Scientific Program

Unlocking Blood Disorders: Targeting Genetics to Identify Early Phenotypes

Scientific Committee on Blood Disorders in Childhood

This session will highlight the recent advancements in understanding the underlying genetic mutations driving von Willebrand disease, neutrophil disorders, and platelet disorders in children, adolescents, and young adults. Improved understanding of these genetic mutations helps to predict disease course, complications, and response to treatment of pediatric patients. This knowledge can also lead to the development of new targeted inhibitor strategies and/or gene therapies. This session will be of broad interest for clinical pediatric and medical hematologists, geneticists, and laboratory scientists interested in various aspects of classical hematology, including bone marrow failure syndromes.

Chair:

Sarah Tasian, MD
Children's Hospital of Philadelphia
Philadelphia, PA

Speakers:

Danny Miller, MD, PhD
University of Washington
Seattle, WA
Decoding the Mysteries of the VWF Gene

Akiko Shimamura, MD, PhD
Boston Children's Hospital
Boston, MA
Neutropenia Uncovered: The Role of Genetic Testing

Alessandra Balduini Sr, MD
Tufts University
Medford, MA
New Insights into Inherited Thrombocytopenias

back to top

The Great Escape- Immune Evasions in Bone Marrow Failure

Scientific Committee on Bone Marrow Failure

This session will explore the role of clonal hematopoiesis (CH) in bone marrow failure (BMF) syndromes. There has been significant progress in understanding the impact of clonal hemopoiesis in immune mediated aplastic anemia in the last 10 years. Several studies have looked at the bulk clonal architecture and have correlated with progression to myelodysplastic syndromes and acute myeloid leukemia. Recent work has improved on this using single cell sequencing to understand the architecture of CH and more importantly the biological implications for disease biology and progression to myeloid neoplasms. The role of somatic myeloid mutations, escape clones like 6pLOH and PIGA will be discussed, with links to ageing and CH. Additionally the role of new acquired BMF syndrome like VEXAS and the implications of clonal dominance linked with ageing will also be discussed

Chair:

Austin G. Kulasekararaj Jr, MD, MBBS, FRCPath, MRCP
King's College Hospital
London, United Kingdom

Speakers:

Fernanda Gutierrez-Rodrigues, PhD
National Heart, Lung and Blood Institutes
Bethesda, MD
Clonal Selection of Somatic Mutations in Immune Mediated BMF

Marcin W Wlodarski, MD, PhD
St. Jude Children's Research Hospital
Memphis, TN
Escape Clones in Immune Mediated BMF

Mrinal M. Patnaik, MD, MBBS
Mayo Clinic
Rochester, 
Clonal Dominance in VEXAS

back to top

JOINT SESSION - New Horizons in RNA Biology in Myeloid Development and Malignancy

Scientific Committee on Epigenetics and Genomics + Scientific Committee on Myeloid Biology

Dynamic RNA regulation is a central determinant of normal hematopoiesis, and a multifaceted driver of blood disease. Every step of the RNA life cycle, from splicing and modification to RNA-protein interactions and non-coding functions, shapes stem cell homeostasis, lineage choice, immune recognition, and malignant transformation. This session will feature recent discoveries that expand the RNA footprint into unexpected facets of cell biology and translate fundamental RNA insights into new therapeutic strategies.

Dr. Ryan Flynn will present the surprising new world of glycoRNAs, small noncoding RNAs bearing sialylated glycans and displayed on the cell surface. He will present chemical tools and conceptual insights into the molecular nature of glycoRNAs, highlighting their conservation across biological systems, and their implications for autoantibodies, immunity, and surface receptor biology.

Dr. Kristin Hope will discuss how RNA-binding proteins regulate normal and leukemic hematopoiesis and therapeutic resistance. She will describe emerging technologies for RNA binding protein-interactome mapping, and discuss how non-coding mutations in untranslated regions of mRNAs can re-wire post-transcriptional programs in leukemia, revealing novel avenues for therapeutic interception.

Prof. Konstantinos Tzelepis will present basic, translational, and clinical advances in targeting the epitranscriptome in myeloid and other malignancies. He will highlight RNA methylation as a driver of myeloid oncogenesis, discuss mRNA and tRNA methyltransferases as druggable vulnerabilities, and present clinical trial data testing RNA methyltransferase inhibition in humans.

Dr. Omar Abdel-Wahab will discuss novel technologies to target myeloid and lymphoid malignancies with splicing factor mutations. He will describe how stereotyped alterations in RNA splicing can be therapeutically exploited with synthetic RNAs that mimic aberrant splicing to drive mutation-dependent cellular toxins. He will also discuss efforts to create mRNA vaccines encoding RNA mis-splicing-derived neoantigens.

