62nd ASH Annual Meeting and Exposition: 802. Chemical Biology and Experimental Therapeutics: Poster I

Oral and Poster Abstracts
Poster

Biological, Leukemia, AML, ALL, Anemias, antibodies, apoptosis, sickle cell disease, Non-Biological, Diseases, CLL, Animal models, cell division, CAR-Ts, Therapies, Combinations, cell regulation, thalassemia, Genetic Disorders, chemotherapy, Adverse Events, MDS, coagulant drugs, Biological Processes, Hemoglobinopathies, white blood cells, PNH, Technology and Procedures, immunotherapy, Cell Lineage, epigenetics, Xenograft models, Lymphoid Malignancies, Study Population, Myeloid Malignancies, Clinically relevant, genetic profiling, hematopoiesis, pharmacology, flow cytometry, molecular interactions, pathways, signal transduction

1547

Discovery of GT19077, a c-Myc/Max Protein-Protein Interaction (PPI) Small Molecule Inhibitor, for Targeting c-Myc-Driven Blood Cancers

Liandong Ma, MD¹^*, Youzhi Tong, PhD²^*, Zhaohui Yang, PhD²^*, Qianxiang Zhou, PhD²^*, Honghua Yan, PhD²^*, Ye Chen²^*, Jie Chen, PhD²^*, Honglei Bi²^*, Jie Pan²^*, Cui Wang²^*, Rou Xu, PhD²^*, Qi Sun, PhD³^* and Luhau Lai, PhD³^*

¹R&D, Kintor Pharmaceutical Limited, Suzhou, China
²Kintor Pharmaceutical Limited, Suzhou, China
³Peking University, Beijing, China

1548

Induction of Fetal Hemoglobin By FTX6058, a Novel Small Molecule Development Candidate

Peter Rahl^*, Ivan Efremov^*, Billy Stuart^*, Keqiang Xie^*, Mark Roth^*, Richard Barnes^*, Kingsley Appiah^*, David Peters^*, Qingyi Li^*, Steven Kazmirski^*, Paul Bruno^*, Kim Stickland^*, Lucienne Ronco^*, Diego Cadavid^*, Lorin Thompson^*, Owen Wallace^* and Christopher Moxham^*

Fulcrum Therapeutics, Cambridge, MA

1549

The Dual PI3Kδγ Inhibitor Duvelisib Potently Inhibits IL-6 Production and Cytokine Release Syndrome (CRS) While Maintaining CAR-T Function in Vitro and In Vivo

Parmeshwar N Amatya, PhD¹^*, Alun J Carter, PhD, BSc¹^*, Julie K Ritchey²^*, Jessica Niswonger³^*, Matthew L Cooper, PhD², Jonathan A Pachter, PhD⁴ and John F. DiPersio, MD⁵

¹Department of Internal Medicine, Division of Oncology, Washington University School of Medicine, Saint Louis, MO
²Division of Oncology, Department of Medicine, Washington University School of Medicine, Saint Louis, MO
³Division of Oncology, Washington University School of Medicine, Saint Louis, MO
⁴Verastem, Needham, MA
⁵Washington University School of Medicine, Saint Louis, MO

1550

An Optimized Crovalimab Dose and Regimen Reduced the Formation of Drug-Target-Drug Complexes in Patients with Paroxysmal Nocturnal Hemoglobinuria from the Phase I/II COMPOSER Trial

Jun-ichi Nishimura, MD, PhD¹, Antoine Soubret, PhD²^*, Simon Buatois, PharmD, PhD²^*, Jean-Eric Charoin²^*, Sasha Sreckovic, MD², Christoph Bucher^2,3^*, Jules Hernández-Sánchez, PhD²^*, Gregor Jordan⁴^*, Julia Ramos, MD, PhD^5,6, Noriko Arase, MD, PhD⁷^*, Masaki Hotta⁸^*, Yoshitaka Isaka⁹^*, Yoshikazu Ito, MD, PhD¹⁰, Yuzuru Kanakura, MD, PhD^1,11^*, Jin Seok Kim, MD, PhD¹²^*, Taroh Kinoshita, PhD¹³, Eiichi Morii¹⁴^*, Jens Panse, MD¹⁵, Regis Peffault De Latour, MD, PhD¹⁶^*, Alexander Röth, MD¹⁷, Hubert Schrezenmeier¹⁸, Simona Sica, MD, PhD^19,20^*, Hiroyuki Takamori, MD¹^*, Yasutaka Ueda¹^*, Sung-Soo Yoon, MD, PhD²¹, Ido Paz-Priel⁶^* and Alexandre Sostelly, PhD²^*

