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Journal Articles

BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice

Blood (2020) 135 (17): 1484–1496.

https://doi.org/10.1182/blood.2019001292

Published: 2020

Includes: Supplemental data

Journal Articles

Preclinical Evaluation for Engraftment of Gene-Edited CD34+ Cells with a Sickle Cell Disease Mutation in a Rhesus Transplantation Model

Naoya Uchida, MD, Linhong Li, PhD, Tina Nassehi, Morgan Yapundich, Jackson Gamer, Claire Drysdale, Juan J. Haro-Mora, PhD, Selami Demirci, PhD, Aylin Bonifacino, MS, Allen Krouse, Nathaniel S. Linde, Cornell Allen, MS, Suk See De Ravin, MD, PhD, Robert E. Donahue, VMD, Harry L. Malech, MD, Madhusudan Peshwa, PhD, John F. Tisdale, MD

Blood (2019) 134 (Supplement_1): 609.

https://doi.org/10.1182/blood-2019-122727

Published: 2019

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

Truncated Erythropoietin Receptors Confer an In Vivo Selective Advantage in Gene-Modified Erythroid Cells Expressing Fetal Hemoglobin Due to BCL11A Interference

Naoya Uchida, MD, Claire Drysdale, Morgan Yapundich, Jackson Gamer, Tina Nassehi, Matthew Wielgosz, Francesca Ferrara, PhD, Juan J. Haro-Mora, PhD, Selami Demirci, PhD, Aylin Bonifacino, MS, Allen Krouse, Nathaniel S. Linde, Robert E. Donahue, VMD, Byoung Y. Ryu, PhD, John F. Tisdale, MD

Blood (2019) 134 (Supplement_1): 2063.

https://doi.org/10.1182/blood-2019-122770

Published: 2019

Journal Articles

Activation of the Receptor Tyrosine Kinase (RET) By GDNF/GFRα1 Improves Cord Blood-Derived HSC in Vitro expansion and In Vivo engraftment

William Grey, PhD MSc, Rakhee Chauhan, PhD, Manuel Garcia-Albornoz, Hector Huerga Encabo, Neil McDonald, PhD, Dominique Bonnet, PhD

Blood (2019) 134 (Supplement_1): 1924.

https://doi.org/10.1182/blood-2019-121331

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

Next-Generation FVIII Mimetic Shows Superior Effect in a FIX- and Fx-Humanized Mouse Model In Vivo

Carsten Dan Ley, PhD, Thomas Lindebo Holm, PhD, Daniel Elenius, PhD, Heidi Lindgreen Holmberg, PhD, Jais Rose Bjelke, PhD, Mette Loftager, PhD, Mette B Hermit, PhD, Ida Hilden, Stine L Kjellev, PhDDVM

Blood (2019) 134 (Supplement_1): 3631.

https://doi.org/10.1182/blood-2019-128159

Published: 2019

Journal Articles

Similar Hemostatic Responses to Hypovolemia in Hemorrhage and Lbnp Reveals a Hyperfibrinolytic Subset of Baboons

Maryanne Herzig, PhD, Morten Zaar, PhD, Chriselda Fedyk, Bijaya Kumar Parida, PhD, Carmen Hinojosa-Laborde, PhD, Robert E Shade, PhD, Heather F. Pidcoke, MD PhD, Victor A Convertion, PhD, Andrew P. Cap, MD PhD

Blood (2019) 134 (Supplement_1): 3633.

https://doi.org/10.1182/blood-2019-131792

Published: 2019

Journal Articles

High Avidity IgM-Based CD20xCD3 Bispecific Antibody (IGM-2323) for Enhanced T-Cell Dependent Killing with Minimal Cytokine Release

Ramesh Baliga, PhD, Keyu Li, PhD, Marigold Manlusoc, Paul Hinton, Dean Ng, PhD, Madeline Tran, Bing Shan, PhD, Hai Lu, Avneesh Saini, PhD, Sachi Rahman, Yuan Cao, PhD, Chitra Saraiya, Marvin Peterson, PhD, Steve Carroll, PhD, Daniel Chen, MD PhD, Bruce Keyt, PhD

Blood (2019) 134 (Supplement_1): 1574.