Co-Chairs:

Vikram Paralkar, MD
University of Pennsylvania Perelman School of Medicine
Philadelphia, PA

Julia Maxson, PhD
Oregon Health and Science University
Portland, 

Speakers:

Ryan A. Flynn, MD, PhD
Boston Children's Hopsital
Boston, MA
New Frontiers in RNA Biology

Kristin Hope, PhD
Princess Margaret Cancer Centre/University Health Network
Toronto, ON, Canada
RNA Binding Proteins in Hematopoiesis and Leukemia  

Konstantinos Tzelepis, PhD
University of Cambridge
Cambridge, United Kingdom
Targeting RNA Modifications in the Clinic

Omar Abdel-Wahab, MD
Memorial Sloan Kettering Cancer Center
New York, NY
Novel Uses of RNA as Therapeutics

back to top

JOINT SESSION - Bridging the Gap: Research and Clinical Implications of Emerging Technologies in Hematology

Scientific Committee on Hematopathology and Clin. Lab. Heme. + Scientific Committee on Lymphoid Neoplasia

This session highlights how emerging technologies are reshaping hematopathology and advancing precision diagnostics in hematologic malignancies. Together, the perspectives outlined by the speakers will illustrate how artificial intelligence (AI), spatial multi-omics, epigenetics, and genetics are driving translational innovation in hematopathology

Dr. Olga Pozdnyakova will discuss how the application of AI to flow cytometry data can enable automated detection and classification of hematological malignancies while also supporting the prediction of cytogenetic and molecular abnormalities, positioning flow cytometry as a potential early surrogate for genomic risk stratification.  

Dr. Mina Xu will explore advances in spatial transcriptomics and multi-omics, demonstrating how these tools reveal tumor architecture and microenvironmental features that can refine diagnosis and inform on risk stratification of hematologic malignancies. She will explain how spatial omics support the discovery of biomarkers, mechanisms of immune evasion, and precision therapeutics. 

Dr. Inaki Martin-Subero will focus on how epigenomic profiling enhances lymphoma classification and identifies clinically relevant molecular subtypes, bridging molecular biology with pathology practice. He will also explore how DNA methylation can be used to decode the life history of malignant cells in hematologic neoplasms that potentially can be used as powerful prognostic markers, risk stratification, and prediction of clinical outcome. 

Dr. David Scott will present, as proof of principle, how the knowledge gained from single cell profiling, spatial transcriptomics, whole genome sequencing and gene expression profiling have contributed to the understanding and classification of aggressive B-cell lymphomas. These technologies have helped to shape the evolutionary trajectories taken from common precursor cells to aggressive B-cell lymphoma. 

Co-Chairs:

Leticia Quintanilla-Fend, MD
University Hospital Tübingen
Tübingen, Germany

Ryan Morin, PhD
Simon Fraser University
Vancouver, BC, Canada

Speakers:

Olga Pozdnyakova, MD, PhD
Hospital of the University of Pennsylvania
Philadelphia, PA
New Developments in AI for Flow Cytometry Diagnostics

Mina L Xu, MD
Yale University School of Medicine
New Haven, CT
Spatial Transcriptomics/Multiomics in Lymphoma and Leukemia

Jose Martin-Subero, PhD
Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS)
Barcelona, Spain
Epigenome in the Classification of Lymphomas

David Scott, MD
BC Cancer
Vancouver, BC, Canada
New Dimensions in the Classification of Aggressive B-cell Lymphomas: What Are We Missing?

back to top

Through Space and Time: Spatiotemporal Organization of Haematopoiesis in Health and Disease

Scientific Committee on Hematopoiesis

Hematopoiesis has long been regarded as a bone-marrow centric, hierarchical system, in which heterotypic cell-cell interactions dictate qualitative and quantitative outputs of hematopoietic stem cell progenitor cells (HSPCs). Recent advances now challenge the concept of “one-niche-fits-it-all.” This session will explore how integrating spatially defined plasticity, chromosomal dosage and systemic feedback sets, reshape our understanding of niche biology, define compartmental resilience and open new avenues for targeting HSPC dysfunction in development, disease and aging.

Dr. Ralf Adams will discuss how vasculature represents a critical component of the bone marrow and is subjected to substantial changes during aging and in response to a broad range of pathophysiological challenges. In this context, his presentation will address the heterogeneity of the marrow compartment in different skeletal elements and the specialization of endothelial cells and vessel subtypes.

Dr. Ana Cvejic will present how inborn aneuploidies such as trisomy 18 and trisomy 21 deregulate hematopoiesis. By integrating single cell RNA-sequencing and multi-omic profiling of chromatin accessibility and gene expression, her team identified rewiring of distinct transcription factor networks through disrupted chromatin architecture. Thus, showing that aneuploidy perturbs gene regulation through both chromosome-specific and chromosome-independent mechanisms, shaping developmental and disease outcomes.

