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Journal Articles

Long-term follow-up of the first in human post-transcriptional genetic silencing of BCL11A in sickle cell disease in a phase 1 pilot and feasibility trial

Blood (2025) 146 (Supplement 1): 2533.

https://doi.org/10.1182/blood-2025-2533

Published: 2025

Journal Articles

Efficient, potent, and specific near-universal base and prime editing for ELANE-mutant severe congenital neutropenia

Jing Zeng, Nola Neri, Josias Soares de Brito, Archana Verma, Sébastien Levesque, Gue-Ho Hwang, Viktoria Jung, Gabriele Casirati, Felicia Andresen, David Justus, Denisa Wagner, John Manis, Danilo Pellin, Pietro Genovese, Akiko Shimamura, Myriam Armant, Scot Wolfe, Peter Newburger, Daniel Bauer

Blood (2025) 146 (Supplement 1): 624.

https://doi.org/10.1182/blood-2025-624

Published: 2025

Journal Articles

First-in-human phase 1 trial of trimeric APRIL (TriPRIL) CAR T cells in relapsed or refractory multiple myeloma: Clinical outcomes and biological correlates of disease response and resistance

Blood (2025) 146 (Supplement 1): 270.

https://doi.org/10.1182/blood-2025-270

Published: 2025

Journal Articles

Highly efficient collection and manufacture of autologous HSC gene therapy cell product for patients with sickle cell disease using a lentiviral vector containing a shmir targeting BCL11a

Blood (2025) 146 (Supplement 1): 6094.

https://doi.org/10.1182/blood-2025-6094

Published: 2025

Journal Articles

Efficient prime editing of human hematopoietic stem cells corrects the recurrent SBDS C.258+2T>C mutation and recovers engraftment potential in shwachman-diamond syndrome

Gue-Ho Hwang, Michael Peters, Felicia Andresen, Jing Zeng, Haarika Kathi, Sébastien Levesque, Chad Harris, Christy Kolsteeg, Nola Neri, Archana Verma, Eva Wauters, Chi Yuan Zhang, Myriam Armant, Christian Brendel, Scot Wolfe, John Manis, Akiko Shimamura, Daniel Bauer

Blood (2025) 146 (Supplement 1): 1063.

https://doi.org/10.1182/blood-2025-1063

Published: 2025

Journal Articles

Human Gene Therapy Optimized for the Platelet Bleeding Disorder Glanzmann Thrombasthenia

Juan Fang, Matthew Hogenauer, Aoife M. Roche, Sevasti B. Koukouritaki, Lily M. Du, Huiqing Xu, John Everett, Frederic D. Bushman, Myriam Armant, Peiman Hematti, David A. Wilcox

Blood (2024) 144 (Supplement 1): 5409.

https://doi.org/10.1182/blood-2024-199243

Published: 2024

Journal Articles

Impact of BEAM-101 Treatment on Red Blood Cell Hemoglobin Expression, Rheology and Sickling Properties: Initial Data from the BEACON Phase 1/2 Study of Autologous CD34 + Base Edited Hematopoietic Stem Cells in Sickle Cell Disease

Priya S. Chockalingam, Ling Lin, Guo Chen, Alex C. Minella, Yinzhong Chen, Vivien Sheehan, Nan Zhang, Myriam Armant, Aliya U. Zaidi, Robert Goodrich, Patrick C. Hines, Sunita Goyal, Amy Simon

Blood (2024) 144 (Supplement 1): 4957.

https://doi.org/10.1182/blood-2024-200875

Published: 2024

Journal Articles

Phase 1 study of CAR-37 T cells in patients with relapsed or refractory CD37 + lymphoid malignancies

Blood (2024) 144 (11): 1153–1167.

https://doi.org/10.1182/blood.2024024104

Published: 2024

Includes: Supplemental data

Journal Articles

Gene Therapy Expressing Platelet-Derived Factor VIII for Correction of Hemophilia Α with a History of Inhibitors

David A. Wilcox, Myriam Armant, Lynn Malec, Qizhen Shi, Bryon D. Johnson, Huiqing Xu, Lily M. Du, Lisa J. Duffy, Frederic D. Bushman, James H. Jerkins, Peiman Hematti, Ruta Brazauskas, Shawn M. Jobe, Parameswaran N. Hari, Mary Eapen

Blood (2023) 142 (Supplement 1): 2249.

https://doi.org/10.1182/blood-2023-172845

Published: 2023

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

Therapeutic Gene Editing of HSCs Ex Vivo without in Vitro Culture Avoids Genotoxicity, Simplifies Procedures, and Preserves Efficiency and Stemness

Jing Zeng, My Anh Nguyen, Marioara-Felicia Ciuculescu, Esther Mintzer, Pengpeng Liu, Linda Yingqi Lin, Tolulope O Rosanwo, Archana Verma, Nola Neri, Stacy A. Maitland, Alden Richter, David G. Justus, Kendell Clement, Christian Brendel, Luca Pinello, John P Manis, Myriam Armant, Scot A. Wolfe, Daniel E. Bauer

Blood (2022) 140 (Supplement 1): 10671–10672.

https://doi.org/10.1182/blood-2022-169900

Published: 2022

Journal Articles

Regulated Expression of GATA1 As a Gene Therapy Cure for Diamond-Blackfan Anemia

Richard A Voit, Xiaotian Liao, Blake Cohen, Myriam Armant, Elena Kamal, Mei-Mei Huang, William Clarke, David A. Williams, Akiko Shimamura, Vijay G. Sankaran

Blood (2022) 140 (Supplement 1): 986–987.

https://doi.org/10.1182/blood-2022-165848

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

Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial

Blood (2022) 140 (Supplement 1): 10665–10667.

