66th ASH Annual Meeting: 802. Chemical Biology and Experimental Therapeutics: Novel Therapeutic Strategies for Hematologic Disorders: From Mechanistic to Preclinical Studies

Symposia: Chemical Biology and Experimental Therapeutics Oral and Poster Abstracts
Oral

Research, Fundamental Science, Sickle Cell Disease, Lymphoid Leukemias, ALL, Acute Myeloid Malignancies, AML, Combination therapy, MDS, Iron Deficiency, Translational Research, Plasma Cell Disorders, Genomics, Hemoglobinopathies, Chronic Myeloid Malignancies, Diseases, Immune mechanism, Treatment Considerations, Lymphoid Malignancies, Metabolism, Myeloid Malignancies, Biological Processes, Emerging technologies, Molecular biology, Technology and Procedures, Multi-systemic interactions, Pathogenesis

Room 33 (San Diego Convention Center)

Moderators:

Giulia Biancon, PhD, ARRAY(0x104e999c) and Valeria Visconte, PhD, Taussig Cancer Institute

Disclosures:

No relevant conflicts of interest to declare.

12:00 PM

151

Novel Small Molecule Inhibitor Against IGF2BP3 with Potent Anti-Leukemic Activity

Amit K. Jaiswal, PhD¹, Michelle L. Thaxton¹^*, Jacob Sorrentino²^*, Georgia Scherer²^*, Gunjan Sharma³^*, Tasha L. Lin⁴, Tiffany M. Tran⁵^*, Alexander J. Ritter⁶^*, Robert Damoiseaux⁷^*, Jeremy R. Sanford⁸^*, Neil Garg⁹^* and Dinesh S. Rao, MD, PhD¹

¹Department of Pathology and Laboratory Medicine, University of California, Los Angeles, Los Angeles, CA
²University of California, Los Angeles
³University of California, Los Angeles, Los Angeles, CA
⁴Department of Medicine, Division of Hematology/Oncology, University of California, Los Angeles, Los Angeles, CA
⁵Department of Medicine, University of California, Los Angeles, Los Angeles, CA
⁶Department of Molecular, Cellular and Developmental Biology and Center for Molecular Biology of RNA, University of California, Santa Cruz, Santa Cruz, CA
⁷MSSR, University of California, Los Angeles
⁸Center for Biomolecular Science & Engineering, University of California, Santa Cruz, Santa Cruz, CA
⁹University of California Los Angeles, Los Angeles

12:15 PM

152

The Transcriptional Activators p300/Crebbp Are Context-Dependent Functional Repressors of Interferon Genes, Offering Therapeutic Promise in AML

Markus Meyerhöfer^1,2^*, Viral Shah^2,3^*, Aarón Gallego-Crespo, M.Sc.^1,4^*, Malte Andreas Behrendt, M.Sc.^1,5^*, Yawen Zhou, PhD^4,6^*, Maria Saura-Pañella^4,6^*, Björn Häupl, PhD^7,8^*, Thomas Kindler, MD^1,4^*, Matthias Theobald, MD^5,6, Falk Butter, PhD^9,10^*, George S Vassiliou, MD, PhD¹¹, Michael Kühn, MD^1,5^*, Brian J P Huntly, MD¹¹^*, Thomas Oellerich, MD^4,8^* and Daniel Sasca, MD^4,6^*

¹Department of Hematology and Oncology, University Medical Center of the Johannes Gutenberg University, Mainz, Germany
²German Cancer Consortium (DKTK), Frankfurt/Mainz, Germany, Mainz, Germany
³University Cancer Center, University Medical Center of the Johannes Gutenberg University, Mainz, Germany
⁴German Cancer Consortium (DKTK), Mainz / Frankfurt am Main, Germany
⁵German Cancer Consortium (DKTK), Frankfurt/Mainz, Mainz, Germany
⁶Department of Hematology and Oncology, Medical Center of the Johannes Gutenberg University, Mainz, Germany
⁷German Cancer Research Center (DKFZ), Heidelberg, Germany
⁸Department of Hematology and Oncology, Johann Wolfgang Goethe University, Frankfurt am Main, Germany
⁹Institute of Molecular Biology, Mainz, Germany
¹⁰Institute of Molecular Virology and Cell Biology, Friedrich-Loeffler-Institut,Greifswald, Greifswald, Germany
¹¹Department of Haematology and Cambridge Stem Cell Institute, University of Cambridge, Cambridge, United Kingdom