Dr. Kita Gritsman will discuss how total hematopoietic stem cell numbers are controlled in the body, including both systemic and local niche-mediated mechanisms of regulation. She will also discuss how the sensing of these signals by HSCs can inform quiescence, self-renewal, and lineage fate decisions.

Chair:

Ingo Ringshausen, MD
University College London
London, United Kingdom

Speakers:

Ralf Adams, DPhil
Max Planck Institute for Molecular Biomedicine
Muenster, Germany
Resilience and Dynamic Expansion and Resilience of Adult Calvarial Bone Marrow

Ana Cvejic, PhD
University of Copenhagen
Copenhagen, Denmark
Time, Place, and Destiny: Fetal Liver Niches Steering HSC Fate

Kira Gritsman, MD, PhD
Albert Einstein College of Medicine
Bronx, NY
Numbers and Fates: Decoding HSC Abundance and Decisions

back to top

Built to Clot, Built to Break: Structural Control of Hemostasis and Fibrinolysis

Scientific Committee on Hemostasis

Hemostasis and fibrinolysis are governed by highly coordinated molecular, structural, and biophysical mechanisms that ensure effective clot formation while preserving the capacity for clot dissolution. Advances in structural biology, biophysics, and molecular medicine have revealed that protein conformation, force-dependent interactions, and dynamic regulatory processes play central roles in controlling these pathways. This session will highlight cutting-edge research that moves beyond traditional physiological descriptions to examine the structural principles that govern coagulation and fibrinolysis. Through complementary perspectives spanning enzymatic modification, redox-dependent protein regulation, and clot architecture, the speakers will demonstrate how mechanistic insights are reshaping our understanding of hemostatic regulation and identifying new opportunities for therapeutic intervention.

Dr. Weikai Li will discuss the structural mechanisms underlying γ-carboxylation of vitamin K-dependent proteins involved in both hemostatic and non-hemostatic pathways. He will describe how propeptide recognition, conformational switching, and compartmentalized catalysis enable processive γ-carboxylation, and how distinct substrate-specific interactions influence modification efficiency and functional outcomes, including anticoagulation, bone formation, and angiogenic signaling pathways.

Dr. Phil Hogg will examine the role of disulfide bond status in shaping coagulation protein structure and regulating immune recognition, directly impacting our understanding of thrombotic disorders and highlighting structural principles informing protein-based therapeutics. Challenging the long-standing view of disulfide bonds as static features, he will present evidence that redox-dependent structural states influence autoantibody recognition in hemophilia A and antiphospholipid syndrome and may represent a broader mechanism governing immune responses to self-antigens.

Dr. Valerie Tutwiler will focus on the structural and mechanical determinants of clot stability and fibrinolysis. She will discuss how fibrin network, protofibril packing density, and mechanical tension regulate susceptibility to clot lysis, and how alterations in these properties contribute to disease and trauma. She will also highlight emerging approaches for biomarker discovery that leverage quantitative assessment of clot structure and mechanics.

Chair:

Mettine Bos, PhD
Leiden University Medical Center
Leiden, Netherlands

Speakers:

Weikai Li, PhD
Washington University in St. Louis
St. Louis, MO
Structural Insights into Mechanisms

Phil Hogg, PhD
Centenary Institute
Sydney, Australia
Novel Regulators of Hemostasis

Valerie Tutwiler, PhD
Rutgers University
Piscataway, NJ
Novel Regulators of Fibrinolysis

back to top

T Resident Memory Cells (Trm) in Health, Disease, and Therapeutics

Scientific Committee on Immunology and Host Defense

T Resident Memory (Trm) cells are increasingly recognized participants in tissue-localized inflammation in infections, tumor immunity, and organ and hematopoietic stem cell transplantation and may in fact play central roles in the more chronic pathology associated with these processes. The molecular and phenotypic definition of Trm and the mechanisms driving their persistence has proven challenging and will be a focus of this session. The speakers will also highlight recent cutting-edge research deciphering mechanisms by which Trm cells are sustained and/or drive chronic inflammation, emphasizing key pathways which could serve as therapeutic targets.

Dr. Jason Schenkel will discuss tissue residency of T cells in the context of infection and cancer. He will review what is known with respect to the ontogeny of tissue resident T cells, as well as the mechanisms that mediate residency. He will also discuss the difficulties of studying tissue resident T cells in vivo, and current methodologies that can be harnessed to study regionalized immunity in different disease contexts. He will also describe next generation therapies that aim to use tissue resident T cells for different disease contexts, including for cancer.