https://doi.org/10.1182/blood-2022-169089

Published: 2022

Journal Articles

Effects of BCL11A Shmir-Induced Post-Transcriptional Silencing on Distributions of HbF in Single-RBCs and Reticulocytes

Nicolas Hebert, Erica B. Esrick, Myriam Armant, Christian Brendel, Marioara Felicia Ciuculescu, Etienne Audureau, John M. Higgins, David A. Williams, Pablo Bartolucci

Blood (2021) 138 (Supplement 1): 967.

https://doi.org/10.1182/blood-2021-150186

Published: 2021

Journal Articles

Effects of BCL11A Shmir-Induced Post-Transcriptional Silencing on Hemoglobin Polymer Inhibition in Single Red Blood Cells at Physiologic Oxygen Tension

Daniel C. De Souza, Erica B. Esrick, Nicolas Hebert, Giuseppe Di Caprio, Marioara Ciuculescu, Emily Morris, Myriam Armant, Bronner P. Gonçalves, Ethan Schonbrun, Christian Brendel, David K. Wood, Pablo Bartolucci, David A. Williams, John M. Higgins

Blood (2021) 138 (Supplement 1): 964.

https://doi.org/10.1182/blood-2021-147314

Published: 2021

Journal Articles

Clonal Tracking By Whole Genome Sequencing Permits Comprehensive Mapping of the Genomic Landscape in Pre- and Post-Gene Therapy Sickle Cell Patients

Alyssa H. Cull, Michael Spencer Chapman, Marioara Ciuculescu, Emily Mitchell, Myriam Armant, Grace Boyd, Erica B. Esrick, David A. Williams, Peter J. Campbell, David G. Kent

Blood (2021) 138 (Supplement 1): 559.

https://doi.org/10.1182/blood-2021-152262

Published: 2021

Journal Articles

Combined +58 and +55 BCL11A enhancer Editing Yields Exceptional Efficiency, Specificity and HbF Induction in Human and NHP Preclinical Models

Blood (2021) 138 (Supplement 1): 1852.

https://doi.org/10.1182/blood-2021-152634

Published: 2021

Journal Articles

Metformin for Treatment of Cytopenias in Children and Young Adults with Fanconi Anemia

Blood (2021) 138 (Supplement 1): 1102.

https://doi.org/10.1182/blood-2021-153598

Published: 2021

Journal Articles

Phase 1 Study of CD37-Directed CAR T Cells in Patients with Relapsed or Refractory CD37+ Hematologic Malignancies

Matthew J. Frigault, Yi-Bin Chen, Kathleen M.E. Gallagher, Nora K. Horick, Areej El-Jawahri, Irene Scarfò, Marc Wehrli, Lu Huang, Keagan Casey, Daniella Cook, Thomas Spitzer, Steven McAfee, Frederic I. Preffer, Myriam Armant, Kit L. Shaw, Heather Daley, Sarah Nikiforow, Jerome Ritz, Marcela V. Maus

Blood (2021) 138 (Supplement 1): 653.

https://doi.org/10.1182/blood-2021-146236

Published: 2021

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Long-term follow-up of the first in human post-transcriptional genetic silencing of BCL11A in sickle cell disease in a phase 1 pilot and feasibility trial

Efficient, potent, and specific near-universal base and prime editing for ELANE-mutant severe congenital neutropenia

First-in-human phase 1 trial of trimeric APRIL (TriPRIL) CAR T cells in relapsed or refractory multiple myeloma: Clinical outcomes and biological correlates of disease response and resistance

Highly efficient collection and manufacture of autologous HSC gene therapy cell product for patients with sickle cell disease using a lentiviral vector containing a shmir targeting BCL11a

Efficient prime editing of human hematopoietic stem cells corrects the recurrent SBDS C.258+2T>C mutation and recovers engraftment potential in shwachman-diamond syndrome

Human Gene Therapy Optimized for the Platelet Bleeding Disorder Glanzmann Thrombasthenia

Impact of BEAM-101 Treatment on Red Blood Cell Hemoglobin Expression, Rheology and Sickling Properties: Initial Data from the BEACON Phase 1/2 Study of Autologous CD34 + Base Edited Hematopoietic Stem Cells in Sickle Cell Disease

Phase 1 study of CAR-37 T cells in patients with relapsed or refractory CD37 + lymphoid malignancies

Gene Therapy Expressing Platelet-Derived Factor VIII for Correction of Hemophilia Α with a History of Inhibitors

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

Therapeutic Gene Editing of HSCs Ex Vivo without in Vitro Culture Avoids Genotoxicity, Simplifies Procedures, and Preserves Efficiency and Stemness

Regulated Expression of GATA1 As a Gene Therapy Cure for Diamond-Blackfan Anemia

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

Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial

Effects of BCL11A Shmir-Induced Post-Transcriptional Silencing on Distributions of HbF in Single-RBCs and Reticulocytes

Effects of BCL11A Shmir-Induced Post-Transcriptional Silencing on Hemoglobin Polymer Inhibition in Single Red Blood Cells at Physiologic Oxygen Tension

Clonal Tracking By Whole Genome Sequencing Permits Comprehensive Mapping of the Genomic Landscape in Pre- and Post-Gene Therapy Sickle Cell Patients

Combined +58 and +55 BCL11A enhancer Editing Yields Exceptional Efficiency, Specificity and HbF Induction in Human and NHP Preclinical Models

Metformin for Treatment of Cytopenias in Children and Young Adults with Fanconi Anemia

Phase 1 Study of CD37-Directed CAR T Cells in Patients with Relapsed or Refractory CD37+ Hematologic Malignancies

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