62nd ASH Annual Meeting and Exposition: 113. Hemoglobinopathies, Excluding Thalassemia—New Genetic Approaches to Sickle Cell Disease: Poster I

Oral and Poster Abstracts
Poster

Adult, Anemias, Biological, HSCs, sickle cell disease, Animal models, bioengineering, Diseases, Non-Biological, sickle cell trait, thalassemia, Therapies, red blood cells, Biological Processes, Hemoglobinopathies, Technology and Procedures, Cell Lineage, Study Population, Clinically relevant, pharmacology, Quality Improvement , flow cytometry, immune mechanism, transplantation, inflammation, mass cytometry, stem cells, metabolomics, pathways

782

The Oral Ferroportin Inhibitor VIT-2763 Prevents Hemolysis and Vaso-Occlusion in a Sickle Cell Disease Model

Rahima Zennadi, PhD¹, Naja Nyffenegger, PhD²^*, Anna Flace²^*, Natarajaswamy Kalleda, PhD²^*, Cédric Doucerain²^*, Giada Ingoglia, PhD²^*, Franz Dürrenberger, PhD²^* and Vania Manolova, Ph.D²^*

¹Medicine, Duke University School of Medicine, Durham, NC
²Vifor (International) Ltd., St. Gallen, Switzerland

783

Characterization of Peripheral Blood Mononuclear Cells Addback Following CD34 Enrichment, Engraftment and T and NK Cells Immune Reconstitution in Patients with High Risk Sickle Cell Disease (SCD) (IND 14359)

Yaya Chu, PhD¹^*, Julie-An Talano, MD², Lee Ann Baxter-Lowe, PhD³, Carolyn A. Keever-Taylor, PhD⁴, Erin Morris, RN, BSN¹^*, Harshini Mahanti, BS, MSCR¹^*, Janet Ayello, MS, MT(ASCP)¹^*, Bryon Johnson, PhD⁵, Rona Singer Weinberg, PhD⁶, Qiuhu Shi, PhD⁷^*, Theodore B. Moore, MD⁸, Sandra Fabricatore, RN, PNP, MSN¹^*, Brenda J. Grossman, MD⁹, Carmella van de Ven, MA¹^*, Shalini Shenoy, MD¹⁰ and Mitchell S Cairo, MD¹

¹Pediatrics, New York Medical College, Valhalla, NY
²Medical College of Wisconsin, Milwaukee, WI
³Pathology, Children’s Hospital Los Angeles, Los Angeles, CA
⁴Medicine, Medical College of Wisconsin, Milwaukee, WI
⁵Division of Hematology, Oncology and Bone Marrow Transplantation, Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI
⁶New York Blood Center, New York, NY
⁷Biostatistics, New York Medical College, Valhalla, NY
⁸UCLA Division of Pediatric Hematology, University of California, Los Angeles, Los Angeles, CA
⁹Pathology and Immunology, Washington University School of Medicine, Saint Louis, MO
¹⁰Department of Pediatrics, Washington University School of Medicine, Saint Louis, MO

784

Oral Administration of FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Has Potent Anti-Sickling Effects in a Sickle Cell Anemia (SCA) Mouse Model, Resulting in Improved RBC Survival and Hemoglobin Levels

Archana Shrestha¹^*, Mengna Chi, PhD², Kimberly Wagner²^*, Adam Drake³^*, Keertik Fulzele, PhD³^*, Sylvie Guichard³^* and Punam Malik, MD²

¹Cincinnati Children's Hospital and Medical Center, Cincinnati, OH
²Cincinnati Children's Hospital Medical Center, Cincinnati, OH
³Forma Therapeutics, Inc., Watertown, MA

785

Validation of a Predictive Score of Acute Chest Syndrome (presev-2 study) in Adults

Christian Kassasseya, MD¹^*, Kene Sekou, MD²^*, Tatiana Besse-Hammer, MD³^*, Ruben Nzouakou, MD⁴^*, Jean-Benoît Arlet, MD⁵^*, Jean Magnang, MD⁶^*, Louis Affo, MD⁷^*, Sandrine Dautheville, MD⁸^*, Stéphanie Ngo, MD⁹^*, Mehdi Khellaf, MD, PhD¹⁰^*, Dapa Aly Diallo, MD, PhD²^* and Pablo Bartolucci, MD, PhD¹¹^*

