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Journal Articles

Single Agent CD45-Targeted Antibody Drug Conjugate Enables Full Mismatch Allogeneic Hematopoietic Stem Cell Transplantation in a Murine HSCT Model

Sharon L. Hyzy, MS, Jennifer L Proctor, BS, Geoff O Gillard, PhD, Katelyn J. Hammond, BS, Ganapathy N. Sarma, PhD, Asim Saha, PhD, Nicholas Clark, PhD, Anjali Bhat, MS, Tahirih L Lamothe, BS, Rahul Palchaudhuri, PhD, Bradley R Pearse, PhD, Charlotte F McDonagh, PhD, Hans-Peter Kiem, MDPhD, John E Wagner, MD, Bruce R. Blazar, MD, Anthony E Boitano, PhD, Michael P Cooke, PhD, John C. Davis, Jr., MD MPH, MS

Blood (2020) 136 (Supplement 1): 31.

https://doi.org/10.1182/blood-2020-140489

Published: 2020

Journal Articles

Reversing Clonal Hematopoiesis and Associated Atherosclerotic Disease By Targeted Antibody-Drug-Conjugate (ADC) Conditioning and Transplant

Karin Gustafsson, PhD, Catherine S Rhee, PhD, Elizabeth W Scadden, Vanessa Frodermann, Rahul Palchaudhuri, PhD, Sharon L. Hyzy, MS, Jennifer L Proctor, BS, Geoff O Gillard, PhD, Anthony E Boitano, PhD, Michael P. Cooke, PhD, Matthias Nahrendorf, PhD, David T. Scadden, MD

Blood (2020) 136 (Supplement 1): 34–35.

https://doi.org/10.1182/blood-2020-143176

Published: 2020

Journal Articles

A Single Dose of a Novel Anti-Human CD117-Amanitin Antibody Drug Conjugate (ADC) Engineered for a Short Half-Life Provides Dual Conditioning and Anti-Leukemic Activity and Extends Survival Compared to Standard of Care in Multiple Preclinical Models of Acute Myeloid Leukemia (AML)

Leanne Lanieri, Tahirih L Lamothe, BS, Oliver Miske, Sean M McDonough, Ganapathy N Sarma, Prashant Bhattarai, Kellie Latimer, Junia Dushime, Nidhi Jain, Rahul Palchaudhuri, PhD, Ryan Knihtila, Bradley R Pearse, PhD, Jennifer L Proctor, BS, Anthony E Boitano, PhD, Michael P Cooke, PhD, John C. Davis, Jr., MD MPH, MS

Blood (2020) 136 (Supplement 1): 47–48.

https://doi.org/10.1182/blood-2020-140422

Published: 2020

Journal Articles

A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates

Blood (2019) 134 (Supplement_1): 610.

https://doi.org/10.1182/blood-2019-125968

Published: 2019

Journal Articles

A Novel Targeted Approach to Achieve Immune System Reset: CD45-Targeted Antibody Drug Conjugates Enable Autologous HSCT, Ameliorate Disease in Autoimmune Models, Potently Kill Human Immune Cells from Normal Donors and MS Patients, and Achieve Immune Depletion in Non-Human Primates (NHP)

Geoff O Gillard, Jennifer L Proctor, BS, Melissa L. Brooks, BS, Tahirih L Lamothe, BS, Sharon L. Hyzy, MS, Oliver Mikse, Sean McDonough, Rahul Palchaudhuri, PhD, Anjali Bhat, MS, Ganapathy N. Sarma, PhD, Prashant Bhattarai, Pranoti Sawant, MS, Bradley R Pearse, PhD, Charlotte F. Mcdonough, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2019) 134 (Supplement_1): 3208.

https://doi.org/10.1182/blood-2019-130752

Published: 2019

Journal Articles

High Dose Hematopoietic Stem Cell Transplantation Leads to Rapid Hematopoietic and Microglia Recovery and Disease Correction in a Mouse Model of Hurler Syndrome

Kevin A. Goncalves, PhD, Sharon L. Hyzy, MS, Melissa L. Brooks, BS, Hans J. Hertzler, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2019) 134 (Supplement_1): 4424.

https://doi.org/10.1182/blood-2019-131189

Published: 2019

Journal Articles

Rapid and Robust Mobilization of CD34+ HSCs without G-CSF Following Administration of Mgta-145 Alone or in Combination with Plerixafor

