Global Forum

Global Head of Hemophilia with Medical Affairs Rare Blood Disorders, Sanofi

Cambridge, United States

Cecile Le Camus is Global Head of Hemophilia with Medical Affairs Rare Blood Disorders at Sanofi, based in Cambridge MA, USA. After 8 years in clinical practice, primarily as an ER clinician, in sports medicine and mountain rescue, Cecile joined the industry 20+ years ago. Cecile has held roles with Sanofi and Roche in France before joining Vertex in 2003., taking on roles of growing responsibility. Cecile joined their Boston Headquarters in 2015 as Global Medical Lead for Cystic Fibrosis, and went on to head their US Medical Affairs, supporting successful launches. In 2021 Cecile joined Sanofi as Global Medical Head for Hemophilia. Cecile is most passionate about including the patient voice, developing effective partnerships with key stakeholders, generating evidence and education that enable healthcare providers, payers and policymakers to make informed decisions for better patient outcomes with a true commitment to patient access to the therapies they need wherever they live.

Global Access Strategy Leader, Roche

Basel, Switzerland

Martynas Aizenas, is the Global Access Strategy Leader for Hematology at Roche is responsible for enabling comprehensive and equitable access for the portfolio of products. Martynas Aizenas has over 15 years of broad cross-functional experience in multiple countries and disease areas in advancing new medicines both locally and globally. For the last 5 years Martynas was leading Roche Global Haemophilia Access strategy.

Consultant Haematologist, Royal Free Hospital

London, United Kingdom

Professor Chowdary is a Professor of Haemophilia and Haemostasis at Dept. of Haematology, University College of London (UCL) and Centre Director for the Katharine Dormandy Haemophilia & Thrombosis Centre, Royal Free Hospital. In addition, she is the Chairperson for the United Kingdom Haemophilia Centre Doctors Organisation and is co-director of the National Haemophilia Database. Her clinical interests include the care of patients with Haemophilia and related bleeding disorders. Her primary research interests focus on strategies for the personalised management of haemophilia, novel therapies in haemophilia, acquired coagulopathy and bleeding. She is an active clinical researcher and is the chief investigator for several non-interventional and interventional studies, including the UK EHL registry and UCL-sponsored gene therapy studies in haemophilia A and B. She has authored more than 100 peer-reviewed publications, including three textbook chapters. She is a chair and member of various national and international working parties, data safety monitoring boards, trial steering committees, and national research peer review panels. She is also a member of numerous professional societies, including the International Society on Thrombosis and Haemostasis, the British Society of Haemostasis and Thrombosis, the European Association of Allied Bleeding disorders and the World Federation of Haemophilia.

Director, Research & Education, WFH

Montreal, Canada

Donna Coffin, MSc is currently the Director, Research & Education at the World Federation of Hemophilia (WFH) in Montreal, Canada. Donna is an epidemiologist, trained at McGill University in Montreal, and has spent much of her career conducting evidence based research for health care and regulatory agencies around the world. Since joining the WFH in 2016, Donna has established the Research & Education department, with focus on the development and implementation of the World Bleeding Disorders Registry. Her team is also responsible for all WFH research activities, including the WFH Annual Global Survey, the WFH Clinical Research Grant Program, and the production of evidence-based advocacy tools used to promote treatment for all.

Assistant Professor of Medicine, Brigham and Women's Faulkner Hospital

Boston, United States

Dr. Nathan Connell is Chief of Medicine at Brigham and Women’s Faulkner Hospital, Vice Chair of Medicine at Brigham and Women’s Hospital, Associate Director of the Boston Hemophilia Center, and Associate Professor of Medicine at Harvard Medical School. His clinical practice focuses on inherited bleeding disorders and he researches cost-effective strategies for the diagnosis and management of hematologic diseases. A graduate of Cornell University, the University of Miami School of Medicine, and the Harvard School of Public Health, he completed an internal medicine residency and a hematology/oncology fellowship at Brown University. In addition to his work defining the field of systems-based hematology, he co-chaired the ASH ISTH NHF WFH Guidelines on the Diagnosis and Management of von Willebrand disease.

