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Journal Articles

Proof of concept for a rapidly switchable universal CAR-T platform with UniCAR-T-CD123 in relapsed/refractory AML

Martin Wermke, Sabrina Kraus, Armin Ehninger, Ralf C. Bargou, Maria-Elisabeth Goebeler, Jan Moritz Middeke, Carla Kreissig, Malte von Bonin, Jan Koedam, Michael Pehl, Martin Bornhäuser, Hermann Einsele, Gerhard Ehninger, Marc Cartellieri

Blood (2021) 137 (22): 3145–3148.

https://doi.org/10.1182/blood.2020009759

Published: 2021

Journal Articles

Mitapivat (AG-348), an Oral PK-R Activator, in Adults with Non-Transfusion Dependent Thalassemia: A Phase 2, Open-Label, Multicenter Study in Progress

Kevin H. M. Kuo, MDMSc,FRCPC, D. Mark Layton, MB BS, Katrin Uhlig, MD MS, Megan Lynch, MSN, NP, Li Liu, PhD, Elliott P. Vichinsky, MD

Blood (2019) 134 (Supplement_1): 2249.

https://doi.org/10.1182/blood-2019-123113

Published: 2019

Journal Articles

Talazoparib Treatment Preferentially Depletes Cohesin-Mutant Clones in New In Vivo M odels of Cohesin-Mutant Myeloid Diseases

Mounica Vallurupalli, MD, Rebecca Gorelov, BS, Jacqueline S Garcia, MD, Elizabeth A Morgan, MD, Benjamin Ebert, MD, Zuzana Tothova, MD PhD

Blood (2019) 134 (Supplement_1): 560.

https://doi.org/10.1182/blood-2019-130308

Published: 2019

Journal Articles

Integrated Immune Signature Analyses Identifies Evolution of Distinct Immunoregulatory Cell Populations Which Control Alloreactivity after Allogeneic HSCT

James Aries, Sarah Charrot, Jennifer Ball, Matthew Mee, Symeon Theocharidis, Sofie Van Gassen, Sameena Iqbal, John G. Gribben, MD DSc, Jeff Davies, MBBS, PhD

Blood (2019) 134 (Supplement_1): 595.

https://doi.org/10.1182/blood-2019-124875

Published: 2019

Journal Articles

Pharmacological Inhibition of the RNA m 6 a Writer METTL3 As a Novel Therapeutic Strategy for Acute Myeloid Leukemia

Konstantinos Tzelepis, Etienne De Braekeleer, PhD MSc, Eliza Yankova, PhD, Justyna Rak, PhD, Demetrios Aspris, MSc, Ana Filipa Domingues, PhD, Richard Fosbeary, Alan Hendrick, PhD, Dan Leggate, PhD, Yaara Ofir-Rosenfeld, PhD, Alexandra Sapetschnig, PhD, Cristina Pina, MDDPhil, Mark Albertella, PhD, Wesley Blackaby, PhD, Oliver Rausch, PhD, George S. Vassiliou, FRCPath, MRCP, PhD, Tony Kouzarides, PhD

Blood (2019) 134 (Supplement_1): 403.

https://doi.org/10.1182/blood-2019-127962

Published: 2019

Journal Articles

Results from a First-in-Human, Proof-of-Concept Phase 1 Trial in Pretreated B-Cell Malignancies for Loxo-305, a Next-Generation, Highly Selective, Non-Covalent BTK Inhibitor

Anthony R. Mato, MDMSCE, Ian W. Flinn, MD PhD, John M. Pagel, MD PhD DSc, Jennifer R. Brown, MD PhD, Chan Y. Cheah, Catherine C. Coombs, MD, Manish R. Patel, MD, S. Michael Rothenberg, MD PhD, Donald E. Tsai, MD PhD, Nora C. Ku, MD, Michael L. Wang, MD

Blood (2019) 134 (Supplement_1): 501.