John F. DiPersio, MD PhD, Jonathan Hoggatt, PhD, Steven Devine, MD, Lukasz Biernat, MD, Haley Howell, BS, BA, Veit Schmelmer, PhD, Jason R. Neale, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD, Kevin A. Goncalves, PhD, Glen D. Raffel, MD PhD, Patrick C. Falahee, PhD, Dwight M. Morrow, PhD, John C. Davis, Jr., MD MPH, MS

Blood (2019) 134 (Supplement_1): 1961.

https://doi.org/10.1182/blood-2019-127523

Published: 2019

Journal Articles

Ex Vivo Expansion with E478 Increases the Rate of CRISPR-Mediated Homology Directed Repair of the Beta-Globin Gene in Human Hematopoietic Stem Cells By >10-Fold and Improves In Vivo Engraftment By >120-Fold

Kevin A. Goncalves, PhD, Megan D. Hoban, PhD, Sharon L. Hyzy, MS, Katia S. George, BA, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2019) 134 (Supplement_1): 4634.

https://doi.org/10.1182/blood-2019-131157

Published: 2019

Journal Articles

Mgta-456, an Aryl Hydrocarbon Receptor (AHR) Antagonist Based Expansion of CD34+ Hematopoietic Stem Cells (HSC), Permits Selection of Better HLA Matched Cord Blood Units (CBUs) and Promotes Faster Neutrophil Recovery and Uniform Engraftment with Potentially Less Acute Graft-Vs-Host Disease (GVHD)

Heather Stefanski, MD PhD, Claudio G Brunstein, MD PhD, David H. McKenna, MD, Darin Sumstad, MLS, Jeffrey S. Miller, MD, Bruce R. Blazar, MD, Todd E. DeFor, MS, Anthony E. Boitano, PhD, Michael P. Cooke, PhD, Glen D. Raffel, MD PhD, John C. Davis, Jr., MD MPH, MS, John E. Wagner, MD

Blood (2019) 134 (Supplement_1): 804.

https://doi.org/10.1182/blood-2019-122329

Published: 2019

Journal Articles

Targeting CD45 with an Amanitin Antibody-Drug Conjugate Effectively Depletes Human HSCs and Immune Cells for Transplant Conditioning

Blood (2018) 132 (Supplement 1): 4526.

https://doi.org/10.1182/blood-2018-99-117167

Published: 2018

Journal Articles

Antibody Drug Conjugates Targeted to CD45 or CD117 Enable Allogeneic Hematopoietic Stem Cell Transplantation in Animal Models

Rahul Palchaudhuri, PhD, Sharon L. Hyzy, MS, Jennifer L Proctor, BS, Hillary L Adams, MS, Bradley R Pearse, PhD, Ganapathy Sarma, PhD, Sharon Aslanian, MS, Geoff Gillard, PhD, Tahirih L Lamothe, Olga Burenkova, BS, Melissa L. Brooks, BS, Andrew D Gabros, Charlotte F McDonagh, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2018) 132 (Supplement 1): 3324.

https://doi.org/10.1182/blood-2018-99-119432

Published: 2018

Journal Articles

Preliminary Phase 2 Results Demonstrate Engraftment with Minimal Neutropenia with MGTA-456, a CD34 + Expanded Cord Blood (CB) Product in Patients Transplanted for Inherited Metabolic Disorders (IMD)

Paul Orchard, MD, Glen D. Raffel, MD, Carolyn H Condon, MPH, Catherine A Monaghan, Demetra Vernet, Suzanne Tracey Sheirr, Jennifer Braun, RN BSN, Ryan Shanley, M.S, Troy C Lund, MD MS, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD, John C Davis, MD MPH, John E. Wagner, MD

Blood (2018) 132 (Supplement 1): 3467.

https://doi.org/10.1182/blood-2018-99-115102

Published: 2018

Journal Articles

CD117-Amanitin Antibody Drug Conjugates Effectively Deplete Human and Non-Human Primate HSCs: Proof of Concept As a Targeted Strategy for Conditioning Patients for Bone Marrow Transplant

