signaling of CART19 and CART22

Updated Results of the Phase I BALLI-01 Trial of UCART22 Process 2 (P2), an Anti-CD22 Allogeneic CAR-T Cell Product Manufactured By Cellectis Biologics, in Patients with Relapsed or Refractory (R/R) CD22+ B-Cell Acute Lymphoblastic Leukemia (B-ALL)

Long Term Durable Responses in Relapsed/Refractory (r/r) ALL, DLBCL, and FL Patients Treated with Tisagenlecleucel and Its Association with Persistence of CAR T-Cells

Single-Cell Multi-Omics Reveals Type-2 Functionality in Maintaining CAR T Cell Longevity Associated with 8-Year Leukemia Remission

The MAGIC Algorithm Probability Predicts Long-Term Outcomes and Treatment Response to Second-Line Therapy for Acute Gvhd

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

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Flares of Acute Graft-Versus-Host Disease: Mount Sinai Acute Gvhd International Consortium (MAGIC) Study

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