62nd ASH Annual Meeting and Exposition: 102. Regulation of Iron Metabolism

Oral and Poster Abstracts
Oral

Anemias, Biological, Diseases, Non-Biological, thalassemia, Therapies, Genetic Disorders, red blood cells, Biological Processes, Hemoglobinopathies, iron deficiency, Cell Lineage, erythropoiesis, Clinically relevant, iron metabolism, microenvironment, pathways

Moderators:

Morgan L. McLemore, MD, Emory University and Francesca Vinchi, PhD, New York Blood Center

Disclosures:

Vinchi: Novartis: Research Funding; Silence Therapeutics: Membership on an entity's Board of Directors or advisory committees, Research Funding; PharmaNutra: Research Funding; Vifor Pharma: Research Funding.

2:00 PM

219

Transgenic Mice Overexpressing Erythroferrone, a Novel Erythrokine, Develop Iron Overload and Multi-Organ Iron-Independent Abnormalities

Richard Coffey, PhD¹, Grace Jung, MS¹^*, Elizabeta Nemeth, PhD¹ and Tomas Ganz, MD, PhD²

¹Center for Iron Disorders, David Geffen School of Medicine at UCLA, Los Angeles, CA
²Center for Iron Disorders, University of California, Los Angeles, Los Angeles, CA

2:15 PM

220

Transferrin Receptor 2 Is a Novel Target to Ameliorate Anemia of Chronic Kidney Disease

Violante Olivari^1,2^*, Mariam Aghajan, PhD³^*, Maria Rosa Lidonnici, PhD⁴^*, Mariateresa Pettinato, MSc^1,2^*, Laura Silvestri, PhD^1,2, Giuliana Ferrari, PhD^1,4, Shuling Guo, PhD³^*, Clara Camaschella, MD² and Antonella Nai, PhD^1,2

¹Vita-Salute San Raffaele University, Milano, Italy
²Division of Genetics and Cell Biology, IRCCS San Raffaele Scientific Institute, Milano, Italy
³Ionis Pharmaceuticals, Inc., Carlsbad, CA
⁴San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy

2:30 PM

221

Xanthine Oxidase Has a Protective Role during Heme Crisis By Binding and Degrading Heme

Heidi M Schmidt^1,2, Scott Hahn, MS³^*, Gowtham K Annarapu, PhD⁴, Mara Carreño⁴^*, Francisco Schopfer⁴^*, Sruti Shiva, PhD¹^*, Dario A. Vitturi, PhD⁵^*, Eric Kelley, PhD⁶^* and Adam C Straub, PhD^1,7^*

¹Department of Pharmacology and Chemical Biology, University of Pittsburgh, Pittsburgh, PA
²Pittsburgh Heart, Lung and Blood Vascular Medicine Institute, Department of Medicine, Univeristy of Pittsburgh, Pittsburgh, PA
³Pittsburgh Heart, Lung and Blood Vascular Medicine Institute, Department of Medicine, University of Pittsburgh, Pittsburgh, PA
⁴University of Pittsburgh, Pittsburgh, PA
⁵Heart, Lung, Blood and Vascular Medicine Institute, Department of Medicine; Department of Pharmacology and Chemical Biology, University of Pittsburgh, Pittsburgh, PA
⁶West Virginia University, Morgantown, WV
⁷Heart, Lung, Blood and Vascular Medicine Institute, Department of Medicine, University of Pittsburgh, Pittsburgh, PA

2:45 PM

222

Eighteen-Month Interim Analysis of Efficacy and Safety of Givosiran, an RNAi Therapeutic for Acute Hepatic Porphyria, in the Envision Open Label Extension

David J. Kuter, MD¹, Sioban Keel, MD², Charles J. Parker, MD³, David C. Rees, MD⁴^*, Ulrich Stölzel, MD⁵^*, Paolo Ventura, MD⁶^*, Manisha Balwani⁷^*, Laurent Gouya, M.D., Ph.D.⁸^*, Amy Simon, MD⁹^*, Shangbin Liu, PhD.⁹^*, John J Ko, PharmD, MSc⁹, Sean Rhyee, M.D., M.P.H⁹^* and Samuel M. Silver, MD, PhD¹⁰

¹Hematology Division, Massachusetts General Hospital, Boston, MA
²Department of Medicine, University of Washington, Seattle, WA
³University of Utah School of Medicine, Salt Lake City, UT
⁴Department of Paediatric Haematology, King's College Hospital, London, United Kingdom
⁵Klinikum Chemnitz, Chemnitz, Germany
⁶Università degli Studi di Modena e Reggio Emilia, Emilia, Italy
⁷Icahn School of Medicine at Mount Sinai, New York
⁸Centre Français des Porphyries, Paris, France
⁹Alnylam Pharmaceuticals, Cambridge, MA
¹⁰University of Michigan, Ann Arbor, MI

3:00 PM

223

FKBP12-Dependent Hepcidin Inhibition: Molecular Mechanism and Potential Therapeutic Implications

Mariateresa Pettinato, MSc^1,2^*, Alessia Pagani, PhD²^*, Alessandro Dulja, MSc^2,3^*, Mariam Aghajan, PhD⁴^*, Silvia Colucci, MSc^5,6^*, Violante Olivari^1,2^*, Antonella Nai, PhD^1,2, Jessica Bordini, PhD⁷^*, Alessandro Campanella, PhD⁷^*, Martina Muckenthaler, MSc, PhD^5,6, Shuling Guo, PhD⁴^*, Clara Camaschella, MD² and Laura Silvestri, PhD^1,2

¹Vita-Salute San Raffaele University, Milano, Italy
²Division of Genetics and Cell Biology, IRCCS San Raffaele Scientific Institute, Milano, Italy
³European Molecular Biology Laboratory, Heidelberg, Germany
⁴Ionis Pharmaceuticals, Inc., Carlsbad, CA
⁵Molecular Medicine Partnership Unit (MMPU), Heidelberg, Germany
⁶Department of Pediatric Hematology, Oncology and Immunology, University of Heidelberg, Heidelberg, Germany
⁷Division of Experimental Oncology, IRCCS San Raffaele Scientific Institute, Milano, Italy

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^*signifies non-member of ASH

102.O1.6 102. Regulation of Iron Metabolism