Bradley R Pearse, PhD, Sean M McDonough, MS, Jennifer L Proctor, BS, Rajiv Panwar, PhD, Ganapathy Sarma, PhD, Molly A McShea, MS, Lena Kien, BS, Junia Dushime, BS, Hillary L Adams, MS, Sharon L. Hyzy, MS, Melissa L. Brooks, BS, Rahul Palchaudhuri, PhD, Qing Li, PhD, Nathan M Kallen, BA, Pranoti Sawant, MS, Charlotte F McDonagh, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2018) 132 (Supplement 1): 3314.

https://doi.org/10.1182/blood-2018-99-114881

Published: 2018

Journal Articles

Single Doses of Antibody Drug Conjugates (ADCs) Targeted to CD117 or CD45 Have Potent In Vivo Anti-Leukemia Activity and Survival Benefit in Patient Derived AML Models

Blood (2018) 132 (Supplement 1): 3316.

https://doi.org/10.1182/blood-2018-99-112726

Published: 2018

Journal Articles

MGTA-456, a First-in-Class Cell Therapy Produced from a Single Cord Blood Unit, Enables a Reduced Intensity Conditioning Regimen and Enhances Speed and Level of Human Microglia Engraftment in the Brains of NSG Mice

Kevin A. Goncalves, PhD, Shuping Li, PhD, Melissa L. Brooks, BS, Sharon L. Hyzy, MS, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2018) 132 (Supplement 1): 115.

https://doi.org/10.1182/blood-2018-99-118258

Published: 2018

Journal Articles

MGTA-145 in Combination with Plerixafor Rapidly Mobilizes High Numbers of Hematopoietic Stem Cells and Graft-Versus-Host Disease Inhibiting Myeloid-Derived Suppressor Cells in Non-Human Primates

Kevin A. Goncalves, PhD, Patrick C. Falahee, PhD, Sharon L. Hyzy, MS, Shuping Li, PhD, Anthony E. Boitano, PhD, Dwight M. Morrow, PhD, Michael P. Cooke, PhD

Blood (2018) 132 (Supplement 1): 116.

https://doi.org/10.1182/blood-2018-99-117370

Published: 2018

Journal Articles

Mgta-456 Contains Large Numbers of Expanded Cord Blood (CB) CD34+CD90+ Hematopoietic Stem Cells (HSC) Which Confer All Engraftment Activity and Correlate with Accelerated Neutrophil Recovery after Myeloablative Conditioning in Patients with Hematologic Malignancy

Anthony E. Boitano, PhD, Michael P. Cooke, PhD, Kevin A. Goncalves, PhD, Darin Sumstad, MLS, John E. Wagner, MD

Blood (2018) 132 (Supplement 1): 2083.

https://doi.org/10.1182/blood-2018-99-114988

Published: 2018

Journal Articles

Non-Genotoxic Conditioning Efficiently Depletes Host Hematopoietic Stem Cells and Facilitates Robust Multi-Lineage Engraftment in a Mouse Model of Fanconi Anemia

Meera Srikanthan, MD, Kevin G. Haworth, PhD, Christina M. Ironside, BS, Jerry Chen, Adam J. Hartigan, PhD, Rahul Palchaudhuri, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD, Hans-Peter Kiem, MD PhD

Blood (2018) 132 (Supplement 1): 2041.

https://doi.org/10.1182/blood-2018-99-116851

Published: 2018

Journal Articles

Aryl Hydrocarbon Receptor Antagonists Expand Adult Hematopoietic Stem Cells from Mobilized Peripheral Blood and Bone Marrow and Increase the Dose of CRISPR/Cas9 Gene-Edited NSG-Repopulating Cells

Megan D. Hoban, PhD, Kevin A. Goncalves, PhD, Jennifer L. Proctor, BS, Hillary L. Adams, MS, Sharon L. Hyzy, MS, Katia S. George, BA, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2017) 130 (Supplement 1): 3341.

https://doi.org/10.1182/blood.V130.Suppl_1.3341.3341

Published: 2017

Journal Articles

The Combination of GROβ and AMD3100 Leads to Rapid and Robust Mobilization of Hematopoietic Stem Cells in Nonhuman Primates

Patrick C. Falahee, PhD, Kevin A. Goncalves, PhD, Sharon L. Hyzy, MS, Jennifer L. Proctor, BS, Jonathan Hoggatt, PhD, Dwight Morrow, PhD, Michael P. Cooke, PhD

Blood (2017) 130 (Supplement 1): 1920.