https://doi.org/10.1182/blood-2019-127509

Published: 2019

Journal Articles

Safety and Antithrombotic Efficacy of the Recombinant Protein C Activator AB002 (E-WE Thrombin) in End Stage Renal Disease Patients on Chronic Hemodialysis

Norah G Verbout, PhD, Christina U Lorentz, PhD, Brandon D Markway, PhD, Joseph J Shatzel, MD, Erik I Tucker, PhD, Andras Gruber, MD

Blood (2019) 134 (Supplement_1): 2433.

https://doi.org/10.1182/blood-2019-124999

Published: 2019

Journal Articles

Mechanistic Evaluation of Efficacy Using Biomarkers of the Oral, Small Molecule Factor D Inhibitor, Danicopan (ACH-4471), in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Antonio M. Risitano, MD PhD, Jong Wook Lee, MD PhD, Rosario Notaro, MD, Robert A. Brodsky, MD, Austin Kulasekararaj, MBBS MDMRCP FRCPath, Jaroslaw P. Maciejewski, MD PhD, Mingjun Huang, PhD, Michael Geffner, MD, Peter Browett, BMedSci, MBChB, FRACP, FRCPA

Blood (2019) 134 (Supplement_1): 2226.

https://doi.org/10.1182/blood-2019-124837

Published: 2019

Journal Articles

Nanoparticles Mimicking Platelets and Platelet Cloaking

Liangfang Zhang, PhD

Blood (2019) 134 (Supplement_1): SCI-38.

https://doi.org/10.1182/blood-2019-121082

Published: 2019

Journal Articles

Abscopal Effect of Radiotherapy and Nivolumab in Relapsed or Refractory Hodgkin Lymphoma (AERN): An International Multicenter Single-Arm Two-Stage Phase II GHSG Trial

Paul J Bröckelmann, MD, Annette Plütschow, Richard Greil, MD, Josée M Zijlstra, MD PhD, Timothy Illidge, MD PhD, Alexander Fosså, MD PhD, Julia Meissner, MD, Andreas Zimmermann, MD, Stephan Mathas, MD, Felicitas Thol, MD, Michael Fuchs, MD, Andreas Engert, MD, Christian Baues, MD

Blood (2019) 134 (Supplement_1): 1547.

https://doi.org/10.1182/blood-2019-124351

Published: 2019

Journal Articles

Characterization of Bypassing Kinases Inferred By Phosphoproteomics in a Midostaurin Resistant Myeloid Cell Line Model

Mahmoud Hallal, BSc, MSc, Sophie Braga-Lagache, MSc, Jovana Jankovic, Rémy Bruggmann, PhD, Ramanjaneyulu Allam, PhD MSc, BSc, Mario P. Tschan, PhD, Cédric Simillion, PhD, Manfred Heller, PhD, Nicolas Bonadies, MD

Blood (2019) 134 (Supplement_1): 2079.

https://doi.org/10.1182/blood-2019-122337

Published: 2019

Journal Articles

Automation Platform for CAR-T Manufacturing: The Benefits and the Clinical Outcomes

Min Wang, PhD, Fengtao You, PhD, Xingbing Wang, MD, Bingzong Li, MD PhD, Hanying Xu, MS, Yu Wang, MS, Guifang Pan, MS, Song Sun, MS, Huimin Meng, MD PhD, Yanmei Zhao, MS, Yu-Sheng Chen, PhD, Bozhen Zhang, MBA, Lin Yang, PhD

Blood (2019) 134 (Supplement_1): 1960.

https://doi.org/10.1182/blood-2019-123656

Published: 2019

Journal Articles

The PRIME Trial: PARP Inhibition in IDH Mutant Effectiveness Trial. a Phase II Study of Olaparib in Isocitrate Dehydrogenase (IDH) Mutant Relapsed/Refractory Acute Myeloid Leukemia and Myelodysplastic Syndrome

Namrata S Chandhok, MD, Wei Wei, MD PhD, Ranjit Bindra, MD PhD, Stephanie Halene, MD, Yu Shyr, Jing Li, PhD, Michael Berens, PhD, Chris Karlovich, PhD, S. Percy Ivy, MD, Thomas Prebet, MD PhD

