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Arthur W. Nienhuis Research Symposium

March 20, 2024
8:00 am-4:45 pm

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This symposium honors the contributions of Dr. Arthur W. Nienhuis, former CEO of St. Jude and a forerunner in the field of hematopoiesis, hematopoietic stem cell transplantation and gene therapy. Former trainees and colleagues of Dr. Nienhuis, many of whom are imminent researchers in the field of gene therapy, will discuss the latest research on emerging therapies for sickle cell disease, as well as for other disorders.

 
 

Agenda

  1. Time Event
    7:30-8:00 am  Check-in and registration
    8:00–8:10 am

    Conference Opening

     

    Conference Hosts: Mitchell Weiss, MD, PhD and Andrew Davidoff, MD, St. Jude Children’s Research Hospital

    8:10–8:30 am

    Opening Remarks

     

    James Downing, MD

    President and CEO, St. Jude Children’s Research Hospital

    8:30–8:40 am Remarks from Doris Duke Charitable Foundation
    8:40–9:00 am

    Cynthia Dunbar

    Distinguished Investigator, National Institutes of Health (NIH)

    Chief, Translational Stem Cell Biology Branch, and Head, Molecular Hematopoiesis Section, National Heart, Lung, and Blood Institute, NIH

    9:00–9:20 am

    Edward Benz, MD

    President and CEO Emeritus, Dana-Farber Cancer Institute 

    Richard and Susan Smith Distinguished Professor of Medicine, Pediatrics and Genetics at HMS and Executive Director of the Cure Sickle Cell Initiative, NIH

    9:20–9:50 am

    David Bodine, PhD

    Chief of the Genetics and Molecular Biology Branch and Head of the Hematopoiesis Section, National Human Genome Research Institute

    9:50–10:10 am

    Amit Nathwani, MD, PhD

    Senior NIHR Investigator, and Professor of Haematology, University College London

    10:10–10:20 am

    David Nathan, MD

    President Emeritus, Dana-Farber Cancer Institute

    Physician-in-Chief Emeritus, Boston Children’s Hospital

    Professor of Pediatrics and Medicine, Harvard Medical School

    10:20–10:40 am Break
    11:00–11:20 am

    Stefan Karlsson, MD, PhD

    Emeritus Professor of Molecular Medicine and Gene Therapy, Lund Stem Cell Center, Department of Laboratory Medicine, Lund University

    11:50 am–12:10 pm

    Stuart Orkin, MD

    Harvard Stem Cell Institute

    David G. Nathan Distinguished Professor of Pediatrics Harvard Medical School

    HHMI Investigator, Boston Children’s Hospital

    12:10–12:30 pm

    Griffin Rodgers, MD, MACP

    Director, National Institute of Diabetes and Digestive and Kidney Diseases

    12:30–1:30 pm Lunch
    1:35–1:55 pm

    Robert Brodsky, MD

    Johns Hopkins Family Professor of Medicine and Oncology, Director, Division of Hematology, Johns Hopkins University School of Medicine

    1:55–2:15 pm

    Timothy Ley, MD

    Lewis T. and Rosalind B. Apple Chair in Oncology, Professor of Medicine and Genetics, Director of the Stem Cell Biology Section, Division of Oncology, Co-Director of the Oliver Langenberg Physician Scientist Training Program (Department of Medicine), Washington University School of Medicine

    2:15–2:35 pm

    Donald Kohn, MD

    Distinguished Professor, Microbiology, Immunology and Moleculary Genetics; Pediatrics (Hematology/Oncology); Molecular and Medical Pharmacology, UCLA Broad Stem Cell Research Center

    2:35–2:45 pm

    Luigi Naldini, MD

    Director, San Raffaele Telethon Institute for Gene Therapy

    Full Professor,  San Raffaele University

    2:45–2:55 pm

    Katherine High, MD

    Visiting Professor, Rockefeller University

    Professor, Pediatrics Emerita, Perelman School of Medicine

    Co-Founder, Former President and Head of R&D, Spark Therapeutics

    2:55–3:15 pm Break
    3:20–3:40 pm

    Alan Schechter, MD

    NIH

    3:40–4:00 pm

    John A. Stamatoyannopoulos, MD

    Director, Altius Institute for Biomedical Sciences

    4:00–4:20 pm

    John Tisdale, MD, PhD

    Senior Investigator, Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, National Institutes of Health