Professor and Hallman Director, University of Waterloo

Waterloo, Canada

Dr. Andrea Edginton is a Professor and the Hallman Director of the School of Pharmacy, University of Waterloo, Canada. She has 120+ peer-reviewed publications in pharmacokinetics and modeling and simulation. Her academic research focuses on the development and application of physiologically-based pharmacokinetic (PBPK) models and simulation techniques in the areas of pharmaceuticals and human health risk assessment. Her research examines how the physiology of sub-populations (e.g. children, patients with disease) affect the PK of drugs through integration with PBPK models for the optimization of drug therapy. Dr. Edginton heads the modeling component of the Web-Accessible Population Pharmacokinetics Service – Hemophilia (WAPPS-Hemo.org) project that uses population pharmacokinetic modeling and Bayesian forecasting for tailoring treatment prophylaxis in persons with hemophilia. This free service is used by over 600 hemophilia treatment centres in over 40 countries. As a result of her research in hemophilia, she sits on the International Prophylaxis Study Group (IPSG) PK Expert Working Group.

Professor, University of Texas

Austin, United States

Miguel Escobar is tenured Professor of Internal Medicine and Pediatrics at the University of Texas Health Science Center, MD Anderson Cancer Center and the McGovern Medical School in Houston, Texas. He is also the Director of the Gulf States Hemophilia and Thrombophilia Center, the Clinical Research Center at the University and the Medication, Therapy and Wellness Center at the Memorial Hermann Hospital in Houston. Dr Escobar received his MD from the Universidad Libre in Cali, Colombia and did his residency in Internal Medicine at the University of Connecticut and fellowship in hematology/oncology at the University of North Carolina at Chapel Hill, USA. Dr Escobar has been involved in many clinical studies, resulting in a range of publications and is a member of several professional organisations. He is a member of the World Federation of Hemophilia Board of Directors and Chair of the Scientific and Standardization Subcommittee on FVIII/FIX & rare coagulation disorders for the ISTH. His main research interest is in haemophilia, congenital and acquired inhibitors and other coagulation deficiencies.

Assistant Professor, Perelman School of Medicine, University of Pennsylvania and Director of Clinical In Vivo Gene Therapy, Children’s Hospital

Philadelphia, United States

Dr. Lindsey George is an Assistant Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania and Director of Clinical In Vivo Gene Therapy at the Children’s Hospital of Philadelphia. Her laboratory studies the molecular basis of coagulation that in diminished or excess functional states leads to disorders of hemostasis and thrombosis, respectively. The work is particularly interested in the intrinsic tenase enzyme complex that, in deficiency states, results in hemophilia A or B. The current primary focus of her lab is the regulation of factor VIII cofactor function with the goal of exploiting our biochemical understanding to develop gene based therapies for hemophilia A. She is additionally interested in understanding the mechanistic basis of translational questions that have emerged from hemophilia gene therapy clinical trials. She was previously the lead clinical principal investigator of early phase hemophilia A and B adeno-associated virus-mediated gene addition trials. Lastly, as the director of the Clinical In Vivo Gene Therapy at the Children's Hospital of Philadelphia, this group long-term aims to safely and efficiently advance translational and clinical research for in vivo gene therapy for children with genetic disorders.