¹Department of Hematology and Oncology, Osaka University Graduate School of Medicine, Osaka, Japan
²F. Hoffmann-La Roche Ltd, Basel, Switzerland
³ANAVEON AG, Basel, Switzerland
⁴Roche Pharma Research and Early Development, Roche Innovation Center Munich, Munich, Germany
⁵Division of Hematology and Oncology, University of California, San Francisco, CA
⁶Genentech, Inc., South San Francisco, CA
⁷Department of Dermatology, Osaka University Graduate School of Medicine, Osaka, Japan
⁸Department of Medical Technology, Osaka University Hospital, Osaka, Japan
⁹Department of Nephrology, Osaka University Graduate School of Medicine, Osaka, Japan
¹⁰Department of Hematology, Tokyo Medical University, Tokyo, Japan
¹¹Department of Hematology, Sumitomo Hospital, Osaka, Japan
¹²Department of Internal Medicine, Yonsei University College of Medicine, Severance Hospital, Seoul, Korea, Republic of (South)
¹³Research Institute for Microbial Diseases, Osaka University, Osaka, Japan
¹⁴Department of Pathology, Osaka University Graduate School of Medicine, Osaka University, Osaka, Japan
¹⁵Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen, Germany
¹⁶CHU Paris GH St Louis Lariboisiere F Widal Hospital, Paris, France
¹⁷Department of Hematology, University Hospital Essen, Essen, Germany
¹⁸Institute of Clinical Transfusion Medicine and Immunogenetics, University of Ulm, Ulm, Germany
¹⁹Dipartimento di Diagnostica per Immagini, Radioterapia Oncologia ed Ematologia, Fondazione Policlinico A. Gemelli IRCSS, Rome, Italy
²⁰Sezione di Ematologia, Dipartimento di Scienze Radiologiche ed Ematologiche, Università Cattolica del Sacro Cuore, Rome, Italy
²¹Department of Internal Medicine, Seoul National University Hospital, Seoul National University College of Medicine, Seoul, Korea, Republic of (South)

1551

An Engineered RNA Platform to Neutralize DNMT1 Function and Control DNA Methylation for Myelodysplastic Syndrome

Carla Lucia Esposito¹^*, Ida Autiero^2,3^*, Mahmoud Adel Bassal, PhD^4,5^*, Sandomenico Annamaria³^*, Simone Ummarino^4,6^*, Marta Borchiellini^7,8^*, Menotti Ruvo^3,9^*, Silvia Catuogno¹^*, Alexander Ebralidze, PhD^4,6^*, Vittorio De Franciscis¹^* and Annalisa Di Ruscio, MD, PhD^7,10

¹Institute for Experimental Endocrinology and Oncology "Gaetano Salvatore", CNR, Naples, Italy
²Molecular Horizon, Bettona, Italy
³Institute of Biostructures and Bioimaging, CNR, Naples, Italy
⁴Harvard Stem Cell Institute, Harvard Medical School, Boston
⁵Cancer Science Institute of Singapore, National University of Singapore, Singapore, Singapore
⁶Harvard Medical School Initiative for RNA Medicine, Boston
⁷Department of Translational Medicine, University of Eastern Piedmont, Novara, Italy
⁸Department of Health Sciences, University of Eastern Piedmont, Novara, Italy
⁹Anbition srl, Naples, Italy
¹⁰Beth Israel Deaconess Medical Center, Boston, MA

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^*signifies non-member of ASH

802.P1.15 802. Chemical Biology and Experimental Therapeutics: Poster I