¹IMRB – Inserm U955 Transfusion et maladies du globule rouge (Equipe 2) Etablissement Français du Sang, APHP Henri Mondor Hospital, Créteil, France
²Centre de Recherche et de Lutte contre la Drépanocytose (CRLD), Bamako, Mali
³CHU Brugmann, Bruxelles, Belgium
⁴Barts Health NHS Trust, London, United Kingdom
⁵Hôpital Européen Georges-Pompidou, Paris, France
⁶Sickle Cell Referral Center, Lomé, Togo
⁷APHP Louis Mourier, Colombes, France
⁸APHP Tenon Hospital, Paris, France
⁹CH Saint-Denis, Saint Denis, France
¹⁰emergency department, Henri Mondor Hospital, Assistance Publique Hopitaux de Paris, Paris XII univers, Creteil, FRA
¹¹Sickle cell referral center, Internal Medicine Unit, Henri Mondor Hospital, Assistance Publique-Hôpitaux de Paris, Creteil, France

786

Ex-Vivo FT-4202 Treatment Improves Hemoglobin Oxygen Affinity and Membrane Health in Red Blood Cells of Patients with Hemoglobin SS and Hemoglobin SC Disease Irrespective of Prior Hydroxyurea Use

Diamantis G. Konstantinidis, PhD, BSc¹^*, Rose Fessler¹^*, Katie Giger Seu, PhD¹, Bhargav Patel^1,2^*, Jennifer B Korpik^1,2^*, Keertik Fulzele, PhD³^*, Sylvie Guichard³^* and Theodosia A. Kalfa, MD, PhD^1,2

¹Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
²Erythrocyte Diagnostic Laboratory, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
³Forma Therapeutics, Inc., Watertown, MA

787

Thrombin-Induced Endothelial Cell Damage Is Mitigated By Human Anti-Thrombin III in a Microfluidic Device

William Wulftange¹^*, Erdem Kucukal, PhD¹^*, Yuncheng Man¹^*, Ran An, PhD¹, Karamoja Monchamp¹^*, Olabimpe Olayiwola, MS¹^*, Jane A. Little, MD², Nigel S. Key, MD² and Umut A. Gurkan, PhD¹

¹Case Western Reserve University, Cleveland, OH
²University of North Carolina at Chapel Hill, Chapel Hill, NC

788

Plasma-Based Inflammatory Signatures in Patients with Sickle Cell Disease during Baseline Health and Acute Pain

Lana Mucalo, MD¹^*, Shuang Jia, MS¹^*, Julie Panepinto, MD, MS², Mark F. Roethle, MS¹^*, Martin J. Hessner, PhD¹^* and Amanda M. Brandow, DO, MS¹

¹Medical College of Wisconsin, Milwaukee, WI
²The Medical College of WI, Milwaukee, WI

789

In Vivo Characterization of Ftx-6058, a Novel Small Molecular Fetal Hemoglobin Inducer for Sickle Cell Disease

Keqiang Xie^*, Mark Roth^*, Ivan Efremov^*, Serena Silver^*, Lucienne Ronco^*, Lorin Thompson^*, Kim Stickland^*, Christopher Moxham^* and Owen Wallace^*

Fulcrum Therapeutics, Cambridge, MA

790

Anti-Inflammatory Activities of Sulfated Non-Anticoagulant Heparin Derivative Beyond Its Anti-Sickling and Anti-Selectin for the Effective Management of Sickle Cell Disease

Noureldien Darwish, MBBCh, PhD¹, Osheiza Abdulmalik, DVM² and Shaker A Mousa, PhD, MSc, BPharm, MBA^3,4

¹The Pharmaceutical Research Institute, ACPHS, Albany, NY
²Division of Hematology, Children's Hosp. of Phila., Philadelphia, PA
³The Pharmaceutical Research Institute, ACPHS, Prof, Rensselaer, NY
⁴Hematology/Vascular, Vascular Vision Pharmaceuticals Co., Rensselaer, NY

791

Sickle Cell Disease Model Mice Lacking 2,3-Dpg Show Reduced RBC Sickling and Improvements in Markers of Hemolytic Anemia

Kelly M. Knee, PhD¹, Amey Barakat, MS¹^*, Lindsay Tomlinson, DVM, DSc²^*, Lila Ramaiah, DVM, PhD³^*, Zane Wenzel, MS⁴^*, Brendon Kapinos⁵^*, Youngwook Ahn⁶^*, Nathanael G. Lintner, PhD⁷^*, S. Denise Field, PhD⁸^*, Reema Jasuja, PhD¹ and Betty A. Pettersen, PhD²^*

