66th ASH Annual Meeting: 113. Sickle Cell Disease, Sickle Cell Trait, and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Identification of New Molecular Targets to Modulate Sickle Cell Disease

Symposia: Sickle Cell Disease, Sickle Cell Trait, and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational Oral and Poster Abstracts
Oral

Research, Fundamental Science, Sickle Cell Disease, Translational Research, Sickle Cell Trait, Drug development, Assays, Thalassemia, Hemoglobinopathies, Diseases, Treatment Considerations, Thrombotic disorders, Technology and Procedures

Room 31 (San Diego Convention Center)

Moderators:

Malgorzata Kasztan, PhD, The University of Alabama at Birmingham and Rami Khoriaty, MD, University of Michigan

Disclosures:

Khoriaty: Fulcrum therapeutics: Research Funding.

Theme of this session is focusing on novel molecular targets and pathways that modulate sickle cell disease severity. This includes new approaches for fetal hemoglobin induction, reduction in hemoglobin S polymerization and red blood cell sicking.

2:00 PM

169

Development of a ZBTB7A and Wiz Dual Degrading, HbF-Activating CELMoD™ for the Treatment of Sickle Cell Disease

Emily Rychak¹^*, Goldie Yuan Lam Lui, PhD¹^*, Cristhian Fimbres¹^*, Christine Kim Kang¹^*, Younguk Sun, PhD¹^*, Sophie Peng¹^*, Jingjing Deng²^*, Sarah Franc¹^*, Eugene Ciccimaro, PhD²^*, Kimberly Peacock¹^*, Minerva Tran¹^*, Andy Christoforou¹^*, Jinyi Zhu¹^*, Geraldine Polido¹^*, Barbra Pagarigan¹^*, Scott Johnson, PhD¹^*, Faye Wang²^*, Paula Katavolos, PhD²^*, Bonny Gaffney¹^*, Estela Garcia, PhD³^*, France Landry²^*, Fletcher Paddison¹^*, Petia Shipkova, PhD²^*, Hannah Powers, PhD¹^*, Giorgio Tamò, PhD⁴^*, August Dietrich², Soraya Carrancio, PhD¹^*, Radha Ramakrishnan, PhD¹^*, Karen Augustine, PhD²^*, Rama Krishna Narla¹^*, Thomas Cummins¹^*, Mark Rolfe¹, Neil Bence¹^* and Antonia Lopez-Girona¹^*

¹Research and Development, Bristol Myers Squibb, San Diego, CA
²Research and Development, Bristol Myers Squibb, Princeton, NJ
³Center for Innovation and Translational Research Europe (CITRE), Bristol Myers Squibb, Sevilla, Spain
⁴Research and Development, Bristol Myers Squibb, Boudry, Switzerland

2:15 PM

170

The Saga Complex Member TADA2B Is a Novel Regulator of γ-Globin Production

Claire Drysdale, BSc^1,2, Rami Khoriaty, MD^3,4,5,6,7 and Greggory Myers, B.S.^1,8

¹Department of Cell and Developmental Biology, University of Michigan Medical School, Ann Arbor, MI
²Medical Scientist Training Program, University of Michigan Medical School, Ann Arbor, MI
³Department of Internal Medicine, Regents of The University of Michigan, Ann Arbor, MI
⁴Cellular and Molecular Biology Program, University of Michigan, Ann Arbor, MI
⁵Rogel Cancer Center, University of Michigan, Ann Arbor, MI
⁶Department of Internal Medicine, University of Michigan Medical School, Ann Arbor, MI
⁷Department of Cell and Developmental Biology, University of Michigan, Ann Arbor, MI
⁸Department of cell and developmental biology, University of Michigan, Ann Arbor

2:30 PM

171

IMA001 Is a Novel Therapeutic Molecule That Reduces Sickling and Hemolytic Anemia Markers in a Sickle Cell Disease Mouse Model