    4:20–4:30 pm

    Douglas Higgs, MD

    Emeritus Professor, Weatherhall Institute of Molecular Medicine 

    4:30–4:45 pm Conference Closing
    4:45–5:45 pm Cocktail Hour
 

Speakers

  1. Edward Benz

    Edward J. Benz, Jr. MD 

    President and CEO Emeritus, Dana-Farber Cancer Institute, Richard and Susan Smith Distinguished Professor of Medicine, Pediatrics and Genetics at HMS and Executive Director of the Cure Sickle Cell Initiative at NIH 

    Edward Benz is a member of the National Academy of Medicine and Fellow of the American Academy of Arts and Sciences, Phi Beta Kappa, Alpha Omega Alpha and Past President of the American Society of Hematology, the American Society of Clinical Investigation, and the Association of American Cancer Institutes. A hematologist with special expertise in complex and inherited anemias, he has made major contributions to the molecular pathobiology of hemoglobinopathies and disorders of the red cell membrane. He has been internationally recognized for his commitment to mentoring, creating a nurturing working environment, and advancing the careers of women and minority faculty, learners and staff.

  2. David Bodine

    David Bodine, PhD

    Chief of the Genetics and Molecular Biology Branch and Head of the Hematopoiesis Section at the National Human Genome Research Institute

    David Bodine received his undergraduate degree from Colby College in 1976, a master's degree in Human Genetics from Rutgers in 1977 and a Ph.D. for his work at the Jackson Laboratory through the University of Maine in 1984. After postdoctoral work at NIH's National Heart, Lung and Blood Institute with Dr. Arthur Nienhuis, Dr. Bodine founded the Hematopoiesis Section in the newly formed Intramural Research Program of NIH's National Human Genome Research Institute (NHGRI) in 1993. In 1995, Dr. Bodine was promoted to Senior Investigator with tenure at NHGRI, and in 2006 was named chief of NHGRI's Genetics and Molecular Biology Branch.

    Dr. Bodine has won numerous awards during his career. As an undergraduate, he received the Webster Chester Biology prize and was awarded an honorary degree from Colby College in 2013. Dr. Bodine received postdoctoral fellowships from the NIH and the Cooley's Anemia Foundation. Having benefited from outstanding mentoring during his career, Dr. Bodine consequently has made mentoring his trainees a priority. In recognition of these efforts, Dr. Bodine was named 2004 NIH Mentor of the Year, and he has been named NHGRI Mentor of the Year three times, most recently in 2012. Nationally, Dr. Bodine has served as the president of the American Society of Gene Therapy, Counselor to the American Society of Hematology, and Associate Editor for the journal Blood

  3. Robert Brodsky

    Robert A. Brodsky, MD

    Johns Hopkins Family Professor of Medicine and Oncology, Director, Division of Hematology, Johns Hopkins University School of Medicine

    Robert Brodsky is the Johns Hopkins Family Professor of Medicine and Oncology, and a member of the Johns Hopkins Kimmel Cancer Center.  He also serves as the Director of the Division of Hematology and the T32 Training Program.

    Dr. Brodsky received his medical degree from Hahnemann University.  He completed his residency in Internal Medicine at Vanderbilt University School of Medicine and his fellowship training in hematology at the National Institutes of Health and in oncology at Johns Hopkins University. 

    Dr. Brodsky’s clinical and academic interests relate to bone marrow failure disorders, hemolytic anemias, and complement.  He and his colleagues pioneered the use of high-dose cyclophosphamide for treating autoimmunity and alloimmunity.  He has a special interest in using post-transplant cyclophosphamide to prevent graft-versus-host disease and to expand the donor pool to include HLA-haploindentical donors for aplastic anemia and severe hemoglobinopathies.