Dr. Nicholas Jarjour will discuss cytokine regulation of tissue-resident memory CD8+ T cells. Stability of Trm is dependent on local maintenance, supported by a remarkable capacity for self-renewal dependent on cytokines.  While the previously prevailing model highlighted exclusive roles for IL-7 and IL-15 in memory CD8+ T cell biology, Dr. Jarjour will describe how common gamma chain cytokines instead collaboratively govern the maintenance of Trm. Potential therapeutic implications of cytokine-based therapies on Trm will also be highlighted.

Dr. Defu Zeng will provide a comprehensive overview of the role of Trm cells in perpetuating pathogenesis of chronic graft-versus-host disease (cGVHD). He will review characteristic molecular and cellular features of Trm subsets in GVHD target tissues, including TCF1+CD69- stem-like (Tsm), TCF1+CD69+ tissue resident progenitor-like (Trmp), and TCF1-CD69+ TRM cells as well as regulation of TSM differentiation into Trmp and Trm.

Chair:

Asha Pillai, MD, PhD
Regeneron Therapeutics
Tarrytown, NY

Speakers:

Jason Schenkel, MD, PhD
MD Anderson Cancer Center
Houston, TX
Trm Phenotype and Functions

Nicholas Jarjour, PhD
Versiti Blood Research Institute
Milwaukee, WI
Molecular Mechanisms of Trm Maintenance, Activation, and Proliferation

Defu Zeng, MD, PhD
The Beckman Research Institute of City of Hope
Duarte, 
Coming to Trms with GVHD

back to top

Iron Homeostasis Across the Lifespan in Health and Disease

Scientific Committee on Iron and Heme

This session will explore the critical role of iron homeostasis across the lifespan, from fetal development to adult disease, highlighting how disruptions in iron regulation contribute to diverse pathological conditions. Through presentations spanning developmental biology, hematologic malignancies, and systemic metabolic regulation, speakers will examine the molecular pathways governing iron balance and the consequences of iron deficiency, overload, and dysregulated signaling. Together, these talks provide a comprehensive perspective on the mechanisms that maintain iron homeostasis and emerging therapeutic approaches to address iron-related disorders.

Dr. Veena Sangkhae will discuss how maternal iron status influences fetal development by regulating iron transport across the maternal–placental–fetal axis. She will describe how iron-dependent epigenetic enzymes regulate chromatin modifications essential for germ cell development, demonstrating how iron deficiency can disrupt these processes and alter sex determination. Dr. Sangkhae will also review mechanisms governing placental iron uptake and delivery to the fetus and how these processes are altered by iron deficiency, iron overload, and β-thalassemia. Findings from human studies and mouse models will illustrate the developmental consequences of abnormal maternal iron delivery.

Dr. Francesca Vinchi will examine the role of iron toxicity in myelodysplastic syndromes, focusing on mechanisms that drive iron overload through ineffective erythropoiesis and chronic transfusion therapy. She will explore how excess iron contributes to disease progression and transformation to acute myeloid leukemia, while highlighting emerging therapies that reduce iron burden, improve erythropoiesis, preserve hematopoietic stem cells, and enhance clinical outcomes.

Dr. Laura Silvestri will discuss the regulation of hepatic bone morphogenic protein (BMP)– small mother against decapentaplegic (SMAD) signaling and its control of hepcidin, the master regulator of systemic iron homeostasis. She will examine the role of TMPRSS6 in modulating this pathway and highlight emerging evidence linking BMP–SMAD signaling to hepatic metabolic reprogramming, revealing new therapeutic opportunities for metabolic disorders such as metabolic dysfunction–associated steatotic liver disease or metabolic dysfunction–associated steatohepatitis.

Chair:

Stefano Rivella, PhD
Children's Hospital of Philadelphia
Philadelphia, PA

Speakers:

Veena Sangkhae, PhD
UCLA David Geffen School of Medicine
Los Angeles, CA
Consequences of Maternal Abnormal Iron Delivery to the Fetus

Francesca Vinchi, PhD
Emory University School of Medicine
Atlanta, GA
The Role of Iron in Disease Progression

Laura Silvestri, PhD
San Raffaele Scientific Institute
Milan, Italy
Iron and Novel Pathologies: Beyond Erythropoiesis

back to top

Rethinking Antiplatelet Strategies: Selective Blockade, Broad-Acting Drugs, and Beyond

Scientific Committee on Megakaryocytes and Platelets

This session will highlight emerging approaches to improve the safety and efficacy of antithrombotic therapy by moving beyond conventional platelet inhibition. Current antiplatelet drugs effectively prevent thrombosis but often increase bleeding risk. The speakers will present complementary strategies that selectively target key signaling pathways driving thrombus propagation and pathological inflammation, with the goal of preserving hemostasis while preventing thrombosis. Together, these talks illustrate a shift toward precision modulation of platelet biology through innovative pharmacologic and molecular approaches, providing attendees with a conceptual framework for developing safer, next-generation antiplatelet therapies.

