2016 Scientific Program

The 2016 Scientific Committee Sessions will be held Saturday, December 3, and Sunday, December 4. Each session will be offered twice. A question-and-answer period will occur at the end of each individual speaker presentation. Invited abstracts of these sessions will be published in the Program Book and on the flash drive containing the annual meeting abstracts.

All Scientific Program sessions will be recorded and made available through ASH On Demandafter the meeting.

Robert Brodsky, MD The Johns Hopkins University School of Medicine Baltimore, MD

Ross Levine, MD Memorial Sloan-Kettering Cancer Center New York, NY

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(Select) Ad Hoc Scientific Committee on Epigenetics and Genomics Joint Session: Scientific Committee on Blood Disorders in Childhood and Scientific Committee on Red Cell Biology Joint Session: Scientific Committee on Hematopoiesis and Scientific Committee on Myeloid Biology Scientific Committee on Bone Marrow Failure Scientific Committee on Hematopathology and Clinical Laboratory Hematology Scientific Committee on Hemostasis Scientific Committee on Immunology and Host Defense Scientific Committee on Iron and Heme Scientific Committee on Lymphoid Neoplasia Scientific Committee on Myeloid Neoplasia Scientific Committee on Plasma Cell Neoplasia Scientific Committee on Platelets Scientific Committee on Stem Cells and Regenerative Medicine Scientific Committee on Thrombosis and Vascular Biology Scientific Committee on Transfusion Medicine Scientific Committee on Transplantation Biology and Cellular Therapies

Enhancers and Chromatin Landscapes in Development and Cancer

Dr. Majeti will focus on chromatin accessibility patterns during normal humanhematopoiesis and AML evolution from pre-leukemic HSCs with a detailed discussion ofcohesin complex mutants.

Dr. Aifantis will focus on how higher order chromosomal structure is altered in leukemia and how key regulators of this process are involved in hematopoietic function and gene expression.

Dr. Ren will present data on state-of-the-art approaches to map human genomic architecture and how this process is altered during malignant transformation.

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AshAlizadeh,MD, PhD Stanford University Stanford,CA

BingRen,PhD University of San Diego La Jolla,CA Organization and Regulation of the Human Genome

IannisAifantis,PhD New York University New York,NY The Impact of 3D Chromosomal Topology in Acute Leukemia

RaviMajeti,MD, PhD Stanford University Stanford,CA Chromatin Accessibility Charts Human Hematopoiesis and Acute Myeloid Leukemia Evolution

Understanding and Repairing Faulty Red Blood Cells

Dr. Dean will focus on the biology of enhancers, gene regulatory elements that regulate transcription through long-range interactions with promoter regions and have highly tissue-specific functions, including in erythroid cells, as well as roles in promoting pathologic gene expression in disease states.

Dr. Lodish will present ongoing work focused on harnessing an integrative, mechanistic understanding of erythroid progenitor cell signaling pathways that control self-renewing cell divisions to envision novel therapies for anemias.

Dr. De Franceschi will describe the development of non-gene therapy strategies for clinical application, including approaches currently under clinical evaluation.

Dr. Cavazzana will present novel therapeutic approaches in an effort to cure the more prevalent inherited blood diseases worldwide. Results of ongoing clinical trials as well as of promising gene editing strategies will be summarized.

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ColleenDelaney,MD, MSc Fred Hutchinson Cancer Research Center Seattle,WA

Alex C.Minella,MD BloodCenter of Wisconsin Milwaukee,WI

AnnDean,PhD National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health Bethesda,MD New Concepts in Genome Regulation

HarveyLodish,PhD Whitehead Institute for Biomedical Research Cambridge,MA PPARa Agonists and TGF Inhibitors Stimulate Red Blood Cell Production by Enhancing Self-Renewal of BFU-E Erythroid Progenitors

LuciaDe Franceschi,MD University of Verona Verona,Italy New Therapeutic Options: Alternates to Gene Therapy for Treating Hemoglobinopathies

MarinaCavazzana,MD, PhD Hpital Necker Enfants Malades Paris,France Gene Therapy Studies in Hemoglobinopathies: Successes and Challenges

Clonal Development of Hematopoietic Stem Cell Specification and Differentiation at Single Cell Resolution

Dr. Dick will describe clonal evolution of human hematopoiesis at single cell resolution.

Dr. Gottgens will present single cell molecular profiling experiments that reveal new aspects of blood stem cell regulation and their perturbation by leukemic factors.

Dr. Rothenberg will present a systems biology level understanding of the transcription networks that control lymphoid cell fate decisions.

Dr. Schroeder will present his work using transcription factor reporters to track myeloid lineage fate determination, and the instructive role of niche and environmental factors.