    Dr. Brodsky has been recognized with numerous honors.  In 2018, he received The Lower Merion/Harriton Alumni Association Distinguished Alumni Award.  In 2013, received the Clinical Research Achievement Award from the Clinical Research Forum for his work in sickle cell disease.  He was a Clinical Research Scholar of the Leukemia and Lymphoma Society of America in 2000.  He is on the editorial board for Blood, is a Section Editor for UpToDate. He is on the Executive Committee of the American Society of Hematology (ASH) and serves as President for ASH. 

  4. Cynthia Dunbar

    Cynthia “Cindy” E. Dunbar, MD

    NIH Distinguished Investigator; Chief, Translational Stem Cell Biology Branch, and Head, Molecular Hematopoiesis Section, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

    Cynthia Dunbar has pursued a career encompassing clinical investigation, laboratory science, and education/administration.  As a translational research scientist, she has made important findings in the areas of hematopoiesis, stem cell biology, leukemogenesis, natural killer cell biology, and gene therapies, focusing on non-human primate models to provide insights not possible from small animal or n vitro studies. She has led landmark clinical trials in gene therapy, autoimmune disease, and bone marrow failure, most recently resulting in FDA approval for a new treatment for aplastic anemia She graduated from Harvard College with a degree in History of Science and received her MD from Harvard Medical School. Following internal medicine training at Boston City Hospital, she came to the NIH as a postdoctoral research fellow in the laboratory of Dr. Arthur Nienhuis, followed by clinical training in hematology at the University of California, San Francisco. In 1991 she returned to the intramural program of the National Heart, Lung, and Blood Institute to set up her own research group. She has published over 290 articles in peer-reviewed journals. She was elected to the American Society for Clinical Investigation, the American College of Physicians, the National Academy of Medicine, and as a Fellow of the AAAS. She has filled leadership roles with major professional societies, including as current Secretary of the American Society of Hematology, and as past President of the American Society for Cell and Gene Therapy. She served as Editor-in-Chief of Blood, the premier hematology journal worldwide, from 2007-2012, the first woman to serve in this position. She has shown great commitment to education and career development of physician-scientists, leading the NIH hematology fellowship program for 17 years, and receiving numerous mentoring awards from NHLBI, NIH and her professional societies. She has worked to enhance the research environment and all aspects of diversity within the NIH intramural program and in biomedicine more generally through her service as founding co-chair and current officer of the NIH Assembly of Scientists and founding member of the NIH Equity Committee.  Outside of work, she lives in downtown DC and is an avid choral musician and spends as much time outdoors as possible in Rock Creek Park and in the Adirondack mountains.

  5. Information to come

  6. Katherine High

    Katherine High, MD

    Katherine High is an internist and hematologist by training. She began her career studying the molecular basis of blood coagulation and the development of novel therapeutics for the treatment of bleeding disorders.  Her pioneering bench-to-bedside studies of gene therapy for hemophilia led to a series of studies that characterized the human immune response to gene delivery vectors. Her work evolved to encompass clinical translation of genetic therapies for multiple inherited disorders, and as the Founding Director of the Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia, Dr. High assembled a multidisciplinary team of scientists and researchers working to discover new gene and cell therapies for genetic diseases and to facilitate rapid translation of preclinical discoveries into clinical application. At CHOP and Penn, Dr. High was an Investigator of the Howard Hughes Medical Institute and held an endowed chair at the medical school.  In 2013, Spark Therapeutics, a biotechnology company based in Philadelphia, was formed based on programs that Dr. High had led at Children’s Hospital and in 2014, she joined Spark full-time as President and Head of R&D; in 2019, Spark was acquired by Roche. While at Spark, she led the team that achieved the first FDA approval of a gene therapy for a genetic disease in the US, for a rare form of congenital blindness, and led the development of a gene therapy for hemophilia B that has now completed Phase 3 testing. Dr. High served a five-year term on the FDA Advisory Committee on Cell, Tissue and Gene Therapies and is a past-president of the American Society of Gene & Cell Therapy (ASGCT). She received her A.B. in chemistry from Harvard University, an M.D. from the University of North Carolina School of Medicine, and business certification from the UNC Business School Management Institute for Hospital Administrators. She currently serves on the Board of Directors of CRISPR Therapeutics (NASDAQ: CRSP), and Incyte Corporation (NASDAQ:INCY), and on the Board of Trustees of the College of Physicians of Philadelphia. She is the recipient of many honors and awards, is the author of more than 200 scientific papers, and the holder of multiple issued patents on gene therapy. She is an elected member of the National Academy of Medicine (US), the American Academy of Arts and Sciences, the Faculty of Pharmaceutical Medicine of the Royal College of Physicians (London), and the National Academy of Sciences (US). She is currently a Visiting Professor at Rockefeller University in New York. 