Dr. Barry Coller will discuss the development of zalunfiban, a novel αIIbβ3 integrin antagonist for the treatment of ST-segment elevation myocardial infarction. His presentation will highlight the clinical rationale, pharmacologic design based on crystal structure data to lock the receptor in its inactive conformation, and its potential to provide rapid platelet inhibition after subcutaneous administration in the ambulance. 

Dr. Xiaoping Du will present a strategy to inhibit integrin outside-in signaling. He will describe how targeting the Gα13–integrin interaction inhibits thrombosis while preserving hemostatic function. This approach not only reduces occlusive thrombosis with minimal bleeding risk but also attenuates thromboinflammatory responses and ischemia-reperfusion injury, demonstrating dual antiplatelet and anti-inflammatory potential. 

Dr. Ingerborg Hers will discuss the emerging use of proteolysis-targeting chimeras (PROTACs) to degrade key platelet signaling proteins. Focusing on BTK degraders and additional targets such as FAK, PYK2, and GSK3, she will illustrate how targeted protein degradation enables sustained suppression of thrombosis-relevant pathways without compromising hemostasis, offering a new paradigm for safer antithrombotic therapy. 

Chair:

Jaehyung Cho, PhD
Washington University School of Medicine
St. Louis, MO

Speakers:

Barry Coller, MD
Rockefeller University
New York, NY
Precision Antiplatelet Therapy: Targeting Platelet-Specific Pathways for Safer and Smarter Inhibition

Xiaoping Du, MD, PhD
University of Illinois College of Medicine
Chicago, IL
The Power of Broad Inhibition: Why Pan-Platelet Blockade Remains the Gold Standard

Ingeborg Hers, PhD
University of Bristol
Bristol, United Kingdom
Striking the Balance: Selectivity vs Breadth in the Next Generation of Antiplatelet Drugs

back to top

Innovations in the Detection and Characterization of Chemoresistant Cells in AML: From Molecular MRD to Molecular Resistance Mechanisms

Scientific Committee on Myeloid Neoplasia

Molecular measurable residual disease (MRD) quantification is increasingly being used for clinical decision-making for patients with acute myeloid leukemia (AML). However, we are still learning which patients and which timepoints are most appropriate for using molecular MRD results. Moreover, the technologies are still evolving, and what works best for one molecular target may not work well for another. At the translational level, understanding the biological features and vulnerabilities of the residual AML cells is of paramount importance for ultimately eradicating them. This session will review the latest in clinical applications, emerging technologies, and strategies for targeting molecular MRD in AML.

Dr. Chris Hourigan will begin with the increasingly important clinical context for molecular MRD testing in AML. International consensus evidence-based guidelines direct the use of the portfolio of complementary methods currently required for optimal AML MRD testing. He will discuss the importance of multi-center and multinational partnerships to generate robust evidence of clinical utility, including proof that intervention on AML MRD test results can meaningfully change clinical outcomes.

Dr. Felicitas Thol will discuss the current status of different molecular MRD detection methods, such as RT-PCR, digital droplet PCR, and next gen sequencing.  She will also describe novel technologies that are emerging for molecular MRD assessment, including the use of circulating AML-derived cell-free nucleic acids, and how they can be employed in clinical care of AML patients.

Dr. Jean-Emmanuel Sarry will discuss the metabolic adaptations of drug tolerant persister (DTP) cells that initiate relapses, such as reliance on oxidative phosphorylation, and whether these dependencies might be novel therapeutic targets in AML. Longitudinal single-cell multi-omics have revealed distinct metabolic states and heterogeneity of persistence mechanisms. He will highlight targetable metabolic, epigenetic and cell surface features of the high OxPHOS DTP phenotype, which may inform a more personalized therapeutic approach.

Chair:

Michele Redell, MD, PhD
Texas Children's Cancer and Hematology Center
Houston, TX

Speakers:

Christopher S Hourigan, MD, DM, DPhil
Virginia Tech FBRI Cancer Research Center
Washington, DC
Current Clinical Applications for Molecular MRD in Myeloid Malignancies

Felicitas R Thol, MD
Hannover Medical School
Hannover, Germany
Emerging Technologies for Molecular MRD Detection

Jean-Emmanuel Sarry, PhD
Cancer Research Center of Toulouse
Toulouse, France
Biological Insights into Resistant Cells

back to top

Back to the Future: Using Old and New Tools to Move the Field of Plasma Cell Research Forward