Professor, University of Washington

Washington, United States

Lou Garrison, PhD, is Professor Emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004. He is also a Visiting Senior Fellow at the Office of Health Economics in London following his sabbatical there in 2012-13. For the first 13 years of his career, he worked in non-profit health policy. Following this, he worked as an economist in the pharmaceutical industry for 12 years. He received a PhD in Economics from Stanford University and has more than 200 publications in peer-reviewed journals. His research interests include national and international health policy issues related to health technology assessment, personalized medicine, benefit-risk analysis, and other topics, as well as the economic evaluation of pharmaceuticals, diagnostics, and other technologies. Dr. Garrison was elected as ISPOR President for July 2016-June 2017, and currently serves as co-chair of ISPOR’s Policy Outlook Committee for the Health Science Policy Council. In September 2022, he was announced as the recipient of the 2022 Avedis Donabedian Outcomes Research Lifetime Achievement Award from ISPOR.

Assistant Professor, Perelman School of Medicine, University of Pennsylvania and Director of Clinical In Vivo Gene Therapy, Children’s Hospital

Philadelphia, United States

Dr. Lindsey George is an Assistant Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania and Director of Clinical In Vivo Gene Therapy at the Children’s Hospital of Philadelphia. Her laboratory studies the molecular basis of coagulation that in diminished or excess functional states leads to disorders of hemostasis and thrombosis, respectively. The work is particularly interested in the intrinsic tenase enzyme complex that, in deficiency states, results in hemophilia A or B. The current primary focus of her lab is the regulation of factor VIII cofactor function with the goal of exploiting our biochemical understanding to develop gene based therapies for hemophilia A. She is additionally interested in understanding the mechanistic basis of translational questions that have emerged from hemophilia gene therapy clinical trials. She was previously the lead clinical principal investigator of early phase hemophilia A and B adeno-associated virus-mediated gene addition trials. Lastly, as the director of the Clinical In Vivo Gene Therapy at the Children's Hospital of Philadelphia, this group long-term aims to safely and efficiently advance translational and clinical research for in vivo gene therapy for children with genetic disorders.

Lexington, United States

Co-Founder and CEO of Guardian Therapeutics, Inc., and Co-Founder and Managing Director of Band Therapeutics, LLC Board Certified American Board of Internal Medicine since 1988 MD from University of Connecticut 1984 BS from Yale University 1980 Selected Bibliography:
1. Kovacevic KD, Grafeneder J, Schörgenhofer C, et al. The von Willebrand factor A-1 domain binding aptamer BT200 elevates plasma levels of von Willebrand factor and factor VIII: a first-in-human trial. Haematologica 2022;107(9):2121-2132.
2. Gelbenegger G, Schoergenhofer C, Derhaschnig U, et al. Inhibition of complement C1s in patients with cold agglutinin disease: lessons learned from a named patient program. Blood Adv 2020;4(6):997-1005.
3. Jäger U, D'Sa S, Schörgenhofer C, et al. Inhibition of complement C1s improves severe hemolytic anemia in cold agglutinin disease: a first-in-human trial. Blood 2019;133(9):893-901
4. Bartko J, Schoergenhofer C, Schwameis M, et al. A Randomized, First-in-Human, Healthy Volunteer Trial of sutimlimab, a Humanized Antibody for the Specific Inhibition of the Classical Complement Pathway. Clin Pharmacol Ther 2018;104(4):655-663.
5. Cataland SR, Peyvandi F, Mannucci PM, et al. Initial experience from a double-blind, placebo-controlled, clinical outcome study of ARC1779 in patients with thrombotic thrombocytopenic purpura. Am J Hematol 2012;87(4):430-2
6. Jilma-Stohlawetz P, Gorczyca ME, Jilma B, Siller-Matula J, Gilbert JC, Knöbl P. Inhibition of von Willebrand factor by ARC1779 in patients with acute thrombotic thrombocytopenic purpura. Thromb Haemost 2011;105(3):545-52.
7. Jilma-Stohlawetz P, Gilbert JC, Gorczyca ME, Knöbl P, Jilma B. A dose ranging phase I/II trial of the von Willebrand factor inhibiting aptamer ARC1779 in patients with congenital thrombotic thrombocytopenic purpura. Thromb Haemost 2011;106(3):539-47
8. Arzamendi D, Dandachli F, Théorêt JF, et al. An anti-von Willebrand factor aptamer reduces platelet adhesion among patients receiving aspirin and clopidogrel in an ex vivo shear-induced arterial thrombosis. Clin Appl Thromb Hemost 2011;17(6):E70-8.