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YiZheng,PhD Cincinnati Children's Hospital Cincinnati,OH

H. LeightonGrimes,PhD Cincinnati Children's Hospital Cincinnati,OH

John E.Dick,PhD University Health Network Toronto,ON,Canada Molecular Events Defining Human Clonal Hematopoiesis at Single Cell Resolution

BertieGottgens,DPhil University of Cambridge Cambridge,United Kingdom Defining Cellular States, Differentiation Trajectories, and Regulatory Networks Through Single Cell Profiling

EllenRothenberg,PhD California Institute of Technology Pasadena,CA Transcription Factor Gene Fluorescent Reporters Track Lineage Fate in Lymphoid Commitment

TimmSchroeder,PhD ETH Zurich Basel,Switzerland Long-term Live Single Cell Quantification of Transcription Factor Dynamics

Ribosomes and Ribosomopathies

Dr. Barna will introduce the concept that not all ribosomes are created equal, and that translation by specialized ribosomes represents a separate layer of gene regulation that determines which mRNAs are effectively translated. Her work provides insights into how mutations in different ribosome proteins lead to a diverse spectrum of clinical features.*Please note that Dr. Barna will only be speaking at the Saturday session.*

Dr. Warren will provide structural insights into the mechanism by which mutations that cause Schwachman-Diamond anemia affect ribosome assembly.

Dr. Zon will describe a zebrafish model of Diamond Blackfan Anemia, and how chemical suppressor screens may lead to the discovery of novel therapeutics to ameliorate clinical aspects of the syndrome.

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NancySpeck,PhD University of Pennsylvania Perelman School of Medicine Philadelphia,PA

MariaBarna,PhD Stanford University Stanford,CA Specialized Ribosomes: A New Frontier in Gene Regulation, Organismal Biology, & Evolution

Alan J.Warren,MD, PhD University of Cambridge Cambridge,United Kingdom Shwachman-Diamond Syndrome and the Quality Control of Ribosome Assembly

Leonard I.Zon,MD Harvard Medical School, Boston Childrens Hospital Cambridge,MA Modeling Diamond Blackfan Anemia and Developing Therapeutics

Minimal Residual Disease in Hematology: Why, When, and How?

Dr. Wood will describe the key immunophenotypic principles that underlie minimal residual disease detection by flow cytometry and illustrate their application and clinical significance to the monitoring of acute leukemia.

Dr. Valk will discuss minimal residual disease detection in acute myeloid leukemia by means of polymerase chain reaction approaches using the multitude of available molecular markers in the context of clonal hematopoiesis.

Dr. Druley will focus on current strategies for using RNA sequencing as a modality for minimal residual disease detection. As we now move into the era of single-cell transcriptomes and error-corrected sequencing, we may move beyond simple quantitation of chromosomal rearrangements to identify also allele- and transcript-specific profiles of cancer cells as a tool for diagnostics, therapy and mechanistic understanding.

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TorstenHaferlach,MD MLL Munich Leukemia Laboratory Munich,Germany

BrentWood,MD, PhD Seattle Cancer Care Alliance Seattle,WA Multiparameter Flow Cytometry as a Powerful Tool

PeterValk,PhD Erasmus University Medical Center Rotterdam,Netherlands Molecular Minimal Residual Disease Detection in Acute Myeloid Leukemia

Todd E.Druley,MD, PhD Washington University School of Medicine in St. Louis St. Louis,MO Novel Technologies to Detect Minimal Residual Disease

Emerging Therapeutics to Alter Hemostasis and Thrombosis

Dr. Lenting will describe studies of the molecular interactions between factor VIII and von Willebrand factor. Detailed understanding of these interactions has recently been uncovered and can be used for development of improved long-acting factor VIII replacement therapies for treatment of hemophilia A.

Dr. Arruda will describe the discovery and biochemical characterization of factor IX Padua and translational studies. This form of factor IX has enhanced procoagulant activity and is being advanced into gene therapy trials for treatment of hemophilia B.

Dr. Coughlin will describe the discovery and characterization of protease-activated receptors (PARs). The work explained how the coagulation protease thrombin activates platelets and other cells and led to the development of the platelet inhibitory drug, vorapaxar. A crystal structure of a PAR-vorapaxar complex has helped to explain properties of the drug.

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AlanMast,MD, PhD BloodCenter of Wisconsin Milwaukee,WI

Peter JLenting,PhD French Institute of Health and Medical Research (INSERM) Le Kremlin-Bictre,France Von Willebrand Factor Interaction with FVIII: Development of Long Acting FVIII Therapies

ValderArruda,MD, PhD The Children's Hospital of Philadelphia, University of Pennsylvania Philadelphia,PA Factor IX Padua: From Biochemistry to Gene Therapy

ShaunCoughlin,MD, PhD University of California San Francisco San Francisco,CA PAR1 Antagonists Development and Clinical Utility

Innate Immunity: The Green Light to Adaptive Responses

Dr. Trinchieri will discuss the role of inflammation, innate resistance and commensal microbiota in carcinogenesis, cancer progression and cancer therapy.