  7. Stefan Karlsson

    Stefan Karlsson, MD, PhD

    Emeritus Professor of Molecular Medicine and Gene Therapy at the Lund Stem Cell Center, Department of Laboratory Medicine, Lund University

    Stefan Karlsson is Emeritus Professor of Molecular Medicine and Gene Therapy at the Lund Stem Cell Center, in the Department of Laboratory Medicine, Lund University, Sweden. He is recognized for significant contributions to the fields of gene therapy and hematopoietic stem cell biology and in 2009 was awarded the Tobias Prize by The Royal Swedish Academy of Sciences.

    Dr. Karlsson's field of research is the regulation of hematopoietic stem cells and development of stem cell expansion protocols and gene therapy. He performed postdoctoral studies with Professor Arthur W. Nienhuis (1983-1986) at the National Heart Lung and Blood Institute (NHLBI) at the National Institutes of Health (NIH), Bethesda Maryland, where he received a Fogarty International Research Collaboration Award. He served as Chief of the Molecular and Medical Genetics Section, Developmental and Metabolic Neurology Branch (DMNB), National Institute for Neurological Disorders and Stroke (NINDS), NIH, 1988-1996. In 1995 he was recruited as a full professor to Lund University, Sweden, where he founded the Division of Molecular Medicine and Gene Therapy and was head of this division for twenty years. He is also a founding member of the Lund Stem Cell Center, since 2003, and was director of the Hemato-Linné Strategic Research Environment (2006-2016) funded through a 10-year Linnaeus grant from the Swedish Research Council. Dr. Karlsson also held a consultant physician position at Skåne University Hospital, Sweden (now retired).

    Dr. Karlsson's early research focused on retroviral vector based gene correction of hematopoietic cells from monogenetic disorders, such as Gaucher’s disease and hemoglobinopathies. The results of these studies led to the first gene therapy clinical trial for the treatment of Gaucher’s disease (1995). He has also developed lentiviral vectors for gene correction of hematopoietic stem cells, and more recently developed preclinical gene therapy models for Gaucher’s disease and Diamond Blackfan anemia.  An equal component of his research has been in the field of hematopoietic stem cell biology, where Dr. Karlsson focused on studying the mechanisms of hematopoietic stem cell expansion and maintenance with major contributions to understanding the role of Tgf-beta and more recently Cripto.

  8. Donald Kohn

    Donald B. Kohn, MD

    Distinguished Professor; Microbiology, Immunology and Moleculary Genetics; Pediatrics (Hematology/Oncology); Molecular and Medical Pharmacology, UCLA Broad Stem Cell Research Center

    Donald Kohn is a Distinguished Professor at the University of California, Los Angeles and a pediatric bone marrow transplant physician. He performs laboratory and clinical studies of gene therapy for blood cell diseases, especially primary immune deficiencies and hemoglobinopathies. His research is focused on developing improved methods for adding or editing genes in human hematopoietic stem cells and evaluating these approaches in early phase clinical trials.

  9. Timothy Ley

    Timothy J. Ley, MD

    Lewis T. and Rosalind B. Apple Chair in Oncology; Professor of Medicine and Genetics; Director of the Stem Cell Biology Section, Division of Oncology; Co-Director of the Oliver Langenberg Physician Scientist Training Program (Department of Medicine) at Washington University School of Medicine in St. Louis

    Timothy Ley is a pioneer in the field of cancer genomics, leading the team that sequenced the first cancer genomes, from patients with Acute Myeloid Leukemia (AML). This work has led to an explosion of new studies of the genomes of many cancer types, which has ushered in a new era of understanding of the mutations that cause this disease. Ley and his colleagues have developed genomic methods to understand how AML starts and evolves, and designed methods to track the clearance of AML cells after initial treatment. These approaches are now being used in clinical studies to better define risk of relapse.