Pediatric Hematologist, Amsterdam UMC

Amsterdam, Netherlands

Samantha C. Gouw, MD, PhD is trained as a pediatric hematologist and clinical epidemiologist. She received her PhD doctorate on the etiology of inhibitor development in children with severe hemophilia A at Utrecht University in 2011. She currently works as a clinician-researcher at the Department of Pediatric Hematology at the Amsterdam University Medical Center. She is the coordinating researcher of a nationwide study and biobank in hemophilia patients at the Department of Clinical Epidemiology at Leiden University Medical center, The Netherlands and is a work package leader in the National Symphony Consortium. She is the lead of the Dutch Hemophilia Patient Registry, and board member of the Dutch Hemophilia Physicians Association and European Association of Hemophilia and Allied Disorders. Her current research focuses on inhibitor development and immune tolerance induction and the quality of hemophilia care, especially e-health and value based health care.

Medical & Humanitarian Aid Director, WFH

Montreal, Canada

Assad Haffar is currently the Medical and Humanitarian Aid Director at the World Federation of Hemophilia (WFH) based in Montreal, Canada. Dr Haffar earned his MD at the University of Damascus in Damascus, Syria. He subsequently studied at the London School of Hygiene and Tropical Medicine, University of LONDON, London, UK, for his Master of Science in occupational medicine and at the University of Environmental and Occupational Health in Kitakyushu City, Japan, for his Diploma in industrial health.

During his career at the International Labor Organization (ILO), Geneva, Switzerland (1990–1995), he coordinated a large program to improve occupational health services and workers education in selected Middle East countries.

Dr Haffar has worked as coordinator for several projects related to occupational health at the Montreal Re-Adaptation Institute and the McGill University in Montreal, Canada.

Since Joining WFH in 2000, Dr Haffar has been involved in several activities, including twinning partnerships, symposia and workshops in the Middle East and African Region. In addition to leading the Humanitarian Aid Department responsible for the WFH Humanitarian Aid Program which is considered the largest global humanitarian aid program that channels clotting factor concentrates to countries where people with hemophilia have limited access to these products, Dr Haffar assisted many countries in the Middle East and African region in joining and becoming members of the WFH, in developing National Hemophilia Care Programs and in establishing the comprehensive care approach in different hemophilia treatment centers. Since becoming the WFH Medical Director in 2018, he designed and led many WFH medical activities.

Global Head of Medical Affairs Rare Diseases, Sanofi Genzyme

Boston, United States

Dr. Hamed is the Global Head of Medical for Rare Disease and Rare Blood Disorders. Alaa and his team are responsible for expanding the strong legacy of Medical Leadership in Sanofi Genzyme Rare Disease, focusing on new evidence generation strategies, providing medical insights to product development, and ensuring effective and meaningful Medical engagement with Rare Disease stakeholders around the world. With a central focus on patient outcomes, and in strong partnerships with external experts and stakeholders, he has spearheaded the development of numerous innovative disease-specific clinical outcome assessments, while regularly representing the company in multiple congress, industry, regulatory, and HTA interactions. Dr. Hamed has been with Sanofi Genzyme for 10 years, prior to that he held various positions in Academia and biotech in HEOR, Medical, Clinical, Regulatory, and Health Services Research. His research has resulted in numerous high-impact publications authored in collaboration with key external experts, and he serves as a reviewer for multiple journals. He holds an MD from Cairo University and both and MBA and MPH from Boston University.

