66th ASH Annual Meeting: 113. Sickle Cell Disease, Sickle Cell Trait, and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Poster I

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

Research, Fundamental Science, Sickle Cell Disease, Combination therapy, Translational Research, Sickle Cell Trait, Drug development, Hemoglobinopathies, Diseases, Devices, Treatment Considerations, Non-Biological therapies, Pharmacology, Biological Processes, Molecular biology, Multi-systemic interactions, Pathogenesis

Halls G-H (San Diego Convention Center)

This session includes cross-sectional pre-clinical and translational studies on sickle cell disease.

1100

Circulating Extracellular Vesicles Enable IFN-I Response in Neutrophils of Sickle Cell Disease Mice

Ayyanar Sivanantham, PhD¹^*, Omika Katoch, PhD²^*, Nicholas Swendrowski, MS²^* and Prithu Sundd, PhD^2,3

¹Thrombosis and Hemostasis Program, VERSISI BLOOD RESEARCH INSTITUTE AND BLOOD CENTER OF WISCONSIN, MILWAUKEE, WI
²Thrombosis and Hemostasis Program, VERSITI Blood Research Institute and Blood Center of Wisconsin, Milwaukee, WI
³Medical College of Wisconsin, Division of Hematology and Oncology, Milwaukee, WI

1101

Voxelotor and Red Blood Cell Pyruvate Kinase Activator Enhance Sickle Erythrocyte Deformability By Calcium Efflux

Zoe Sekyonda, BS¹^*, Solomon Oshabaheebwa, MSc, BSc²^*, Ada Chang, BSc³^*, Jane A. Little, MD³ and Umut Gurkan, PhD⁴

¹Case Western Reserve University, Cleveland, OH
²Case Western Reserve University, Cleveland Heights, OH
³University of North Carolina, Chapel Hill, NC
⁴Case Western Reserve University, Shaker Heights, OH

1102

Platelet RIG-I Promotes Flu-Induced Severe Lung Injury in Sickle Cell Disease

Tomasz Kaminski, PharmD¹, Nicholas Swendrowski, MS²^*, Omika Katoch, PhD²^* and Prithu Sundd, PhD^2,3

¹Thrombosis and Hemostasis Program, VERSITI Blood Research Institute, Milwaukee
²Thrombosis and Hemostasis Program, VERSITI Blood Research Institute, Milwaukee, WI
³Medical College of Wisconsin, Division of Hematology and Oncology, Milwaukee, WI

1103

IMA001 Is a Novel Therapeutic Molecule That Improves Hematological Parameters during Vaso-Occlusion in Sickle Cell Disease In Vivo

Marielle Margier, PhD¹^*, Giulia Scorrano, PhD¹^*, Cecile Cros, PhD²^*, Julie Charmetant³^*, Thiago Trovati Maciel, PhD⁴^*, Ousmane Alfa Cisse, MD, MSc⁵^*, Wassim El Nemer, PhD^6,7 and Matthias Canault, PhD⁵^*

¹LGD SARL, Velaux, France
²Nuvamid SA, Nyon, Switzerland
³LGD SARL, Velaux, -- select, France
⁴Institut Imagine - INSERM U1163, Paris, France
⁵NUVAMID SA, NYON, Switzerland
⁶French Blood Establishment(EFS), Marseille, France
⁷Établissement Français du Sang PACA-Corse, Aix Marseille Université, CNRS, UMR 7268 ADES, Marseille, France

1104

Small Activating RNA-Mediated Induction of HBG Via Liposome Delivery for In Vivo Treatment of Sickle Cell Disease and Beta-Thalassemia

Laura Sinigaglia, PhD¹^*, Marcella O'Reilly, PhD²^*, Rhea Bhalla, MSc³^*, Khoa Chung, PhD²^*, Pardis Piri Dizaji, MSc²^*, Victoria Begley, PhD⁴^*, Rose Hodgson, PhD²^*, Gabriela Lin, PhD²^*, Konstantinos Vanezis, PhD⁴^*, Julieta Tesone, PhD⁴^*, Helen Paterson, PhD⁴^*, Shreyasi Swamy, MSc²^*, Henrik Hansen, PhD⁵^*, Natalia Izotova, PhD⁶^*, Bruno Doreste, PhD⁶^*, Yulia Lomonosova, PhD⁷^*, Luke Haslett, PhD⁶^*, Robert Habib, MBA^8,9^*, Robert F Place, PhD⁷^*, Jon Voutila, PhD⁷^*, Vikash Reebye, PhD^8,10^*, Troels Koch, PhD⁶^* and Brid M Ryan, PhD MPH¹¹^*

