65th ASH Annual Meeting & Exposition: 113. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Emerging Therapies for Sickle Cell Disease

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

Research, Translational Research, Therapies

Room 29 (San Diego Convention Center)

Moderators:

Huihui Li, PhD, Ichan School of Medicine at Mount Sinai and Shaniqua Johnson Anum, MD, ARRAY(0xe6e4e1c)

Disclosures:

No relevant conflicts of interest to declare.

9:30 AM

Protein Phosphatase 6 Complex: Novel Regulator of Fetal Hemoglobin and Potential Therapeutic Target in Sickle Cell Disease

Scott A. Peslak, MD, PhD^1,2, Talla Abbas^1,2^*, Eugene Khandros, MD, PhD², Elizabeth A. Traxler, MD, PhD^2,3, Osheiza Abdulmalik, DVM²^*, Belinda M. Giardine⁴^*, Cheryl A. Keller, PhD^4,5^*, Ross C. Hardison, PhD⁴^*, Junwei Shi, PhD⁶^* and Gerd A. Blobel, MD, PhD²

¹Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA
²Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
³Division of Hematology/Oncology, Department of Medicine, Hospital of University of Pennsylvania, Philadelphia, PA
⁴Department of Biochemistry and Molecular Biology, The Pennsylvania State University, University Park, PA
⁵Genomics Research Incubator, The Pennsylvania State University, University Park, PA
⁶Department of Cancer Biology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA

9:45 AM

Predictive Biomarker Analysis from the GBT021601 Survival Study in Townes Sickle Mice

Mira Patel Pochron¹^*, Jiaxin Sun¹^*, Alka A Potdar, PhD¹^*, Carsten Alt¹^*, Francis DeGuzman¹^*, Michael W. Connor¹^*, Brian Cathers¹^*, Kobina Dufu, PhD¹^* and David R Archer, PhD²

¹Pfizer Inc, South San Francisco, CA
²Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta, Emory University Department of Pediatrics, Atlanta, GA

10:00 AM

Ineffective Erythropoiesis Negatively Impacts the Erythroblastic Island in Sickle Cell Disease

Auria Godard¹^*, Julien Grenier, PhD¹^*, Jonatane Andrieu¹^*, Sophie Beley¹^*, Pascale Poullin²^*, Sandra Manceau³^*, Laure Joseph, MD⁴^*, Maria De Grandis, PhD¹^* and Wassim El Nemer, PhD¹

¹EFS PACA-Corse, Aix Marseille University, UMR 7268 ADES, Marseille, France
²AP-HM, Marseille, FRA
³Centre De RéféRence Des Syndromes DréPanocytaires Majeurs,, Paris, France
⁴Necker Hospital (APHP), Paris, France

10:15 AM

Evaluation of GBT021601 As a Therapeutic Agent to Restore Bone Marrow Health and Effective Erythropoiesis in a Sickle Mouse Model

Britney Hernandez¹^*, Anupama Priyadarshini¹^*, Zachary Kostamo, BS¹^*, Yankai Zhang¹^*, Ashwin P Patel, MD, PhD, MPH¹, Michael A Pendergast¹^*, Madhulika Jupelli²^*, Heli Huang²^*, Annie Nguyen Dang²^*, Mira Patel Pochron²^*, Kobina Dufu, PhD²^* and Vivien A Sheehan, PhD¹

¹Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA
²Pfizer Inc, South San Francisco, CA

10:30 AM

Clinical and Hematopoietic Profiles Associated with Sustained Hydroxyurea Response for Patients with Sickle Cell Disease

Luisanna M Sanchez Ventura, MD¹, Ijele Adimora, MD, MBA², Emani C. Elliot¹^*, Titilope Fasipe, MD, PhD¹ and Jonathan M. Flanagan, PhD¹

¹Texas Children's Cancer & Hematology Center, Baylor College of Medicine, Houston, TX
²The Johns Hopkins University School of Medicine, Baltimore, MD

10:45 AM

UM171 Enhances Fitness and Engraftment of Gene Modified Hematopoietic Stem Cells from Sickle Cells Disease Patients

Boya Liu, PhD¹^*, Denise Klatt²^*, Yu Zhou²^*, John Manis, MD³, Guy Sauvageau, MD/PhD⁴, Christian Brendel, PhD¹^* and David A. Williams, MD¹

¹Boston Children's Hospital, Boston, MA
²Boston Children's Hospital, Boston
³Children's Hospital Boston, Boston, MA
⁴Institut de recherche en Immunologie et Cancer, Université de Montréal, Montreal, QC, Canada

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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: Emerging Therapies for Sickle Cell Disease