Marielle Margier, PhD¹^*, Giulia Scorrano, PhD¹^*, Cecile Cros, PhD²^*, Julie Charmetant³^*, Thiago Trovati Maciel, PhD⁴^*, Ousmane Alfa Cisse, MD, MSc⁵^*, Wassim El Nemer, PhD⁶ and Matthias Canault, PhD⁵^*

¹LGD SARL, Velaux, France
²Nuvamid SA, Nyon, Switzerland
³LGD SARL, Velaux, -- select, France
⁴Institut IMAGINE - INSERM U1163, Paris, FRA
⁵NUVAMID SA, NYON, Switzerland
⁶Établissement Français du Sang PACA-Corse, Aix Marseille Université, CNRS, UMR 7268 ADES, Marseille, France

2:45 PM

172

A Novel Small Molecule Drug-Based Approach for Treating Beta-Globinopathies

Jun Liu, MD, PhD¹^*, Ziyang Shen²^*, Yanhua Dong³^*, Na Yu²^*, Li Chai, MD⁴^*, Daniel G. Tenen, MD⁵^*, Daniel E. Bauer, MD, PhD⁶, Adelaide Herman²^*, Braden Pate⁷^* and Kim Vanuytsel, PhD⁸

¹Mass General Brigham, Cambridge, MA
²Mass General Brigham, Boston, MA
³Boston Children's Hospital, Boston
⁴Associate Director of Adult Transfusion Medicine, Brigham And Women's Hospital, Boston, MA
⁵Harvard Stem Cell Institute, Boston, MA
⁶Department of Pediatrics, Harvard Medical School, Cambridge, MA
⁷Boston University, Boston
⁸Boston University, Boston, MA

3:00 PM

173

A Novel Direct Hemoglobin S Polymerization Inhibitor for the Treatment of Sickle Cell Disease – In Vivo Efficacy of Ilx-002 in Humanized Mice

Osheiza Abdulmalik, PhD¹^*, Kandace Gollomp, MD², Veronica Bochenek, BA²^*, Conroy O Field, MS, BS³^*, Mariana Macias¹^*, Benita Balogun¹^*, Salma Roland¹^*, Martin K Safo, PhD⁴^*, Yan Zhang, PhD⁵^*, Akua Donkor, PhD⁵^*, Abdelsattar Omar, PhD⁶^*, Moustafa El-Araby, PhD⁶^*, David R. Light, PhD⁷, Clark Brown, MD, PhD⁷ and Andrew N Fleischman, MD⁸^*

¹Children's Hospital of Philadelphia, Philadelphia, PA
²Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
³Division of Hematology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA
⁴Virginia Commonwealth University, Richmond, VA
⁵Department of Medicinal Chemistry and the Center for Drug Discovery, Virginia Commonwealth University, Richmond, VA
⁶King Abdulaziz University, Jeddah, Saudi Arabia
⁷800 East Leigh Street, Suite 19, Illexcor Therapeutics, Richmond, VA
⁸Illexcor Therapeutics, Richmond, VA

3:15 PM

174

Molecular Mechanism Underlying Excessive Phosphatidylserine Exposure in Sickle Red Blood Cells and Potential Therapeutics

Pengfei Liang, PhD¹, Ke Zoe Shan¹^*, Ryan Chou¹^*, Yui Chun Serena Wan, BS¹, Martha Delahunty, PhD²^*, Sanjay Khandelwal, PhD¹^*, Samuel Francis, MD³, Gow Arepally, MD¹, Marilyn J. Telen, MD² and Huanghe Yang, PhD¹

¹Duke University School of Medicine, Durham, NC
²Duke University Medical Center, Durham, NC
³Emergency Medicine, Duke University School of Medicine, Durham, NC

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^*signifies non-member of ASH

113.O1.6 113. Sickle Cell Disease, Sickle Cell Trait, and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Identification of New Molecular Targets to Modulate Sickle Cell Disease