¹Rare Genetic Diseases, MiNA Therapeutics Ltd, London, United Kingdom
²Rare Genetic Diseases, MiNA Therapeutics, London, United Kingdom
³Rare Genetic Diseases, MiNa Therapeutics, London, United Kingdom
⁴Rare Genetic Diseases, MiNA therapeutics, London, United Kingdom
⁵MiNA therapeutics, London, United Kingdom
⁶Rare Genetic Disease, MiNA therapeutics, London, United Kingdom
⁷Rare Genetic Disease, MiNA Therapeutics, London, United Kingdom
⁸Mina Therapeutics Ltd, London, United Kingdom
⁹Rare Genetic Disease, MiNA therapeutics, london, United Kingdom
¹⁰Imperial College London, London, United Kingdom
¹¹Genetic Medicine, MiNA Therapeutics, London, United Kingdom

1105

Single Cell Hemoglobin Quantification Reveals HbF and HbS Increases in Sickle Erythrocytes from Patients Treated with Hydroxyurea

Laura Bencheikh, PhD¹^*, Laura Matabishi-Bibi, PhD¹^*, Gabriela Luszczewska²^*, Meghan Perkins, PhD¹^*, Nicolas Hebert, PhD^3,4^*, Nagamy Rose Balli²^*, Yosr Zaouali⁵^*, Vincent Malcor Deydier De Pierrefeu, PhD⁶^*, Fatima Bensiradj, MD⁶^*, Caroline Barau, PhD⁷^*, France Pirenne, MD, PhD^8,9,10, Carlo Brugnara, MD¹¹, Martin H. Steinberg, MD¹², Haydar Frangoul, MD¹³, Marie Cambot, PhD¹^* and Pablo Bartolucci, MD, PhD¹⁴^*

¹INNOVHEM, Créteil, France
²INNOVHEM, Creteil, France
³Université Paris-Est Créteil, INSERM, IMRB, Laboratory of excellence LABEX GRex, Créteil, France, Créteil, France
⁴University Paris-Est-Créteil, IMRB, Inserm U955, Laboratory of excellence LABEX, Établissement Français du Sang (EFS), Créteil, France
⁵Sickle Cell Referral Center - UMGGR, Plateforme D'Expertise Maladies Rares Grand Paris, Creteil, France
⁶Sickle Cell Referral Center, Department of Internal Medicine, Henri-Mondor University Hospital- UPEC, AP-HP, Creteil, France
⁷Plateforme de Ressources Biologiques, Henri Mondor Hospital, AP-HP, Creteil, France
⁸Université Paris-Est Créteil , Henri Mondor Hospital, Créteil, France
⁹Transfusion et maladies du globule rouge - Equipe Pirenne, Institut Mondor de Recherche Biomedicale (IMRB), Creteil, France
¹⁰Etablissement Francais Du Sang - Université Paris-Est Créteil Hospital, Creteil, France
¹¹Lab of Medicine and Pathology, Children’s Hospital, Harvard Medical School, Boston, Boston, MA
¹²Boston University Chobanian & Avedisian School of Medicine, Boston, MA
¹³Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville, TN
¹⁴University Paris-Est-Créteil, IMRB, Inserm U955,, Henri Mondor University Hospitals, APHP, Sickle Cell Referral Center-UMGGR, Creteil, France

1106

IHP-102 Reduces Hypoxia-Induced Pain Behaviors in Townes SCD Mice

Kennedy N Goldsborough, PhD¹^*, Israel Almeida²^*, Ashwin P. Patel, MD, PhD, MPH³, John Paderi, PhD⁴^* and Vivien Sheehan, MD, PhD⁵

¹Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA
²Department of Biology, Emory University, Atlanta, GA
³Department of Pediatrics, Emory University School of Medicine, Atlanta, GA
⁴IHP Therapeutics, San Carlos, CA
⁵Emory University School of Medicine, Atlanta, GA

1107

Dual Activation of PKR and PKM2 Reduced the Development of Fibrosis and Iron Deposition in a Sickle Cell Disease Nephropathy Mouse Model

Trang Nguyen¹^*, Christy Ingersoll, MBA¹^*, Katie Lizotte, ScB²^*, Chi-Chao Chen, MD, PhD²^*, Erin Tsai, MS¹^*, Megan Wind-Rotolo, PhD¹ and Hui Gao, PhD¹^*