https://doi.org/10.1182/blood-2019-131650

Published: 2019

Journal Articles

IMPACT-TB: A Phase II Trial Assessing the Capacity of Low Dose Imatinib to Induce Myelopoiesis and Enhance Host Anti-Microbial Immunity Against Tuberculosis. Imatinib Mesylate per Oral As a Clinical Therapeutic for TB

Cynthia R. Giver, PhD, Pamela A Shaw, PhD, Helen Fletcher, PhD, Deepak Kaushal, PhD, Garcia Pamela, Deborah Omoyege, Gregory Bisson, MD, Tawanda Gumbo, MD, Robert Wallis, MD, Edmund K. Waller, PhDMD, Daniel Kalman, PhD

Blood (2019) 134 (Supplement_1): 1050.

https://doi.org/10.1182/blood-2019-130275

Published: 2019

Journal Articles

Purification Platelets from Mouse Blood with Sickle Cell Disease Using Iohexol Gradient Medium

Md Nasimuzzaman, PhD

Blood (2019) 134 (Supplement_1): 4888.

https://doi.org/10.1182/blood-2019-128459

Published: 2019

Journal Articles

Non-Genotoxic Anti-CD117 Antibody Conditioning Results in Successful Hematopoietic Stem Cell Engraftment in Patients with Severe Combined Immunodeficiency

Rajni Agarwal, MD, Christopher C. Dvorak, MD, Hye-Sook Kwon, PhD, Janel R Long-Boyle, PharmD, PhD, Susan S Prohaska, PhD MS, Janice W Brown, MD, Anne Le, Alyssa Guttman-Klein, Irving L Weissman, MD, Morton J Cowan, MD, Aaron C. Logan, MD PhD, Kenneth I. Weinberg, MD, Robertson Parkman, MD, Maria-Grazia Roncarolo, MD, Judith A Shizuru, MD PhD

Blood (2019) 134 (Supplement_1): 800.

https://doi.org/10.1182/blood-2019-126239

Published: 2019

Journal Articles

USP Potency Adjusted Bovine Mucosal Heparins (BMH) Are Comparable to Porcine Mucosal Heparin (PMH) at Equivalent Levels

Ahmed Kouta, Walter Jeske, PhD, Rick Duff, Debra Hoppensteadt, PhD, Omer Iqbal, MD, Jawed Fareed, PhD

Blood (2019) 134 (Supplement_1): 165.

https://doi.org/10.1182/blood-2019-131943

Published: 2019

Journal Articles

An All Antibody Approach for Conditioning Bone Marrow for Hematopoietic Stem Cell Transplantation with Anti-cKIT and Anti-CD47 in Non-Human Primates

Kristopher D Marjon, PhD, James Y Chen, MD PhD, Jiaqi Duan, MS, Timothy S Choi, BS, Kavitha Sompalli, MS, Dongdong Feng, PhD, Ofelia Mata, Sharline Chen, B.S., Leslie Kean, MD PhD, Mwe Mwe Chao, MD, Mark Chao, MD PhD, Chris H Takimoto, MD PhD, Balaji Agoram, PhD, Cynthia E. Dunbar, MD, Ravi Majeti, MD PhD, Irving L Weissman, MD, Jie Liu, PhD MD, Jens-Peter Volkmer, MD

Blood (2019) 134 (Supplement_1): 4428.

https://doi.org/10.1182/blood-2019-131490

Published: 2019

Journal Articles

A Phase I/II Study of NMS-03592088, a FLT3, KIT and CSF1R Inhibitor, in Patients with Relapsed or Refractory AML or CMML

Blood (2019) 134 (Supplement_1): 3855.

https://doi.org/10.1182/blood-2019-121954

Published: 2019

Journal Articles

Preclinical Development of a Bcma Targeting Antibody-Drug Conjugate with Novel Payload for Multiple Myeloma Therapy

Andrew Hau, PhD, Tong Zhu, PhD, Rengang Wang, PhD, Megan Lau, BS, Lingna Li, PhD, Xiaoqing Li, PhD, Yingqing Sun, PhD, Ernest Kovacs, PhD, Alisher Khasanov, PhD, Dalun Deng, PhD, Zheng Yan, PhD, Robert D. Knight, MD, Gunnar F. Kaufmann, PhD, Henry Ji, PhD, Hui Li, PhD, Hong Zhang, PhD

