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, Viral, Adult, Translational Research, Sickle Cell Trait, Drug development, CHIP, Assays, Genetic Disorders, Genomics, Hemoglobinopathies, Diversity, Equity, and Inclusion (DEI), Hematopoiesis, Pediatric, Supportive Care, Diseases, Devices, Immune mechanism, Treatment Considerations, Infectious Diseases, Biological therapies, Non-Biological therapies, Pharmacology, Biological Processes, Molecular biology, Technology and Procedures, Multi-systemic interactions, Study Population, Human, Pathogenesis, Animal model, Maternal Health

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