12:30 PM

153

Pharmacological Targeting of the Heme-Regulated Inhibitor Kinase Overcomes Ineffective Erythropoiesis in Patients with Myelodysplastic Syndromes with Ringed Sideroblasts and Sickle Cell Disease

Vera Adema, PhD¹^*, Giulia Faccincani, MS¹^*, Amy Cooper, PhD²^*, Juan Jose Rodriguez-Sevilla, MD¹^*, Jieqing Chen²^*, Natthakan Thongon, PhD¹, Teresa Perry²^*, Jennifer Tran²^*, Fernando Alvarez²^*, Zhijun Kang²^*, Vijayan Ramaswamy²^*, Roopa Thapar²^*, Troy Johnson²^*, Guillermo Garcia-Manero, MD¹, Faika Mseeh²^*, David Kummer²^*, Philip Jones²^*, Timothy Heffernan, PhD²^* and Simona Colla, PhD¹

¹Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX
²Institute for Applied Cancer Science (IACS), The University of Texas MD Anderson Cancer Center, Houston, TX

12:45 PM

154

Large-Scale In Vivo Zebrafish Drug Screen Uncovers Novel Insights into Leukemia Stem Cell Biology

Majd Al-Hamaly¹^*, Yelena Chernyavskaya¹^*, Meghan Haney, MD²^*, Jeffery Jolly³^*, Ronald Bruntz³^* and Jessica Blackburn³^*

¹University of Kentucky, Lexington, KY
²Cincinnati Children's Hospital, Cincinnati, OH
³University of Kentucky, Lexington

1:00 PM

155

Pharmacological Targeting of IRF4 As a Therapeutic Strategy for Multiple Myeloma

Michael P. Agius, PhD¹^*, Laura Hevenor²^*, N Connor Payne, PhD^3,4,5^*, Qi Liu, PhD⁶^*, Hyuk-Soo Seo, PhD⁷^*, Romanos Sklavenitis-Pistofidis, MD^2,8,9, Daniel Heilpern-Mallory²^*, Puspalata Bashyal¹⁰^*, Michelle P. Aranha, PhD^2,8,9^*, Elizabeth D. Lightbody, PhD^2,8,9, Ralph Mazitschek, PhD^4,5^*, Sirano Dhe-Paganon, PhD¹¹^*, Jun Qi, PhD⁷^* and Irene Ghobrial, MD^2,8,9

¹Dana Farber Cancer Institute, Boston, MA
²Dana-Farber Cancer Institute, Boston, MA
³Harvard T.H. Chan School of Public Health, Boston
⁴Center for Systems Biology, Massachusetts General Hospital, Boston, MA
⁵Department of Chemistry and Chemical Biology, Harvard Medical School/MGH, Boston, MA
⁶Department of Cancer Biology, Dana Farber Cancer Institute, Boston
⁷Department of Cancer Biology, Dana Farber Cancer Institute, Boston, MA
⁸Harvard Medical School, Boston, MA
⁹Broad Institute of MIT and Harvard, Cambridge, MA
¹⁰Dana Farber Cancer Institute, Boston
¹¹Department of Cancer Biology, Dana-Farber Cancer Institute, Boston, MA

1:15 PM

156

Potent in Vitro and In Vivo Efficacy of Hdz-C123A, a GSPT1 Degrader-Antibody Conjugate Targeting CD123 in Acute Myeloid Leukemia

Xiaowu Dong, PhD^1,2^*, Yu Guo³^*, Jingyu Zhang³^*, Zheyuan Shen³^*, Shuang Wu³^*, Shuangshuang Geng³^*, Xinna Ma¹^*, Miao Hu, MD, phd¹ and Xinglu Zhou¹^*

¹HealZen Therapeutics Co., Ltd, Hangzhou, China
²Hangzhou Institute of Innovative Medicine, Institute of Drug Discovery and Design, College of Pharmaceutical Sciences, Zhejiang University, Hangzhou, China
³Hangzhou Institute of Innovative Medicine, Zhejiang University, Hangzhou, China

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^*signifies non-member of ASH

802.O1.6 802. Chemical Biology and Experimental Therapeutics: Novel Therapeutic Strategies for Hematologic Disorders: From Mechanistic to Preclinical Studies