Scientific Committee on Plasma Cell Neoplasia

The last decade has brought impactful technological advancements to the medical sciences. From the detailed rendering of cell topology and transcriptional states offered by spatial transcriptomics, through the growing use of gene editing to study gene dependencies, to the use of artificial intelligence to predict cell-cell interactions, novel tools have been critical in deepening our understanding of cancer biology. It is therefore an opportune time to review how technological advancements and the masterful adaptation of well-established tools and models have been instrumental in advancing the field of myeloma pathobiology. Through this session, the audience will learn how classical approaches such as biochemistry and transgenic mouse models and cutting-edge multiomic techniques alike have informed discoveries about multiple myeloma intrinsic vulnerabilities, shed light on mechanisms of disease pathogenesis, and provided potential biomarkers of treatment response. Specifically:

Dr. Xin Gu will share her elegant biochemical and structural biology work that led to the identification of the midnolin-proteasome pathway as a ubiquitin-independent degradation mechanism of IRF4 and other transcription factors critical to multiple myeloma pathogenesis. Her findings suggest that midnolin is a novel, potentially targetable dependency in plasma cell neoplasia.

Dr. Marta Chesi will review the pivotal role of immunocompetent mouse models in the study of multiple myeloma pathobiology. She will then discuss her own seminal generation of mouse models engineered to recapitulate myeloma-bone marrow microenvironment interaction, thus enabling accurate, preclinical studies of novel immune and cellular therapies.

Dr. Mirco Friedrich will discuss how the integration of single-cell RNA and T-cell receptor sequencing with proteomic-based determination of antigen repertoires has led to the identification of a novel population of cytotoxic T cells in multiple myeloma. He will then share with us how these newly identified T cells are determinants of outcome in patients treated with immunotherapy.

Chair:

Giada Bianchi, MD
Dana-Farber Cancer Institute
Boston, MA

Speakers:

Xin Gu, PhD
Dana-Farber Cancer Institute, Harvard Medical School
Boston, MA
Show Me Your Face and I Will Understand Your Purpose: How Protein Structure Informs Function

Marta Chesi, PhD
Mayo Clinic Arizona
Scottsdale, AZ
Running on Wheels: How Mouse Models Inform the Study of Plasma Cell Biology

Mirco Julian Friedrich, MD, PhD
German Cancer Research Center (DKFZ)
Heidelberg, Germany
Getting by With a Little Help From Friends: Novel Approaches to Study Myeloma Microenvironment

back to top

Understanding and Targeting Metabolism in Erythrocyte Development and Disorders

Scientific Committee on Red Cell Biology

Red blood cell production is tightly regulated by metabolic pathways that control differentiation, proliferation, and survival of progenitor cells. Emerging evidence highlights the pivotal role of metabolic reprogramming in supporting the energy demands and biosynthetic processes required for red blood cell maturation. Disruptions in these metabolic pathways can impair erythropoiesis, contributing to the pathophysiology of red blood cell disorders such as thalassaemia and myelodysplastic syndromes. Understanding these metabolic programs offers new therapeutic opportunities. Targeting glucose, purine, pyrimidine, amino acid, and mitochondrial metabolism, may enhance erythroid differentiation and improve red blood cell production in disorders with defective erythropoiesis. The session will kick-off with an introduction of some key metabolic pathways that are critical for erythropoiesis. Specifically, the session Chair will highlight how many are expressed at higher levels in red blood cells than in other cell types due to the action of a few key transcription factors on erythroid-specific gene promoters and enhancers. 

Dr. Jian Xu will discuss the link between glutamine metabolism and heme biosynthetic pathways in terminal erythropoiesis. He will discuss the important role played by glutamine synthetase in detoxification of haem, and how this is derailed in beta-thalassemia. This new understanding could pave the way for novel therapies to mitigate ineffective erythropoiesis.

Dr. Marlies Rossmann will discuss unpublished work on the interplay between pyrimidine synthesis and ferroptosis in erythropoiesis. Ferroptosis is an iron-dependent lipid peroxidation process that leads to a unique form of cell death. It is a prevalent mechanism of ineffective erythropoiesis and red blood cell destruction in beta-thalassemia. Targeting ferroptosis could be of therapeutic benefit.

Dr. Lucia De Franceschi will discuss the critical role played by pyruvate kinase (PK) in red blood cell metabolism. Mature red blood cells lack mitochondria and thus are heavily dependent upon glycolysis for generation of ATP for their energy requirements. PK in the final enzyme in the glycolysis pathway, so red blood cells are particularly sensitive to the loss of PK enzymatic activity. ATP requirements are also higher when red blood cells experience oxidative stress, for example in thalassaemia. Dr Franceschi will summarize extensive clinical trial data of the PK allosteric agonist, mitapivat, in the treatment of PK deficiency and other red blood cell diseases.