Professor, Pathology & Molecular Medicine, McMaster University

Hamilton, Canada

Dr. Hayward is an MD PhD and clinical and laboratory hematologist who is Professor of Pathology and Molecular Medicine, and Medicine, at McMaster University, and the Head of Coagulation for the Hamilton Regional Laboratory Medicine Program in Canada. Dr. Hayward has published extensively, with an H-index of 52 and over 10,000 citations. She is considered an expert on platelet disorders and the clinical and laboratory diagnostic evaluation of bleeding disorders. Dr. Hayward is the Immediate Past President of the Thrombosis Hemostasis Societies of North America (THSNA) and is also a Past President of the International Society for Laboratory Hematology (ISLH) and the North American Specialized Coagulation Laboratory Association. She is the Director of the McMaster University Fellowship Programs in Advanced Laboratory Coagulation/Hemostasis and Advanced Clinical and Laboratory Coagulation/Hemostasis and supervises Hematology and General Pathology Trainees during their Coagulation Rotation. As a clinician scientist, Dr. Hayward has received many honors and awards, including the Gold Medal award from McMaster University and from the Royal College of Physician and Surgeons of Canada.

Head, Hemophilia Center Cliniques Universitaires Saint-Luc

Brussels, Belgium

Cedric Hermans currently heads the Division of Haematology, the Hemostasis and Thrombosis Unit as well as the Hemophilia Center of the Saint-Luc University Hospital in Brussels, Belgium. He was appointed Associate Professor at the Medical School of the Catholic University of Louvain in 2003, Full Professor in 2012 and Vice-Dean in 2015. Professor Hermans has (co)-authored more than 40 original articles in international journals and is a member of several scientific societies and international advisory boards and collaborative research projects. He is Member of the Royal Academy of Medicine of Belgium and Fellow of the Royal Colleges of Physicians of London and Edinburgh.He was president of EAHAD and is currently member of the Board of Directors of the World Federation of Haemophilia and the Editor-in-Chief of the Haemophilia Journal. His main research interests lie in the area of haemostasis and thrombosis, especially clinical studies on the treatment modalities and the wide spectrum of complications of haemophilia in both developed and developing countries, as well as new anticoagulants and the management of thrombosis.

Professor, McMaster University

Hamilton, Canada

Alfonso Iorio, MD, PhD, FRCP(C) Department of Health Research Methods, Evidence and Impact & Department of Medicine, McMaster University, Canada Alfonso Iorio is a Professor and Chair Department of Health Research Methods, Evidence, and Impact at McMaster University Canada. He is Director of the Health Information Research Unit (HiRU, http://hiru.mcmaster.ca/hiru) and the Hamilton-Niagara Hemophilia Program. He received his medical and Ph.D. degrees from the University of Perugia, Italy. He is the Principal Investigator of the Web Application for Population Pharmacokinetic in Hemophilia (WAPPS) project (www.wapps-hemo.org), co-investigator of the Patient Reported Outcomes, Burden, and Experiences (PROBE), and Chair of the Canadian Bleeding Disorders Registry (CBDR). He is the past chair of the WFH Data and Demographics Committee and co-chair of the World Bleeding Disorders Registry (WBDR). He is Associate Editor for bleeding disorders of the Cochrane Collaboration, Thrombosis Research. He serves on the Editorial Boards of numerous journals, including the Journal of Thrombosis and Haemostasis, and Haemophilia. Outside Hemophilia, his current research interests include internet-based knowledge dissemination, systematic review and meta-analysis methodology, risk prediction and stratification, and artificial intelligence application to all of them. He has authored over 300 peer-reviewed papers totalling over 20,000 citations and an H-index of 59 (details available on his personal page Google Scholar). He has given many lectures, including educational lectures at many international conferences.

