65th ASH Annual Meeting & Exposition: 102. Iron Homeostasis and Biology: Exploring Molecular Mechanisms and Therapeutic Options in Iron Homeostasis

Symposia: Iron Homeostasis and Biology Oral and Poster Abstracts
Oral

Research, clinical trials, Translational Research, Clinical Research, Diseases, Therapies, Maternal Health

Room 25 (San Diego Convention Center)

Moderators:

Immacolata Andolfo, immacolata.andolfo@unina.it, and Sarah Ducamp, PhD, Boston Children's Hospital and Harvard Medical School

Disclosures:

No relevant conflicts of interest to declare.

In this session, we will delve into both new molecular mechanisms and therapeutic options in iron homeostasis. Our exploration will span a diverse array of topics. This includes uncovering the novel role of PCBP1 in the regulation of iron efflux through intestinal ferroportin. Moreover, we will investigate the iron regulation orchestrated by Irf5 in macrophages within erythromyeloblastic islands.

Additionally, we will extensively examine emerging therapeutic opportunities designed to uphold iron homeostasis. This comprehensive evaluation will encompass analysis of clinical trials that specifically target rare conditions, such as congenital hypotransferrinemia and erythropoietic protoporphyria.

Conducting innovative structure-function analysis, we will unlock insights into the structural predictions that will facilitate the pharmacological targeting of human erythroferrone in the future.

As a crucial facet, we recognize that iron plays an indispensable role in maternal and fetal health throughout pregnancy. In light of this, we will dissect the synergistically adverse effects resulting from the interaction between maternal iron deficiency and inflammation on the embryo during pregnancy.

4:30 PM

919

The Iron Chaperone Poly C Binding Protein 1 Regulates Iron Efflux through Intestinal Ferroportin in Mice

Yubo Wang¹^*, Olga Protchenko¹^*, Kari Huber¹^*, Minoo Shakoury-Elizeh¹^* and Caroline Philpott, MD²

¹NIDDK, Bethesda, MD
²National Institutes of Health, Bethesda, MD

4:45 PM

920

Irf5 Expression in Macrophages Contributes to Iron Regulation within Erythromyeloblastic Islands

Rachel Josselsohn^1,2, Anshul Vagrecha, MD, MBBS³, Margaret LaPan³^*, Julien Papoin, MS³^*, Jonah Kamen³^*, Young Min Cho³^*, Riaz Rao³^*, Laurel Romano, PhD⁴^*, Nathan Salomonis, PhD⁵^*, Theodosia A. Kalfa, MD, PhD⁶, Lionel Blanc, PhD^2,3 and Betsy J. Barnes, DPhil³

¹Feinstein Institutes for Medical Research, Feinstein Institute For Medical Research, Manhasset, NY
²Donald and Barbara Zucker School of Medicine at Hofstra/Northwell, Hempstead, NY
³Feinstein Institutes for Medical Research, Northwell, Manhasset, NY
⁴Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁵Division of Biomedical Informatics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁶Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH

5:00 PM

921

Alphafold2-Assisted Structure-Function Analysis of Human Erythroferrone Relevant for Its Iron-Regulatory Function

Daniel Srole¹^*, Chun-Ling Jung¹^*, Alan J Waring²^*, Elizabeta Nemeth, PhD¹ and Tomas Ganz¹

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

5:15 PM

922

Treatment of Congenital Hypotransferrinemia Patients with Human Apotransferrin Resolves Anemia and Reduces Iron Accumulation

Ilona Kleine Budde, PhD¹^*, Cristina Díaz De Heredia, MD²^*, Raffaella Mariani, MD, PhD³^*, Alberto Piperno, MD, PhD^4,5^*, Katja Moser, MD⁶^*, Mayka Sanchez, PhD^7,8^*, Thomas Bodewes, MD, PhD¹^* and Maya Isaila, MD, PhD¹^*

¹Medical and Clinical Affairs, Prothya Biosolutions, Amsterdam, Netherlands
²Hospital Universitari Vall d’Hebron, Vall d’Hebron Institut de Recerca, Barcelona, Spain
³Centre for Rare Diseases, European Reference Network, EuroBloodNet, Fondazione IRCCS San Gerardo dei Tintori, Monza, ITA
⁴Genetic, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy
⁵Tettamanti Research Centre, Monza, Italy
⁶Klinik für Kinder- und Jugendmedizin, Klinikum Aschaffenburg-Alzenau, Aschaffenburg, Germany
⁷Department of Basic Sciences, Iron Metabolism: Regulation and Diseases Group, Universitat Internacional De Catalunya, Sant Cugat Del Vallès-Barcelona, Spain
⁸Diagnostics in Inherited Blood Diseases, BloodGenetics S.L., Esplugues de Llobregat, Spain

5:30 PM

923

Interim Analyses from the Beacon Trial: A Phase 2, Randomized, Open-Label Trial of Bitopertin in Erythropoietic Protoporphyria

Gayle Ross, MD¹^*, Peter Stewart, MD²^*, George Mensing, MPH³^*, Melanie Chin, PhD³^*, Haley Howell, BS³^*, Heidi Mangus³^* and William Savage, MD, PhD³

¹Royal Melbourne Hospital, Melbourne, Australia
²Royal Prince Alfred Hosptial, Sydney, Australia
³Disc Medicine, Watertown, MA

5:45 PM

924

Synergy between Iron Deficiency and Inflammation Adversely Affects Pregnancy

Veena Sangkhae, PhD¹, Allison L Fisher, PhD²^*, Vida Zhang, PhD¹^*, Tomas Ganz, MD, PhD¹ and Elizabeta Nemeth, PhD¹

¹Center for Iron Disorders, David Geffen School of Medicine at UCLA, Los Angeles, CA
²Division of Nephrology, Program in Membrane Biology, Massachusetts General Hospital, Harvard Medical School, Boston, MA

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^*signifies non-member of ASH

102.O1.6 102. Iron Homeostasis and Biology: Exploring Molecular Mechanisms and Therapeutic Options in Iron Homeostasis