¹Agios Pharmaceuticals, Inc., Cambridge, MA
²Employee of Agios Pharmaceuticals, Inc. at time of research, Cambridge, MA

1108

Quinine-Hemin Compound As an Anti-Inflammatory Therapy in Sickle Cell Disease

Kai Dou, PhD¹^*, Rikang Wang, PhD²^*, Weili Bao, MS³^*, Yunfeng Liu, PhD³, Shan Su, PhD³, Avital Mendelson, PhD⁴, Cheryl Lobo, PhD⁵, Patricia Shi, MD⁶, Xiuli An, MD, PhD⁷, Karina Yazdanbakhsh, PhD³ and Hui Zhong, PhD⁸

¹Lab of immune regulation, Lindsley F. Kimball Research Institute, New York Blood Center, New York, NY
²Department of Neurosurgery, Peking University Shenzhen Hospital, Shenzhen, China
³Laboratory of Complement Biology, Lindsley F. Kimball Research Institute, New York Blood Center, New York, NY
⁴Laboratory of Stem Cell Biology and Engineering, Lindsley F. Kimball Research Institute, New York Blood Center, New York, NY
⁵Laboratory of Blood-Borne Parasites, Lindsley F. Kimball Research Institute, New York Blood Center, New York, NY
⁶New York Blood Center, New York, NY
⁷Laboratory of Membrane Biology, Lindsley F. Kimball Research Institute, New York Blood Center, New York, NY
⁸Laboratory of Immune Regulation, Lindsley F. Kimball Research Institute, New York Blood Center, New York, NY

1109

Cereblon-Based Compounds for Induction of HbF

Aline Renata Pavan, PhD¹^*, Victor Gabriel de Faria Pastre, BSc¹^*, Carolina Lanaro, BSc, PhD¹^*, Dulcineia Albuquerque, PhD¹^*, Joshua P. Smalley, PhD²^*, Urvashi Patel, BSc²^*, John W. R. Schwabe, PhD³^*, Shaun M. Cowley, PhD⁴^*, James T. Hodgkinson, PhD²^*, Jean Leandro Dos Santos, PhD⁵^* and Fernando Ferreira Costa, MD, PhD¹

¹Hematology and Hemotherapy Center, University of Campinas - UNICAMP, Campinas, Brazil
²Leicester Institute of Structural and Chemical Biology and School of Chemistry, University of Leicester, Leicester, United Kingdom
³Leicester Institute of Structural and Chemical Biology and Department of Molecular and Cell Biology, University of Leicester, Leicester, United Kingdom
⁴Department of Molecular and Cell Biology, University of Leicester, Leicester, United Kingdom
⁵School of Pharmaceutical Sciences, Sao Paulo State University, Araraquara, Brazil

1110

Novel Loci Associated with Acute Chest Syndrome in Sickle Cell Disease Patients

Anvita Kulshrestha¹, Melanie E. Garrett, MS¹^*, Marilyn J. Telen, MD² and Allison E. Ashley-Koch, PhD¹^*

¹Duke Molecular Physiology Institute, Duke University Medical Center, Durham, NC
²Duke University Medical Center, Durham, NC

1111

Daprodustat Induces Fetal Hemoglobin and Erythropoietic Drive in an in Vitro and In Vivo Model of Sickle Cell Disease

Malini Sharma, BA¹, Fabliha Ahmed Chowdhury, PhD², Stefanie Taiclet, BS²^*, Brian J Philips, PhD³^*, Katherine C Wood, PhD^2,3, Adam C Straub, PhD^2,3^* and Julia Xu, MD, MS^4,5

¹University of Pittsburgh School of Medicine, Pittsburgh, PA
²Department of Pharmacology and Chemical Biology, University of Pittsburgh, Pittsburgh, PA
³Pittsburgh Heart, Lung, and Blood Vascular Medicine Institute, Department of Medicine, University of Pittsburgh, Pittsburgh, PA
⁴Division of Classical Hematology, Department of Medicine, University of Pittsburgh, Pittsburgh, PA
⁵University of Pittsburgh, Pittsburgh, PA

1112

Iron Deficiency in HbSC Disease Is Associated with Less Sickle Cell Disease-Related Complications – a Rationale for Repetitive Phlebotomy As Disease Modifying Therapy