Blood (2019) 134 (Supplement_1): 3909.

https://doi.org/10.1182/blood-2019-129168

Published: 2019

Journal Articles

Fatty Acid Binding Protein FABP5: A Novel Therapeutic Target in Acute Myeloid Leukemia

Metis Hasipek, PhD, Yihong Guan, PhD, Dale Grabowski, BS, Jaroslaw P. Maciejewski, MD PhD, Hetty E. Carraway, MD MBA, Babal K. Jha, PhD

Blood (2019) 134 (Supplement_1): 2553.

https://doi.org/10.1182/blood-2019-130475

Published: 2019

Journal Articles

A Novel Machine Learning Derived Genomics-Based Scoring System Is Highly Predictive of Outcome in Core Binding Factor Acute Myeloid Leukemia

Nikhil Patkar, MD, Chinmayee Kakirde, Anam Fatima Shaikh, Shrinidhi Nathany, MD, Gaurav Chatterjee, MD, Prashant Tembhare, MD, Dhanlaxmi Lalit Shetty, PhD, Anant Gokarn, MD, Sachin Punatar, MBBS,MDDM MDcal Oncology, Avinash Bonda, MD DM, Lingaraj Nayak, MDDM MDcal Oncology, Hasmukh Jain, MD DM, Navin Khattry, MD DM MDcal Oncology, Bhausaheb Bagal, MD DM, Manju Sengar, MD DM, Sumeet Gujral, MD, Papagudi Ganesan Subramanian, MD

Blood (2019) 134 (Supplement_1): 2710.

https://doi.org/10.1182/blood-2019-128500

Published: 2019

Journal Articles

Efficacy Proof of Concept for Allogeneic CD123 Targeting CAR T-Cells Against Primary Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): Efficient Control of Tumor Progression in PDX Model and Potential Loss of CD123 Expression in Relapsed Disease

Blood (2019) 134 (Supplement_1): 2659.

https://doi.org/10.1182/blood-2019-126869

Published: 2019

Journal Articles

CXCR4-Targeted Positron Emission Tomography Imaging of Central Nervous System B-Cell Lymphoma

Peter Herhaus, MD, Jana Lipkova, Felicitas Lammer, MD, Julia Slotta-Huspenina, MD, Benedikt Wiestler, MD, Tibor Vag, MD, Stefan Habringer, MD, Igor Yakushev, MD, Constantin Lapa, MD, Tobias Pukrop, MD, Dirk Hellwig, MD, Andreas Buck, MD, Martina Deckert, MD, Hans-Jürgen Wester, PhD, Florian Bassermann, MD PhD, Wolfgang Weber, MD, Markus Schwaiger, MD, Björn Menze, PhD, Ulrich Keller, MD

Blood (2019) 134 (Supplement_1): 2900.

https://doi.org/10.1182/blood-2019-126024

Published: 2019

Journal Articles

Patient Specific Comorbidities Impact Overall Survival in Myelofibrosis

Andrew Sochacki, MD, Cosmin Adrian Bejan, PhD, Shilin Zhao, PhD, Travis Spaulding, MD, Shannon Stockton, MD, Alexander Silver, Dixon Dorand, MD PhD, Siwei Zhang, PhD, Thomas Stricker, MD PhD, Yaomin Xu, PhD, Michael R. Savona, MD

Blood (2019) 134 (Supplement_1): 2959.

https://doi.org/10.1182/blood-2019-121513

Published: 2019

Journal Articles

Mutational Type and Configuration of an Individual Gene May Differentially Impact the Clinical and Phenotypic Features

Yasunobu Nagata, MD PhD, Hideki Makishima, MD PhD, Cassandra M Kerr, MS, Bhumika J. Patel, MD, Hassan Awada, Vera Adema, PhD, Valeria Visconte, PhD, Sudipto Mukherjee, MD PhD MPH, Thomas Laframboise, PhD, Aziz Nazha, MD, Tomas Radivoyevitch, PhD, Mikkael A. Sekeres, MD MS, Seishi Ogawa, MD PhD, Jaroslaw P. Maciejewski, MD PhD