    Dr. Ley has mentored more than 60 pre- and post-doctoral fellows in his laboratory. Most of these individuals now hold positions in academic medicine or at pharmaceutical companies. He has won several teaching and mentoring awards at Washington University, and received the Basic Science Mentoring Award from the American Society of Hematology in 2008. Ley was a key advocate for establishing the extramural Loan Repayment Programs at the National Institutes of Health, and has authored a number of reports on the physician-scientist workforce in the United States.

    Dr. Ley has won a number of awards for his work, and also has served in many national leadership positions. He was president of the American Society for Clinical Investigation, chair of the Board of Scientific Counselors of NHGRI, and served on the National Cancer Advisory Board. He is a member of National Academy of Sciences, the National Academy of Medicine, and the American Academy of Arts and Sciences.

    Dr. Ley earned his bachelor’s degree from Drake University and his medical degree from Washington University School of Medicine in 1978. He went on to perform his residency in internal medicine at Massachusetts General Hospital and completed fellowships in hematology and oncology at the National Institutes of Health and at Washington University School of Medicine, before joining the faculty at Washington University in 1986.

  10. Luigi Naldini

    Luigi Naldini, MD, PhD

    Director, San Raffaele Telethon Institute for Gene Therapy; Full Professor at the San Raffaele University

    Luigi Naldini is Director of the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and full professor at the San Raffaele University in Milan, Italy. Since 1996, he pioneered the development of lentiviral vectors for gene therapy, which have become one of the most widely used tools in biomedical research and, upon clinical testing, have been providing a long-sought hope of cures for several otherwise deadly human diseases. Throughout this time, he has been investigating strategies to overcome the major hurdles to safe and effective gene therapy, bringing about solutions that not only translated into new treatments but also allowed novel insights into hematopoietic stem cell (HSC) function and tumor angiogenesis. By applying microRNA regulation to vector design, his work introduced a broadly used approach to make gene transfer stringently responsive to cell type and differentiation. By leveraging on the discovery of a subset of proangiogenic monocytes which contribute to tumor growth, he developed a new gene therapy strategy by which the monocyte progeny of transplanted HSC is engineered to target immune stimulatory cytokines, such as interferon, to tumors, avoiding systemic toxicity and inducing tumor-specific immune responses. A first-in-human trial of this strategy is ongoing for glioblastoma multiforme sponsored by the spin-off Genenta Sciences (Nasdaq: GNTA). Dr. Naldini also pioneered applications of gene editing in gene therapy, providing the first demonstration of T-cell receptor genetic editing and of correction of disease-causing mutations in human HSC. He also reported the first proof of hit-and-run epigenetic editing, stably silencing genes without modifying the DNA sequence. This discovery led to a spinoff recently merged with a sister company in Boston (Chroma Medicine). With the SR-Tiget clinical unit, Dr. Naldini contributed to first-in-human testing and further clinical development of HSC gene therapy for immunodeficiencies, storage diseases and hemoglobinopathies, with >140 patients treated up to date with excellent safety and efficacy. Some of these therapies have been licensed to pharma and have reached the market (Strimvelis for ADA-SCID in 2016 and Libmeldy for MLD in 2020).

    Dr. Naldini received his medical degree from the University of Torino, Italy, and his PhD from the University of Rome. He is Member of the European Molecular Biology Organization (EMBO), has been President of the European Society of Gene and Cell Therapy (ESGCT), and was appointed as expert on the “Human Gene Editing Study” of the US National Academies of Sciences and of Medicine, and on the Italian National Committee for Biosafety, Biotechnology and Life Sciences. He was awarded the Outstanding Achievement Award from the American Society of Gene and Cell Therapy (ASGCT) and from ESGCT, an Honorary doctorate from the Vrije University, Brussel, the Jimenez Diaz Prize, the Beutler Prize from the American Society of Hematology and the Jeantet-Collen Prize for Translational Medicine. He was nominated “Grande Ufficiale dell’Ordine al Merito della Repubblica Italiana”, one of the highest-ranking honor in Italy and elected member of “Accademia dei Lincei”, the oldest and most prestigious National Academic Society.