Marissa J.M. Traets, MD¹^*, Aida Kidane, MD^2,3^*, Jennifer Bos¹^*, Brigitte A. van Oirschot¹^*, Sigrid van der Veen, MSc⁴^*, Mandy N. Lauw, MD, PhD⁵, Erfan Nur, MD, PhD^6,7, Bart J. Biemond, MD, PhD⁸, Marjon H. Cnossen, MD, PhD⁹^*, Eduard J. Van Beers, MD, PhD¹⁰, Richard van Wijk, PhD¹ and Minke A.E. Rab, MD, PhD^1,11

¹Central Diagnostic Laboratory - Research, University Medical Center Utrecht, Utrecht, Netherlands
²Department of Pediatric Hematology, Erasmus MC Sophia Children's Hospital, Rotterdam, Netherlands
³Section on Social and Cognitive Developmental Neuroscience, National Institutes of Mental Health, Bethesda, MD
⁴Center for Benign Hematology, Thrombosis and Hemostasis - Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Netherlands
⁵Department of Hematology, Erasmus University Medical Center, Rotterdam, Netherlands
⁶Department of Hematology, Amsterdam UMC location University of Amsterdam, Amsterdam, Netherlands
⁷Department of Molecular Hematology, Sanquin Research and Landsteiner Laboratory, Amsterdam, Netherlands
⁸Department of Hematology, Amsterdam University Medical Centers, Amsterdam, Netherlands
⁹Department of Pediatric Hematology and Oncology, Erasmus MC Sophia Children’s Hospital, Erasmus University Medical Center, Rotterdam, Netherlands
¹⁰Center for Benign Hematology, Thrombosis and Hemostasis - Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
¹¹Department of Hematology, Erasmus MC, Rotterdam, Netherlands

1113

Short-Term Fostamatinib Treatment Impacts Hematopoiesis and Downregulates Thromboinflammation in Townes Sickle Mice

Bindu Parachalil Gopalan, PhD¹^*, Sayuri Kamimura, MS²^*, Pradeep Dagur, PhD³^*, Niharika Shah, MD⁴^*, Martha Quezado, MD⁴^*, Zenaide Quezado, MD² and Arun S. Shet, MD, PhD¹

¹Sickle Thrombosis and Vascular Biology Lab, Cellular and Molecular Therapeutic Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
²Department of Perioperative Medicine, National Institutes of Health Clinical Center, National Institutes of Health, Bethesda, MD
³Flow Cytometry Core, National Heart Lung, and Blood Institute (NHLBI), National Institutes of Health (NIH), Bethesda, MD
⁴Laboratory of Pathology, National Cancer Institute, National Institutes of Health, Bethesda, MD

1114

The Impact of Chronic Hemolysis on Placental Angiogenesis in a Mouse Model of Sickle Cell Disease

Nirupama Ramadas, PhD, MSc, BSc¹^*, Joshua Dutton²^*, Ashlyn Lowery²^* and Erica Sparkenbaugh, PhD^3,4

¹Blood Research Center, University of North Carolina at Chapel Hill, Carrboro, NC
²Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill, NC
³Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC
⁴Blood Research Center, University of North Carolina Chapel Hill, Chapel Hill, NC

1115

A Novel Oral Prodrug AN233 Induces Fetal Hemoglobin Expression in Sickle Cell Disease Mice and Baboons

Chithra D. Palani, PhD^1,2, Abraham Nudelman, PhD³^*, Ada W. Rephaeli, PhD⁴^*, Donald E. Lavelle, PhD⁵, Manickam Alagar, PhD⁶^*, Nikhil Patel, MD⁷^*, Zhi Wenbo, PhD⁸^* and Betty S. Pace, MD^2,9

¹Department of Pediatrics, Augusta University, Augusta, GA
²Georgia Cancer Center, Augusta University, Augusta, GA
³Chemistry Department, Bar Ilan University, Ramat Gan, Israel
⁴Felsenstein Medical Research Center, Petach Tikva, Israel
⁵Department of Medicine, University of Illinois at Chicago, Chicago, IL
⁶Stem Cell Processing Lab, Transfusion Services, Wellstar MCG Health, Augusta, GA
⁷Department of Pathology and Laboratory Medicine, Medical College of Georgia at Augusta University, Augusta, GA
⁸Center for Biotechnology and Genomic Medicine, Augusta University, Augusta, GA
⁹Division of Hematology/Oncology, Augusta University, Augusta, GA

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^*signifies non-member of ASH

113.P1.47 113. Sickle Cell Disease, Sickle Cell Trait, and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Poster I