65th ASH Annual Meeting & Exposition: 802. Chemical Biology and Experimental Therapeutics: Novel Targets in Blood Disorders

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

Marriott Grand Ballroom 8-9 (Marriott Marquis San Diego Marina)

Moderators:

Vidyasagar Koduri, MD, PhD, Dana-Farber Cancer Institute and Fabiana Perna, MD, PhD, Moffitt Cancer Center

Disclosures:

No relevant conflicts of interest to declare.

9:30 AM

Small-Molecule U2 Auxiliary Factor Homology Motif (UHM) Domain Inhibitors Cause Splicing Pattern Changes in U2AF1 Mutant Leukemia Cells and Induce Sub-G1 Cell Cycle Arrest

Chao-Yie Yang, PhD¹, Xinrui Yuan, PhD²^*, Mona Kazemi Sabzvar²^*, Arda Durmaz, PhD³^*, Krishnapriya Chinnaswamy⁴^*, Jeanne Stuckey, PhD⁵^*, Seth J Corey, MD⁶, Jaroslaw P. Maciejewski, M.D., Ph.D., FACP⁷ and Valeria Visconte, PhD⁸

¹University of Tennessee Health Science Center, Department of Pharmaceutical Sciences, Memphis, TN
²University of Tennessee Health Science Center, Memphis, TN
³Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, CLEVELAND, OH
⁴University of Michigan, Ann Arbor, MI
⁵Life Science Institute, Univerisity of Michigan, Ann Arbor, MI
⁶Departments of Pediatrics and Cancer Biology, Cleveland Clinic, Cleveland, OH
⁷Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
⁸Taussig Cancer Institute, Cleveland, OH

9:45 AM

116

Functional Precision Medicine Vs Genomics Vs Clinical Experience: Feasibility Results from the Multicentric, Prospective, Randomized Controlled Exalt-2 Trial

Lukas Kazianka¹^*, Tea Pemovska¹^*, Johannes Rohrbeck, MD²^*, Christoph Kornauth, MD²^*, Alexander Pichler¹^*, Christiane Agreiter, MSc¹^*, Simone Lubowitzki, PhD¹^*, Katharina Prochazka, MD, PhD³^*, Peter Neumeister, MD³^*, Clemens Schmitt, MD⁴, Richard Greil, MD⁵, Wolfgang Willenbacher, MD PhD⁶, Dominik Wolf, MD⁶^*, Ulrich Jaeger, MD¹^*, Ingrid Simonitsch-Klupp, MD²^* and Philipp Bernhard Staber, MD, PhD¹

¹Department of Medicine I, Division of Hematology & Hemostaseology, Medical University of Vienna, Vienna, Austria
²Department of Pathology, Medical University of Vienna, Vienna, Austria
³Medical University of Graz, Graz, Austria
⁴Kepler Universitätsklinik Linz, Linz, Austria
⁵Paracelsus Medical University, Department of Internal Medicine III with Hematology, Medical Oncology, Hemostaseology, Infectiology and Rheumatology, Oncologic Center, Salzburg Cancer Research Institute - Laboratory for Immunological and Molecular Cancer Research (SCRI-LIMCR), Salzburg, Austria
⁶Department of Medicine V, Medical University of Innsbruck, Innsbruck, Austria

10:00 AM

117

Discovery of BCL-XL Heterobifunctional Degrader with Potentially Improved Therapeutic Window and Minimal Platelet Toxicity for Hematological Malignancies

Yang Xie¹^*, Weiqiang Xing²^*, Waiting Lai²^*, Meixin Tao²^*, Liuge Gu²^*, Min Cao²^*, Jianxiong Diao³^*, Xinyu Bai³^*, Mingchen Chen³^*, Ying Lei²^* and Taylor B. Guo²^*

¹neoX Biotech, Boston, MA
²neoX Biotech, Shanghai, China
³neoX Biotech, Beijing, China

10:15 AM

118

The Atypical Protein Kinase WNK1 Controls Leukemia Progression in TAL/LMO T-Cell Acute Lymphoblastic Leukemia

Anna Montanaro¹^*, Anna D'Antuono¹^*, Lucas Moron Dalla Tor¹^*, Pietro Andrei¹^*, Filippo Cavazzini¹^*, Elisa Cerretani²^*, Federica Vento²^*, Elisa Simoncini¹^*, Andrea Gherli¹^*, Raffaella Zamponi¹^*, Bruno Lorusso¹^*, Giannalisa Todaro³^*, Mariateresa Giaimo¹^*, Roberta La Starza⁴^*, Cristina Mecucci⁴, Gabriella Sammarelli³^*, Matteo Marchesini^1,5^*, Luca Pagliaro¹^* and Giovanni Roti^1,3

¹Department of Medicine and Surgery, University of Parma, Parma, Italy
²Department of Medical Science, University of Ferrara, Ferrara, Italy
³Hematology and BMT Unit, Azienda Ospedaliera-Universitaria di Parma, Parma, Italy
⁴Department of Medicine, Hematology and Clinical Immunology, University of Perugia, Perugia, Italy
⁵Istituto Romagnolo per lo Studio dei Tumori "Dino Amadori", Meldola, Italy

10:30 AM

119

Transcription Factor Redistributors Pharmacologically Actuate Non-Canonical Gene Networks to Drive AML Differentiation

Samuel Taylor, PhD, BSc¹, Jacob Stauber¹^*, Oliver Bohorquez¹^*, Emily Schwenger¹^*, Sriram Sundaravel, PhD, MS¹^*, Abdelbasset Farahat, PhD²^*, Boris Bartholdy, PhD¹^*, David Boykin, PhD³^*, Gregory Poon, PhD, BPharm³^* and Ulrich Steidl, MD, PhD⁴

¹Albert Einstein College of Medicine, Bronx, NY
²Northstate University, Elk Grove, CA
³Georgia State University, Atlanta, GA
⁴Blood Cancer Institute, Department of Oncology, Albert Einstein College of Medicine, Bronx, NY

10:45 AM

120

Clonal Targeting of DNA Damage Response Pathways Eradicates Myeloproliferative Neoplasms

Monika Toma, PhD¹^*, Margaret Nieborowska-Skorska, MD, PhD¹^*, Adam Karami¹^*, Monika Pepek²^*, Tomasz Stoklosa, MD, PhD² and Tomasz Skorski, MD, PhD¹

¹Fels Cancer Institute for Personalized Medicine, Temple University School of Medicine, Philadelphia, PA
²Department of Tumor Biology and Genetics, Medical University of Warsaw, Warsaw, Poland

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^*signifies non-member of ASH

802.O1.6 802. Chemical Biology and Experimental Therapeutics: Novel Targets in Blood Disorders