Blood (2019) 134 (Supplement_1): 5623.

https://doi.org/10.1182/blood-2019-132080

Published: 2019

Journal Articles

From Graft-Versus-Host Disease to Lymphoma Pathogenesis: Emerging Roles for Stromal Notch Ligands in Hematology

Ivan Maillard, MD PhD

Blood (2019) 134 (Supplement_1): SCI-47.

https://doi.org/10.1182/blood-2019-121067

Published: 2019

Journal Articles

Fully Closed, Large-Scale, and Clinical Grade Cell Sorting of Hematopoietic Stem Cell (HSC)-Enriched CD90 + Cells for Transplantation and Gene Therapy

Stefan Radtke, PhD, Margaret Cui, Anai M Perez, Yan-Yi Chan, Stefanie Schmuck, BS, Andrew J Berger, PhD, Thomas Eunson, BSc, Hans-Peter Kiem, MD PhD

Blood (2019) 134 (Supplement_1): 3246.

https://doi.org/10.1182/blood-2019-125969

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

Seroprevalence of Pre-Existing Nabs Against AAV1, 2, 5, 6 and 8 in South African Hemophilia B Patient Population

Anna Majowicz, PhD, Nolukholo Ncete, MBBCH, Floris van Waes, MSc, Nikki Timmer, BSc, Sander J van Deventer, MD PhD, Johnny N Mahlangu, MBBCH, MMed, FCPath, Valerie Ferreira, PhD

Blood (2019) 134 (Supplement_1): 3353.

https://doi.org/10.1182/blood-2019-128217

Published: 2019

NARROW

BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice

Preclinical Evaluation for Engraftment of Gene-Edited CD34+ Cells with a Sickle Cell Disease Mutation in a Rhesus Transplantation Model

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

Truncated Erythropoietin Receptors Confer an In Vivo Selective Advantage in Gene-Modified Erythroid Cells Expressing Fetal Hemoglobin Due to BCL11A Interference

Activation of the Receptor Tyrosine Kinase (RET) By GDNF/GFRα1 Improves Cord Blood-Derived HSC in Vitro expansion and In Vivo engraftment

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

Next-Generation FVIII Mimetic Shows Superior Effect in a FIX- and Fx-Humanized Mouse Model In Vivo

Similar Hemostatic Responses to Hypovolemia in Hemorrhage and Lbnp Reveals a Hyperfibrinolytic Subset of Baboons

High Avidity IgM-Based CD20xCD3 Bispecific Antibody (IGM-2323) for Enhanced T-Cell Dependent Killing with Minimal Cytokine Release

IMPACT-TB: A Phase II Trial Assessing the Capacity of Low Dose Imatinib to Induce Myelopoiesis and Enhance Host Anti-Microbial Immunity Against Tuberculosis. Imatinib Mesylate per Oral As a Clinical Therapeutic for TB

Purification Platelets from Mouse Blood with Sickle Cell Disease Using Iohexol Gradient Medium

Non-Genotoxic Anti-CD117 Antibody Conditioning Results in Successful Hematopoietic Stem Cell Engraftment in Patients with Severe Combined Immunodeficiency

USP Potency Adjusted Bovine Mucosal Heparins (BMH) Are Comparable to Porcine Mucosal Heparin (PMH) at Equivalent Levels

An All Antibody Approach for Conditioning Bone Marrow for Hematopoietic Stem Cell Transplantation with Anti-cKIT and Anti-CD47 in Non-Human Primates

A Phase I/II Study of NMS-03592088, a FLT3, KIT and CSF1R Inhibitor, in Patients with Relapsed or Refractory AML or CMML

Preclinical Development of a Bcma Targeting Antibody-Drug Conjugate with Novel Payload for Multiple Myeloma Therapy

From Graft-Versus-Host Disease to Lymphoma Pathogenesis: Emerging Roles for Stromal Notch Ligands in Hematology

Fully Closed, Large-Scale, and Clinical Grade Cell Sorting of Hematopoietic Stem Cell (HSC)-Enriched CD90 + Cells for Transplantation and Gene Therapy

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)

Seroprevalence of Pre-Existing Nabs Against AAV1, 2, 5, 6 and 8 in South African Hemophilia B Patient Population

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American Society of Hematology

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American Society of Hematology

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