  11. David Nathan

    David G. Nathan, MD

    President Emeritus, Dana-Farber Cancer Institute; Physician-in-Chief Emeritus, Boston Children’s Hospital; Professor of Pediatrics and Medicine, Harvard Medical School

    David G. Nathan is a graduate of Harvard College (1951) and Harvard Medical School (1955).  He was an intern and senior resident in medicine at the then Peter Bent Brigham Hospital and a Clinical Associate at the National Cancer Institute.  From 1959 to 1966 he was a hematologist at the Peter Bent Brigham Hospital and then became Chief of the Division of Hematology and Oncology at Children's Hospital and Dana-Farber Cancer Institute.  From 1985 to 1995 he was Physician-in-Chief of the Children's Hospital and from 1995 to 2000 was President of Dana Farber Cancer Institute.  Dr. Nathan's research has focused on the inherited disorders of red cells and granulocytes and particularly on thalassemia and sickle cell anemia.  His contributions include the introduction of effective treatment of iron overload and of hydroxyurea, the only FDA approved drug for prevention of the symptoms of sickle cell anemia. He has been instrumental in the development of the prenatal diagnosis of the hemoglobinopathies. He has trained over 100 hematologists many of whom hold leading positions in pediatrics and internal medicine.  His textbook entitled Hematology of Infancy and Childhood is the leading text in the field. He is the author of two popular books: Genes Blood and Courage published by the Harvard University Press in 1995, and The Cancer Treatment Revolution published by John Wiley and Sons in March 2007. Dr. Nathan is a member of the American Society for Clinical Investigation, the Association of American Physicians, the American Pediatric Society, the Institute of Medicine, the American Academy of Arts and Sciences and the American Philosophical Society.  He is the recipient of numerous awards including the National Medal of Science, the Stratton medal of the American Society of Hematology (of which he was President), The Walker Prize of the Boston Museum of Science, The John Howland medal of the American Pediatric Society, The George M. Kober Medal of the Association of American Physicians, The John Stearns Medal for Lifetime Achievement in Medicine of the New York Academy of Medicine, Harvard University Honorary Doctor of Science, The Wallace Coulter Award of the American Society of Hematology, Clarkson University Honorary Doctor of Science, The Dr. von Hauner Medal, Dr. von Hauner Children's Hospital (Munich, Germany), Sultan Bin Khalifa Grand International Award (Abu Dhabi) and the Boston Children's Hospital's Inaugural Lifetime Impact Award.

  12. Amit Nathwani, MD, PhD

    Amit Nathwani, MD, PhD

    Senior NIHR Investigator, and Professor of Haematology, UCL

    Amit Nathwani is a Senior NIHR Investigator, and Professor of Haematology at UCL. He received his medical degree from the University of Aberdeen and PhD in Molecular Biology from Open University, UK. After completing postgraduate training in Haematology, he embarked on a pivotal journey at St. Jude Children's Research Hospital in Memphis, Tennessee. It was here, under the guidance of the esteemed Dr. Arthur Nienhuis, that Professor Nathwani developed a pioneering gene therapy approach for haemophilia B. This served as an important catalyst that changed the gene therapy landscape for monogenethic disorder. This was followed by development of technologies in his laboratory at UCL that led to successful gene therapy of haemophilia A.

    Professor Nathwani's research extends to a diverse range of translational gene transfer approaches focused on inherited disorders such as Fabry's and Gaucher's disease. These achievements culminated in the co-founding of Freeline Therapeutics in 2015, a UCL-spinout company aimed at advancing gene therapy solutions. In 2019, Professor Nathwani founded NovalGen to accelerate cutting-edge research from his academic laboratory into disruptive immunotherapy approaches for both cancer and non-malignant conditions.

    Professor Nathwani is the recipient of the Ham-Wasserman Award, ESGCT Outstanding Achievement Award, Human Gene Therapy Award, and UCL Enterprise Award for his pioneering work in gene therapy and authored >100 peer-reviewed scientific papers.