Dr. Gajewski will discuss innate immune sensing of cancer via the host Stimulator of INterferon Genes (STING) pathway and how this presents therapeutic opportunities to activate effective anti-tumor immunity.

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StanleyRiddell,MD Fred Hutchinson Cancer Research Center Seattle,WA

GiorgioTrinchieri,MD National Cancer Institute, National Institutes of Health Bethesda, Innate Immune Signaling in Regulation of Immunity

ThomasGajewski,MD, PhD University of Chicago Medical Center Chicago,IL Innate Immune Sensing in Anti-Tumor Immunity and Cancer Immunotherapy

From Iron Trafficking to Iron Traffic Jam

Dr. Carlomagno will discuss recent findings on the importance of ferritinophagy to maintain iron homeostasis in vivo. She will also present new data on the role of iron in regulating cell cycle progression and genome stability

Dr. Lakhal-Littleton will discuss the studies in tissue-specific and global hepcidin/ferroportin gene knockouts;offering insight into the interplay between cellular and systemic mechanisms in the regulation of iron levels in the heart and in its healthy functioning. Her studies raise the possibility that the hepcidin/ferroportin axis may also be important in other hepcidin and ferroportin-expressing tissues such as the kidney, the brain and the placenta.

Dr. Knutson will present recent insights from studies of knockout mouse models that aim to identify how various cells and organs, including the heart, take up non-transferrin-bound iron.

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Maria DomenicaCappellini,MD University of Milan - Fondazione iRCCS Ca' Granda Ospedale Policlinico Milan Milan,Italy

FrancescaCarlomagno,MD, PhD Federico II University of Naples Naples,Italy Ferritinophagy and Cell Cycle Control

SamiraLakhal-Littleton,DPhil University of Oxford Oxford,United Kingdom Ferroportin Mediated Control of Iron Metabolism and Disease

MitchellKnutson,PhD University of Florida Gainesville,FL Non-Transferrin-Mediated Iron Delivery

Emerging Biology Leading to New Therapies in Follicular Lymphoma

Dr. Pasqualucci will introduce general concepts about the cell of origin in follicular lymphoma and the mechanisms associated with clonal evolution. She will then examine the genetic events that take place early in the history of the tumor clone and focus on the role of histone/chromatin modifier genes, including the methyltransferase KMT2D and the acetyltransferases CREBBP/EP300, in the stepwise progression of the disease from a subclinical state to a pathological entity.

Dr. Fitzgibbon will provide an introduction to genomic discovery in follicular lymphoma. He will review the next generation sequencing tools that are being used to identify genetic predisposition factors and to perform molecular profiling to identify signaling mutations that may be targeted therapeutically and which provide insights into disease prognosis.

Dr. Smith will focus her discussion on emerging new treatment approaches for follicular lymphoma based on the novel concepts and new targets described by Drs. Nadel and Fitzgibbon. She will focus on the disease heterogeneity and prognosis, the clinical unmet needs, and how clinical integration of the new molecular tools is leading to an evolution in the therapeutic regimens for patients with follicular lymphoma.

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WendyStock,MD The University of Chicago Chicago,IL

LauraPasqualucci,MD Columbia University New York City,NY Genetic-Driven Disruption of Epigenetic Circuits As Early Steps In The Pathogenesis Of Follicular Lymphoma

JudeFitzgibbon,PhD Queen Mary University of London London,United Kingdom Genomic Discovery, Prognosis, and Target Therapy Development

Sonali M.Smith,MD The University of Chicago Medicine Chicago,IL Follicular Lymphoma Therapy Based on Biological Insights and Novel Concepts

Focusing on Myeloid Neoplasia Through Splicing

Dr. Krainer will update the audience on the spliceosoma complex and splicing machinery. He will discuss functional implications in normal and pathological conditions.

Dr. Halene will focus on the pathogenetic mechanisms underlying specific mutations in MDS.

Dr. Walter will discuss the clinical implications of spliceosome gene mutations in MDS, their distribution in diverse subgroups and their prognostic significance. He will explore novel therapeutic approachesbased on use of drugs that modulate splicing to treat spliceosome mutant MDS.

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CristinaMecucci,MD, PhD University of Perugia Perugia,Italy

AdrianKrainer,PhD Cold Spring Harbor Laboratory Cold Spring Harbor,NY Spliceosome: Physiology and Disease Pathogenesis

StephanieHalene,MD, PhD Yale University School of Medicine New Haven,CT Functional Consequences of Spliceosome Mutations

Matthew J.Walter,MD Washington University in St. Louis St. Louis,MO Clinical Implications of Spliceosome Mutations: Epidemiology, Clonal Hematopoiesis, and Potential Therapeutic Strategies

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2016 Scientific Program