https://doi.org/10.1182/blood.V130.Suppl_1.1920.1920

Published: 2017

NARROW

Single Agent CD45-Targeted Antibody Drug Conjugate Enables Full Mismatch Allogeneic Hematopoietic Stem Cell Transplantation in a Murine HSCT Model

Reversing Clonal Hematopoiesis and Associated Atherosclerotic Disease By Targeted Antibody-Drug-Conjugate (ADC) Conditioning and Transplant

A Single Dose of a Novel Anti-Human CD117-Amanitin Antibody Drug Conjugate (ADC) Engineered for a Short Half-Life Provides Dual Conditioning and Anti-Leukemic Activity and Extends Survival Compared to Standard of Care in Multiple Preclinical Models of Acute Myeloid Leukemia (AML)

A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates

A Novel Targeted Approach to Achieve Immune System Reset: CD45-Targeted Antibody Drug Conjugates Enable Autologous HSCT, Ameliorate Disease in Autoimmune Models, Potently Kill Human Immune Cells from Normal Donors and MS Patients, and Achieve Immune Depletion in Non-Human Primates (NHP)

High Dose Hematopoietic Stem Cell Transplantation Leads to Rapid Hematopoietic and Microglia Recovery and Disease Correction in a Mouse Model of Hurler Syndrome

Rapid and Robust Mobilization of CD34+ HSCs without G-CSF Following Administration of Mgta-145 Alone or in Combination with Plerixafor

Ex Vivo Expansion with E478 Increases the Rate of CRISPR-Mediated Homology Directed Repair of the Beta-Globin Gene in Human Hematopoietic Stem Cells By >10-Fold and Improves In Vivo Engraftment By >120-Fold

Mgta-456, an Aryl Hydrocarbon Receptor (AHR) Antagonist Based Expansion of CD34+ Hematopoietic Stem Cells (HSC), Permits Selection of Better HLA Matched Cord Blood Units (CBUs) and Promotes Faster Neutrophil Recovery and Uniform Engraftment with Potentially Less Acute Graft-Vs-Host Disease (GVHD)

Targeting CD45 with an Amanitin Antibody-Drug Conjugate Effectively Depletes Human HSCs and Immune Cells for Transplant Conditioning

Antibody Drug Conjugates Targeted to CD45 or CD117 Enable Allogeneic Hematopoietic Stem Cell Transplantation in Animal Models

Preliminary Phase 2 Results Demonstrate Engraftment with Minimal Neutropenia with MGTA-456, a CD34 + Expanded Cord Blood (CB) Product in Patients Transplanted for Inherited Metabolic Disorders (IMD)

CD117-Amanitin Antibody Drug Conjugates Effectively Deplete Human and Non-Human Primate HSCs: Proof of Concept As a Targeted Strategy for Conditioning Patients for Bone Marrow Transplant

Single Doses of Antibody Drug Conjugates (ADCs) Targeted to CD117 or CD45 Have Potent In Vivo Anti-Leukemia Activity and Survival Benefit in Patient Derived AML Models

MGTA-456, a First-in-Class Cell Therapy Produced from a Single Cord Blood Unit, Enables a Reduced Intensity Conditioning Regimen and Enhances Speed and Level of Human Microglia Engraftment in the Brains of NSG Mice

MGTA-145 in Combination with Plerixafor Rapidly Mobilizes High Numbers of Hematopoietic Stem Cells and Graft-Versus-Host Disease Inhibiting Myeloid-Derived Suppressor Cells in Non-Human Primates

Mgta-456 Contains Large Numbers of Expanded Cord Blood (CB) CD34+CD90+ Hematopoietic Stem Cells (HSC) Which Confer All Engraftment Activity and Correlate with Accelerated Neutrophil Recovery after Myeloablative Conditioning in Patients with Hematologic Malignancy

Non-Genotoxic Conditioning Efficiently Depletes Host Hematopoietic Stem Cells and Facilitates Robust Multi-Lineage Engraftment in a Mouse Model of Fanconi Anemia

Aryl Hydrocarbon Receptor Antagonists Expand Adult Hematopoietic Stem Cells from Mobilized Peripheral Blood and Bone Marrow and Increase the Dose of CRISPR/Cas9 Gene-Edited NSG-Repopulating Cells

The Combination of GROβ and AMD3100 Leads to Rapid and Robust Mobilization of Hematopoietic Stem Cells in Nonhuman Primates

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