Assistant Research Professor of Pediatrics Wells Center for Pediatric Research, Indiana University School of Medicine

Indianapolis, United States

Dr. Radek Kaczmarek is an Assistant Research Professor of Pediatrics at Gene and Cell Therapy Group, Wells Center for Pediatric Research, Indiana University School of Medicine (Indianapolis, IN). He completed his PhD at Hirszfeld Institute of Immunology and Experimental Therapy in Wroclaw, Poland. His research interests include immune responses to gene and protein replacement therapies for hemophilia A and B. Radek chairs the Coagulation Products Safety, Supply and Access (CPSSA) Committee of the World Federation of Hemophilia (WFH), is a member of the Scientific and Standardization Committee (SSC) Gene Therapy Working Group of the International Society on Thrombosis and Hemostatis (ISTH) and a member of the Medical and Scientific Advisory Group of the European Haemophilia Consortium (EHC).

Henry Hamilton Chair in Hematology, The University of Iowa Carver College of Medicine

Iowa City, United States

Steven Lentz, MD, PhD Professor University of Iowa Iowa, United States Steven Lentz is the Henry Hamilton Chair in Hematology and the former Chief of the Hematology Section at the University of Iowa in Iowa, United States. Dr Lentz is recognised for his research on mechanisms of haemostasis, thrombosis and vascular dysfunction. Dr Lentz directs a translational and clinical investigation program in hemostasis and thrombosis, and he has served as the principal investigator of numerous clinical trials in von Willebrand disease, haemophilia and thrombosis at the Iowa Hemophilia and Thrombosis Center. He has mentored many pre-doctoral and post-doctoral trainees and directs training programs in hematology and medical scientist (MD-PhD) career development.

Adult Hematologist-Oncologist, Indiana Hemophilia and Thrombosis Center (IHTC)

Indianapolis, United States

Dr. Magdalena Lewandowska serves as an adult hematologist at the Indiana Hemophilia and Thrombosis Center in Indianapolis, IN. She graduated from Jagiellonian University in Cracow, Poland, and completed postgraduate medical education in Internal Medicine and Hematology/Oncology at St. Elizabeth’s Medical Center in Boston, MA. She is involved in medical education and clinical research, with a special interest in hemophilia, rare bleeding disorders, thrombosis, and taking care of women with bleeding disorders.

Professor, Queen's University

Kingston, Canada

David Lillicrap is a Professor in the Department of Pathology and Molecular Medicine at Queen’s University, Kingston, Canada. He is the recipient of a Canada Research Chair in Molecular Hemostasis. Dr. Lillicrap is a member of the World Federation of Hemophilia’s (WFH) Medical Advisory Board and is the Chair of the WFH’s Research Committee. He is the Immediate Past Chairman of the International Society for Thrombosis and Haemostasis’ Scientific and Standardization Committee, and is a member of the Council of ISTH. Dr. Lillicrap’s research interests are centered on molecular aspects of the hemostatic system with particular emphasis on the potential of molecular genetics and molecular biology to address questions relating to pathological hemostasis. The main foci of interest of his research group are the development and evaluation of novel therapies for hemophilia A, investigation of the immune response to FVIII and characterization of the biology and pathobiology of von Willebrand factor.

Professor, University of the Witwatersrand and NHLS

Johannesburg, South Africa

Johnny Mahlangu is a Professor in Haematology and Head of Department of Molecualr Medicine and Haematology in the Faculty of Health Sciences of the University of the Witwatersrand and the National Health Laboratory Service. He is also a Consultant Clinical Haematologist in Charge of the Haematology at the Charlotte Maxeke Johannesburg Academic Hospital and Director of the International Haemophilia Training Centre in Johannesburg. Johnny received his undergraduate and postgraduate training in science and medicine at the University of the Witwatersrand with haematology specialist and clinical haematology sub-specialist qualifications through the Colleges of Medicine of South Africa. He has published manyand presented over 500 abstracts, plenaries and key-note addresses at national and international meetings. His main area of research is novel therapies in bleeding disorders in which he has served as Principal Investigator for many international multicentre studies. Prof Mahlangu is current President of the College of Pathologists in South Africa and Chair of the South African Medical Research Council Board and Chair of the International Society on Haemostasis and thrombosis scientific and standardization Committee on Factor VIII, FIX and rare bleeding disorders.