  13. Stuart Orkin

    Stuart H. Orkin, MD

    David G. Nathan Distinguished Professor of Pediatrics at Harvard Medical School, and an HHMI Investigator at Boston Children’s Hospital

    Stuart Orkin is the David G. Nathan Distinguished Professor of Pediatrics at Harvard Medical School, and an HHMI Investigator at Boston Children’s Hospital. Orkin has defined the molecular basis of human blood disorders and mechanisms governing blood cell development. He received a BS from MIT and an MD from Harvard Medical School. He was a USPHS Research Associate in the Laboratory of Molecular Genetics, NICHHD, NIH under the supervision of Dr. Philip Leder from 1973-1975. Following his experience at NIH, Dr. Orkin returned to Boston where he undertook additional training in pediatrics and pediatric hematology/oncology. He served as Chairman of the Department of Pediatric Oncology at the Dana Farber Cancer Institute from 2000-2016.

    He provided the first comprehensive molecular dissection of an inherited disorder (the thalassemia syndromes), and characterized genes responsible for other human blood disorders, including X-linked chronic granulomatous disease (the first positional cloning).  Orkin identified the first hematopoietic transcription factors (the GATA family) and characterized their roles in blood cell development and cancer. His studies of BCL11A, a repressor of fetal hemoglobin (HbF), have elucidated the regulation of globin gene switching and led to novel genetic therapies of the thalassemias and sickle cell disease.

    Dr. Orkin was elected to the National Academy of Sciences (NAS), National Academy of Medicine (NAM), American Academy of Arts and Sciences, and the American Philosophical Society, His honors and awards include the E. Mead Johnson Award, Warren Alpert Prize, Helmut Horten Foundation Prize, Distinguished Research Award from the Association of American Medical Colleges (AAMC), E. Donnall Thomas, Dameshek and Basic Science Mentor Awards of the American Society of Hematology (ASH), Jessie the Stevenson Kovalenko Medal of the National Academy of Sciences (2013), William A. Allan Award of the American Society of Human Genetics (2014), George M. Kober Medal of the American Association of Physicians (2018), Mechthild Esser Nemmers Prize in Medical Science of Northwestern University (2018), King Faisal Prize in Medicine (2020), Harrington Prize for Innovation in Medicine (2020), Tobias Prize Lecture of the International Society of Stem Cell Research (2021), Gruber Prize in Genetics (2021), Canadian Gairdner International Award (2022), Society of Memorial Sloan Kettering Prize (2023), George Stamatoyannopoulos Mentorship Award of the American Society of Gene and Cell Therapy (2023), Honorary Doctorate (PhD honoris causa) University of Montreal, and the Elaine Redding Brinster Prize (2024).

  14. Griffin Rodgers

    Griffin P. Rodgers, MD, MACP

    Director of the National Institute of Diabetes and Digestive and Kidney Diseases

    Griffin Rodgers was named Director of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)--one of the National Institutes of Health (NIH)--on April 1, 2007. He had served as NIDDK’s Acting Director since March 2006 and had been the Institute’s Deputy Director since January 2001. As the Director of NIDDK, Dr. Rodgers provides scientific leadership and manages a staff of over 600 employees and a budget of ~$2.3 billion.  Dr. Rodgers received his undergraduate, graduate and medical degrees from Brown University in Providence, R.I. He performed his residency and chief residency in internal medicine at Barnes Hospital and the John Cochran VA, respectively, at Washington University in St. Louis, MO. His fellowship training in hematology was in a joint program of the NIH with George Washington University. In addition to his medical and research training, he earned an MBA, with a focus on the business of medicine/science, from Johns Hopkins University in 2005, and a Masters in Legal Studies in 2017.  As a research investigator, Dr. Rodgers is widely recognized for his contributions to the development of the first effective — and FDA approved — therapy for sickle cell anemia. In addition, he and his collaborators have reported on a modified blood stem-cell transplant regimen that is highly effective in reversing sickle cell disease in adults and is associated with relatively low toxicity. He has been honored for his research with numerous awards including the 1998 Richard and Hinda Rosenthal Foundation Award, the 2000 Arthur S. Flemming Award, the Legacy of Leadership Award in 2002, a Mastership from the American College of Physicians in 2005, the Herbert C. Nickens Award 2018 and a Fellowship in the Royal College of Physicians (London) in 2018, among others.  Dr. Rodgers is a member of the American Society of Hematology, the American Society of Clinical Investigation, the Association of American Physicians, the American Academy of Arts and Sciences, the American Association for the Advancement of Science, and the National Academy of Medicine, among others. 