Blood (2019) 134 (Supplement_1): 2992.

https://doi.org/10.1182/blood-2019-128893

Published: 2019

Journal Articles

Liver-Directed AAV Gene Therapy for Gaucher Disease

Carlos J Miranda, PhD, Miriam Canavese, PhD, Elisa Chisari, MS, Jalpa Pandya, MPhil, Clement Cocita, PhD, Maria Portillo, MRes, Jenny McIntosh, PhD, Azadeh Kia, PhD, Jonathan H. Foley, PhD, Allison Dane, PhD, Jey M Jeyakumar, PhD, Rose Sheridan, PhD, Romuald Corbau, Amit C. Nathwani, MD PhD

Blood (2019) 134 (Supplement_1): 3354.

https://doi.org/10.1182/blood-2019-124280

Published: 2019

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Proof of concept for a rapidly switchable universal CAR-T platform with UniCAR-T-CD123 in relapsed/refractory AML

Mitapivat (AG-348), an Oral PK-R Activator, in Adults with Non-Transfusion Dependent Thalassemia: A Phase 2, Open-Label, Multicenter Study in Progress

Talazoparib Treatment Preferentially Depletes Cohesin-Mutant Clones in New In Vivo M odels of Cohesin-Mutant Myeloid Diseases

Integrated Immune Signature Analyses Identifies Evolution of Distinct Immunoregulatory Cell Populations Which Control Alloreactivity after Allogeneic HSCT

Pharmacological Inhibition of the RNA m 6 a Writer METTL3 As a Novel Therapeutic Strategy for Acute Myeloid Leukemia

Results from a First-in-Human, Proof-of-Concept Phase 1 Trial in Pretreated B-Cell Malignancies for Loxo-305, a Next-Generation, Highly Selective, Non-Covalent BTK Inhibitor

Safety and Antithrombotic Efficacy of the Recombinant Protein C Activator AB002 (E-WE Thrombin) in End Stage Renal Disease Patients on Chronic Hemodialysis

Mechanistic Evaluation of Efficacy Using Biomarkers of the Oral, Small Molecule Factor D Inhibitor, Danicopan (ACH-4471), in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Nanoparticles Mimicking Platelets and Platelet Cloaking

Abscopal Effect of Radiotherapy and Nivolumab in Relapsed or Refractory Hodgkin Lymphoma (AERN): An International Multicenter Single-Arm Two-Stage Phase II GHSG Trial

Characterization of Bypassing Kinases Inferred By Phosphoproteomics in a Midostaurin Resistant Myeloid Cell Line Model

Automation Platform for CAR-T Manufacturing: The Benefits and the Clinical Outcomes

The PRIME Trial: PARP Inhibition in IDH Mutant Effectiveness Trial. a Phase II Study of Olaparib in Isocitrate Dehydrogenase (IDH) Mutant Relapsed/Refractory Acute Myeloid Leukemia and Myelodysplastic Syndrome

Fatty Acid Binding Protein FABP5: A Novel Therapeutic Target in Acute Myeloid Leukemia

A Novel Machine Learning Derived Genomics-Based Scoring System Is Highly Predictive of Outcome in Core Binding Factor Acute Myeloid Leukemia

Efficacy Proof of Concept for Allogeneic CD123 Targeting CAR T-Cells Against Primary Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): Efficient Control of Tumor Progression in PDX Model and Potential Loss of CD123 Expression in Relapsed Disease

CXCR4-Targeted Positron Emission Tomography Imaging of Central Nervous System B-Cell Lymphoma

Patient Specific Comorbidities Impact Overall Survival in Myelofibrosis

Mutational Type and Configuration of an Individual Gene May Differentially Impact the Clinical and Phenotypic Features

Liver-Directed AAV Gene Therapy for Gaucher Disease

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