Professor, University of Sheffield

United Kingdom

Professor Michael Makris MD Michael Makris is emeritus professor of haemostasis and thrombosis at the University of Sheffield. He is a past president of the European Association for Haemophilia and Allied Disorders (EAHAD). Professor Makris is the project lead for the EUHASS bleeding disorder adverse event reporting scheme in Europe. He is on the Editorial Board of the British Journal of Haematology, Journal of Thrombosis and Haemostasis, Seminars in Thrombosis and Haemostasis, and Blood Transfusion. He is currently associate editor of Research and Practice in Thrombosis and Haemostasis and will be taking over as Editor-in-Chief for this journal in January 2024.

Director, Global Development, WFH

Montreal, Canada

Salome Mekhuzla, WFH Director, Global Development leads a team responsible for implementing various healthcare development and capacity-building programs around the globe. Originally from the country of Georgia, her prior experience while working with European and US-based international NGOs on civil society development, advocacy and inter-ethnic relations in the South Caucasus region serves her well in her current role. She holds a MSocSc degree in Transformation in South Caucasus from Tbilisi State University, Center for Social Sciences and a MSc in Nationalism Studies from the University of Edinburgh. Her work takes her many places and she loves the multicultural nature of the WFH, believing it is this diversity that give our community such great strength.

Head - Gene Therapy Program, WFH

Montreal, Canada

Mayss Naccache is currently sector Head, Gene Therapy Program within the Research & Education department of the World Federation of Hemophilia. Since joining the WFH in 2017, Mayss managed the launch of the World Bleeding Disorders Registry, the development of the Gene Therapy registry, and the organization of the Gene Therapy Round Table series and Global Forum meetings. Mayss holds a Masters in genetics and recently completed her MBA.

Professor, MD, Department of Pediatrics - Nara Medical University

Kashihara, Japan

Brief biography
1991. Graduation. Jichi Medical University (MD)
2000. Nara Medical University, Graduate School of Medicine (PhD)
2002. Postdoctoral fellow (PJ Fay Lab.). University of Rochester, NY, USA
2012. Associate Professor. Department of Pediatrics, Nara Medical University
2021. Professor. Department of Pediatrics, Nara Medical University Specialty Structure and function analysis of FVIII, VWF, emicizumab Characterization of clotting factor inhibitor Clinic on hemophilia and other bleeding disorders Society Director, The Japanese Society on Thrombosis and Hemostasis Councilor, The Japanese Society of Hematology, The Japanese Pediatric Society Associate Editor, Thrombosis Journal Awards The Japanese Society on Thrombosis and Hemostasis - Researcher’s Award (2000) Bayer Hemophilia Foundation - Special Project Award (2009, 2013) Japan Bioindustry Association - Bioindustry Award “Emicizumab” (2020)

Clinical Medical Director Program Lead, Regeneron Pharmaceuticals, Inc.

United States

Meagan O’Brien is a Senior Medical Director in Early Clinical Development and Clinical Experimental Sciences at Regeneron Pharmaceuticals. Dr. O’Brien received her medical degree from Harvard Medical School and completed medical residency and a fellowship in infectious diseases in New York City at Columbia Presbyterian and New York University Medical Schools, respectively. Dr. O’Brien practiced medicine in New York City, US for 15 years and conducted basic science, translational, and clinical academic research prior to joining Regeneron in 2017 to work in Early Clinical development, with a focus on immunology, allergy, infectious diseases, and rare disease. Dr. O’Brien has led Phase 1, Phase 2, and Phase 3 studies involving investigational products targeting allergic diseases and COVID-19, respectively, and is a clinical liason for discovery related to genetic medicines, infectious diseases, and allergy. Dr. O’Brien is the clinical lead for the First in Human CRISPR-based Factor 9 gene insertion study for Hemophilia B which is being developed by Regeneron and Intellia Therapeutics.