  15. Information to come

  16. John Stamatoyannopoulos

    John A. Stamatoyannopoulos, MD

    Director, Altius Institute for Biomedical Sciences

    John Stamatoyannopoulos is Director of the Altius Institute for Biomedical Sciences in Seattle. He is internationally recognized for his work on human genome regulation and its role in common diseases and traits. His laboratory created foundational technologies for illuminating genome function; pioneered mapping of the regulatory regions encoded in the human and mouse genomes; and discovered that most genetic changes underlying common human diseases and traits impact gene regulation. Dr. Stamatoyannopoulos has delivered over 250 invited and named lectures worldwide and is one of the world’s most highly cited researchers.

    Dr. Stamatoyannopoulos holds degrees in Biological Sciences, Symbolic Systems, and Classics from Stanford University, and an M.D. from the University of Washington School of Medicine. He completed medical training at Harvard Medical School, including Internal Medicine at Brigham and Women's Hospital, and Oncology and Hematology at the Dana Farber Cancer Institute and Massachusetts General Hospital. Dr. Stamatoyannopoulos is an elected Fellow of the American Association for the Advancement of Science, and an elected Member of both the American Society for Clinical Investigation and the Association of American Physicians.

  17. John Tisdale

    John Tisdale, MD, PhD

    Senior Investigator, Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute; National Institutes of Health

    John Tisdale received his medical degree from the Medical University of South Carolina in Charleston after obtaining his B.A. in Chemistry from the College of Charleston.  He completed an internal medicine and chief residency at Vanderbilt University Medical Center in Nashville and then trained in hematology in the Hematology Branch, National Heart, Lung and Blood Institute (NHLBI), where he served as a postdoctoral fellow. He joined the Molecular and Clinical Hematology Branch of NHLBI in 1998 and is now the Chief of the Cellular and Molecular Therapeutics Branch. In 2020 the College of Charleston recognized Dr. Tisdale as one of their Top 25 History makers in honor of the school's 250-year anniversary and was Samuel J. Heyman Service to America Medal finalist. He was recently elected to the American Society for Clinical Investigation and is a member of the American Society of Hematology. Dr. Tisdale’s research and clinical work center on sickle cell disease. His group focuses on developing curative strategies for sickle cell disease through transplantation of allogeneic or genetically modified autologous bone marrow stem cells. He has over 200 publications describing his work. 

  18. Neal Young

    Neal Young, MD

    Chief of the Hematology Branch of the National Heart, Lung, and Blood Institute

    Neal Young is Chief of the Hematology Branch of the National Heart, Lung, and Blood Institute. His research interests are normal and aberrant hematopoiesis, autoimmunity in hematology, the genetics and genomics of aplastic anemia and related syndromes, and viral infections of blood forming cells. His career has been wide ranging, from basic molecular biology, virology, immunology, and cell biology to translational research, epidemiology, and pioneering interventional clinical trials. The Hematology Branch clinic is the major American referral center for marrow failure syndromes. Results from his work have deeply informed our understanding of the pathophysiology of human disease and development of effective treatments, for aplastic anemia, paroxysmal nocturnal hemoglobinuria, myelodysplastic syndromes and related diseases. He has published more than 450 original research articles and 100s of reviews and chapters, including many monographs and more than two dozen articles in the New England Journal of Medicine. His trainees are the current leaders and international experts in the field of marrow failure. His accomplishments have been recognized by the American Society of Hematology with the E. Donnall Thomas and Beutler Prizes, and awards such as the Adolfo Storti Award, the Erasmus Prize, and lifetime honorary membership in the Mexican Society for Hematology. For public service, he received the Heyman Service to America Award for civil service and the Vietnam People’s Award for his innovative teaching program in that country. Dr. Young recently completed a Visiting Fellow at New College in Oxford.

 
 
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