Chair:

Andrew Perkins, MD, PhD, MBBS
Monash University
Melbourne, Victoria, Australia

Speakers:

Jian Xu, PhD
St. Jude Children's Research Hospital
Memphis, TN
Metabolic Switches Underpin Erythropoiesis

Marlies Petra Rossmann, MD, PhD
University of Rochester Medical Center
Rochester, 
The Interplay Between Pyrimidine Synthesis and Ferroptosis in Erythropoiesis

Lucia De Franceschi, MD
University of Verona
Verona, Italy
Developing Metabolism-Targeted Therapies in Red Cell Disorders

back to top

Balancing the Blood: Immune Control of Stem Cell Fidelity

Scientific Committee on Stem Cells and Regenerative Medicine

Maintaining a robust pool of hematopoietic stem cells (HSCs) is fundamental to sustaining blood production and overall health across the lifespan. This session delves into recent discoveries of the dynamic ways immunity influences HSC fitness—from prenatal development through aging. Immune signals begin shaping HSCs even before birth, as perinatal inflammatory stimuli imprint long-lasting effects on their postnatal function. Throughout life, innate and adaptive immune cells act as vigilant guardians, not only responding to inflammatory stress but also curbing the expansion of dysfunctional or premalignant HSCs. As the research into these connections is fairly nascent, the aim of the session is for the audience to gain an understanding of the intricate dialogue between the immune system and HSCs and how these interactions maintain blood system resilience and inform strategies for disease prevention and therapy. 

Dr. Anna Beaudin will discuss how distinct fetal hematopoietic stem and progenitor cell populations interpret inflammatory signals differently and how their prenatal immune experiences can leave lasting effects on the postnatal immune system, shaping future responses to infection and disease.

Dr. Masayuki Yamashita will discuss the role of adaptive immune cells in recognition and clearance of neoantigen-expressing mutant HSCs and the potential of this quality control mechanism to be exploited to prevent or eradicate clonal or premalignant hematopoiesis. 

Dr. Leila Perié will present new technology for in vivo cell division assessment that can help reveal the impact of intrinsic and extrinsic factors on healthy and malignant hematopoiesis and on cell-based therapies within distinct tissue microenvironments.

Chair:

Teresa Bowman, PhD
Albert Einstein College of Medicine
Bronx, NY

Speakers:

Anna Beaudin, PhD
University of Utah School of Medicine
Salt Lake City, UT
The Impact of Perinatal Inflammation on HSC Fitness

Masayuki Yamashita, MD, PhD
St. Jude Children’s Research Hospital
Memphis, TN
Regulation of HSCs by the Adaptive Immune System

Leila Perié, PhD
IIS Biogipuzkoa
San Sebastian, Spain
Regulation of HSCs by the Innate Immune System

back to top

How Clots Form and Fall Apart: From Engineered Vessels to Targeted Therapies

Scientific Committee on Thrombosis and Vascular Biology

This session will showcase technologies that capture complex interactions that influence the development of a thrombus. Understanding thrombosis requires dissecting the dynamic interplay between vessel wall, blood elements, mechanoforces, and biorheology. Vascular organoids and vessel-on-a-chip systems replicate physiological flow and shear conditions. This session will showcase state of the art microvascular and macrovascular thrombosis models side by side, because thrombotic diseases differ by location and mechanisms. Microvascular thrombosis drives conditions like sepsis and thrombotic microangiopathies, while macrovascular thrombosis underlies stroke and myocardial infarction. Attendees will learn how accurate models can enable disease-specific insights, inform targeted therapy development, and foster drug development. They will also learn how some of these new models could be leveraged to foster our understanding of vascular biology. 

Chair:

Vivien Chen, PhD, MBBS
The University of Sydney School of Medicine
Sydney, Australia

Speakers:

Wilbur A. Lam, MD, PhD
Emory University
Atlanta, GA
Vessel on a Chips, Organoids and Clots: A New Era in Thrombosis Research

Pierre Mangin, PhD
INSERM U1255
Strasbourg, France
Sticky Surfaces: Mechanistic Insights Into Macrovascular Clot Formation

Xiaowei Wang, PhD
Baker Heart and Diabetes Institute
Melbourne, Australia
Beyond Platelets and Coagulation: Novel Anti-Thrombotic Targets

back to top

Cutting-Edge Insights into Pro-Thrombotic Anti-Platelet Factor 4-Associated Disorders

Scientific Committee on Transfusion Medicine

This session will unify very novel and timely developments in pro-thrombotic anti-platelet factor 4 (PF4)-associated disorders. Monoclonal Gammopathy of Thrombotic Significance (MGTS) is a recently identified thrombotic entity characterized by the production of persistent, monoclonal anti-PF4. Vaccine-induced thrombotic thrombocytopenia (VITT) is an anti-PF4 mediated condition that was discovered after the implementation of COVID-19 adenovirus vector vaccines, and is characterized by thrombocytopenia and/or arterial and venous thrombosis, also at unusual sites such as cerebral venous sinus thrombosis. Heparin-induced thrombocytopenia (HIT) is an anti-PF4/heparin-mediated condition due to heparin exposure, for instance during cardiac surgery, and is also characterized by the presence of thrombocytopenia and thrombosis. This session will reveal striking similarities and differences between these disorders, with a focus on immunopathogenesis, diagnostics and management, aiding our understanding of these complex anti-PF4 mediated thromboinflammatory responses.