¹Rare Disease Research Unit, Pfizer Inc., Cambridge, MA
²Drug Safety Research and Development, Pfizer Inc, Cambridge, MA
³Drug Safety Research and Development, Pfizer Inc, Pearl River, NY
⁴Primary Pharmacology Group- Medicine Design, Pfizer Inc., Groton, CT
⁵Discovery Sciences, Pfizer Inc, Groton, CT
⁶Emerging Science and Innovation, Pfizer Inc, Cambridge, MA
⁷Medicine Design-Chemical Biology, Pfizer Inc, Cambridge, MA
⁸Medicine Design- Chemical Biology, Pfizer Inc, Cambridge, MA

792

Mass-Spectrometry Analysis of Urinary Biomarkers of Endothelial Injury in Sickle Cell Anemia Patients

Harold Girum Dorsey, BS¹, Tatiana Ammosova, PhD²^*, Santosh L. Saraf, MD³, Victor R. Gordeuk, MD⁴, Sergei Nekhai, PhD⁵ and Marina Jerebtsova, PhD⁶

¹Howard University College of Medicine, Silver Spring, MD
²Center for Sickle Cell Disease, Howard University, Washington, DC
³Department of Medicine, University of Illinois at Chicago, Chicago, IL
⁴Sickle Cell Center, Division of Hematology and Oncology, Department of Medicine, University of Illinois at Chicago, Chicago, IL
⁵Center for Sickle Cell Disease and Department of Medicine, Howard University, Washington, DC
⁶Department of Microbiology, Howard University, McLean, VA

793

Reconstruction of Sickle Cell Disease with Circulating Sickling Red Blood Cells in Novel Humanized Cytokines and Liver Mistrg Mice

Yuanbin Song, MD^1,2, Rana Gbyli, MS³^*, Liang Shan, PhD⁴^*, Wei Liu, Ph.D.³^*, Yimeng Gao, PhD³^*, Amisha Patel, MS³^*, Xiaoying Fu, MD^3,5^*, Xiaman Wang, MD^6,7^*, Mina L. Xu, MD⁸^*, Ashley Qin³^*, Emanuela Bruscia, MD⁹^*, Toma Tebaldi, PhD³^*, Giulia Biancon, PhD³, Padmavathi Mamillapalli, BS³^*, David Urbonas¹⁰^*, David Gonzales, MS¹¹^*, Diane S. Krause, MD, PhD^12,13,14,15, Jonathan Alderman, PhD¹⁰^*, Richard Flavell, PhD¹⁰^* and Stephanie Halene, MD ^3,16,17,18

¹Department of Hematology and Oncology, Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou, China
²Section of Hematology, Department of Internal Medicine and Yale Comprehensive Cancer Center, Yale Cancer Center, New Haven, CT
³Section of Hematology, Department of Internal Medicine and Yale Comprehensive Cancer Center, Yale University School of Medicine, New Haven, CT
⁴Department of Medicine, Division of Infectious Diseases, Washington University School of Medicine, St Louis, MO
⁵Department of Laboratory Medicine, Shenzhen Children’s Hospital, Shenzhen, China
⁶Department of Hematology and Oncology, Yale cancer center, New haven, CT
⁷Deparment of Heamatology of the Second Affiliated Hospital, Xi’an Jiaotong University Health Science Center, Xi'an, China
⁸Department of Pathology and Department of Laboratory Medicine, Yale University School of Medicine, New Haven, CT
⁹Yale University School of Medicine, New Haven, CT
¹⁰Department of Immunobiology, Yale University School of Medicine, New Haven, CT
¹¹Department of Genetics, Yale University School of Medicine, New haven, CT
¹²Yale Stem Cell Center, Yale University, New Haven, CT
¹³Laboratory Medicine, Yale University School of Medicine, Yale University, New Haven, CT
¹⁴Laboratory Medicine, Yale University School of Medicine, New Haven, CT
¹⁵Yale Cooperative Center for Excellence in Hematology, Yale University, New Haven, CT
¹⁶Yale Univ. School of Medicine, New Haven, CT
¹⁷Section of Hematology, Department of Internal Medicine, Yale School of Medicine, New Haven, CT
¹⁸Department of Internal Medicine, Section of Hematology, Yale University School of Medicine and Yale Cancer Center, New Haven, CT

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^*signifies non-member of ASH

113.P1.33 113. Hemoglobinopathies, Excluding Thalassemia—New Genetic Approaches to Sickle Cell Disease: Poster I