Dr. Anand Padmanabhan will discuss the recent developments of MGTS. Serological heterogeneity of  anti-PF4 and its contribution to diagnostic challenges will be discussed. Dr. Padmanabhan will also detail emerging treatment approaches, including drugs of various classes: anticoagulants, antiplatelet agents and plasma cell directed therapies.

Dr. Jing Jing Wang will discuss recent advances in understanding the pathogenesis of VITT. Using antibody proteomics, her work identified highly stereotyped anti-PF4 responses shared between VITT and VITT-like syndromes following natural adenoviral infection, implicating a common adenoviral trigger. She will present evidence identifying adenoviral protein VII as the inciting antigen through molecular mimicry with PF4, and discuss how somatic hypermutation redirects antiviral antibodies toward pathogenic PF4 recognition. The implications for autoimmune antibody evolution and safer adenoviral vector design will also be discussed.

Dr. Lubica Rauova will discuss the pathogenesis of HIT, focusing on the role of heparin and its interaction with PF4 in triggering pathogenic antibody formation that leads to thrombocytopenia and thrombosis.

Chair:

Rick Kapur, MD, PhD, MSc
Amsterdam University Medical Center Location University of Amsterdam
Amsterdam, Netherlands

Speakers:

Anand Padmanabhan, PhD, MBBS
Mayo Clinic
Phoenix, AZ
The Clone Wars: Monoclonal Gammopathy of Thrombotic Significance (MGTS)

Jing Jing Wang, PhD
College of Medicine and Public Health, Flinders Health and Medical Research Institute, Flinders University,
Adelaide, Australia
Pulling the Antibody Trigger in Vaccine-Induced Thrombotic Thrombocytopenia (VITT)

Lubica Rauova, MD, PhD
The Childrens Hospital of Philadelphia
Philadelphia, PA
Heparin-Induced Thrombocytopenia (HIT): How Heparin Sparks Thrombosis

back to top

Power Sharing: Transplantation and Cellular Therapy in Autoimmunity

Scientific Committee on Transp Biology and Cellular Therapies

Hematologists and hematology researchers have been at the forefront of the pre-clinical development, manufacture and clinical application of cellular therapies, and in particular, redirected chimeric antigen receptor cytotoxic therapies in the treatment of malignancy. The powerful and targetable manipulation of immune effector function, and in particular their ability to profoundly deplete B lymphocytes, has now captured the imagination of clinicians and researchers who treat patients with immune dysfunction including auto-immunity. The power of immune therapies is now shared across a range of connective tissue disorders and immune driven pathologies including rheumatoid arthritis, systemic lupus, multiple sclerosis and auto-immune nephritides. Hematologists, hematology scientists, transplant team members and cellular therapy logistic experts will all be increasingly involved in the delivery of these therapies in a widening group of indications and in patients with a range of prior therapies and disease-specific comorbidities. In this session we will learn of cutting-edge developments in the application of cellular therapies in these disorders. Overall, this session will explore the scientific rationale for cellular therapy, the factors that determine case selection, the biomarkers of response and factors that determine on-target or off-target toxicity

Dr. Maria Raimondo will discuss the mechanistic and long-term immune consequences of B-cell depletion strategies using T cell redirection in rheumatology including the possible future application of in vivo CAR-T cell strategies.

Dr. Huji Xu will then outline the biology and therapeutic application of cell products other than T cells, including natural killer cells, which may provide potent B cell control without the risks of long-lived T cell activation in the treatment of autoimmune conditions. Whilst the emphasis to date on immune manipulation has concentrated on autoreactive B cell depletion, promotion.

Dr. Jeffrey Bluestone will outline the preclinical and clinical findings of using CAR- redirected regulatory T cell populations to reestablish immune tolerance in rheumatoid arthritis.

Chair:

David Ritchie, MD, PhD
Royal Melbourne Hospital
Melbourne, Australia

Speakers:

Maria Gabriella Raimondo, MD
Department of Medicine 3 - Rheumatology and Immunology, Friedrich-Alexander-University Erlangen-Nuremberg and Uniklinikum Erlangen
Erlangen, Germany
Ex Vivo or in Vivo CAR-T for Autoimmunity

Huji Xu, MD
Tsinghua University School of Medicine
Beijing, China
Induced to Kill; Alternative Sources of Cells for the Therapy of Autoimmunity

Jeffery Bluestone, PhD
University of California San Francisco
San Francisco, CA
Promoting the Peacekeepers: Treg Adoptive Therapies to Re-Establish Tolerance