CEO, Irish Haemophilia Society

Dublin, Ireland

Brian O’Mahony is Chief Executive of the Irish Haemophilia Society. He represents the society on the statutory National Haemophilia Council, the statutory Hepatitis C Consultative Council and he is the Vice Chair of the Tender Commission established by the Irish Government for the Procurement of Factor Concentrates and haemophilia medications. He previously served as Chair of the Irish Haemophilia Society for 17 years from 1987 to 2003. He served as President of the European Haemophilia Consortium (EHC) for 8 years from 2011 to 2019 and as President of the World Federation of Hemophilia (WFH) for 10 years from 1994 to 2004. He is currently a member of the Coagulation Product Safety Supply and Access (CPSSA) committee of the WFH and of the Medical and Scientific Advisory Group (MASAG) of the EHC. His publications for WFH include Monographs on Advocacy, Developing Haemophilia Organisations, Economics and Health Technology Assessments and A Guidebook on National Tender Systems. He is the author or co-author of more than 80 peer reviewed journal articles, and he is an ad-hoc reviewer for the journal Haemophilia. He is a Fellow of the Academy of Clinical Science and Laboratory Medicine (Ireland) and a Fellow of the Institute of Biomedical Sciences (UK). He has post graduate qualifications in Management and in Occupational Health and Safety and is currently an assistant adjunct Professor in Health Service Management in Trinity College, Dublin. Brian O’Mahony has severe Haemophilia B and is participating in a Gene Therapy clinical trial.

Director, Institute for Experimental Haematology and Transfusion Medicine

Bonn, Germany

Professor Johannes Oldenburg is the Director of the Institute of Experimental Haematology and Transfusion Medicine and the Haemophilia Centre at the University Clinic in Bonn, Germany. He is a Member of the Blood Committee for the German Ministry of Health. He is also a Member of the Editorial Boards for Blood, The Journal of Thrombosis and Haemostasis and Haemophilia. In 1988, he completed his medical degree at the University of Bonn. He completed his specialty training in Transfusion Medicine and became a Lecturer at the Institute of Experimental Haematology and Transfusion at the University of Bonn. He also completed specialty training in Medical Genetics at the University of Wuerzburg, where he became a Senior Lecturer at the Institute of Human Genetics until 2000. Prior to his current position, he served as Assistant Professor and leader of the research group ‘Molecular Haemostasis’ at the Institute of Experimental Haematology and Transfusion Medicine at the University of Bonn, and as Head of the Departments of Immune Haematology and Molecular Haemostasis at the Institute of Transfusion Medicine and Immune Haematology in Frankfurt. Professor Oldenburg has a research interest in the molecular genetics of blood coagulation factors, in haemophilia and vitamin K pathways.

Professor, Hemocentro Unicamp

Sao Paulo,Brazil

Dr. Ozelo is Associate Professor of Internal Medicine Department and Director of Haematology and Transfusion Medicine from the University of Campinas (UNICAMP) in Campinas, São Paulo, Brazil. She received her medical degree (1994), specialist training in haematology and transfusion medicine (1994-1997), and Ph.D. (2004) at UNICAMP before undertaking a postdoctoral fellowship in David Lillicrap’s laboratory, at Queen’s University in Kingston, Ontario, Canada, focused on gene therapy for hemophilia (2006–2009). She is also Director of the WFH International Haemophilia Training Centre (IHTC) from Hemocentro UNICAMP. In 2012, she was nominated as a member of the Novo Nordisk Haemophilia Foundation Council and was member of the Executive Committee of the World Federation of Haemophilia from 2012 to 2014. Dr. Ozelo is involved with several research projects, including gene therapy for haemophilia, risk factors for inhibitor development, immune tolerance induction, and the management of musculoskeletal complications of haemophilia, and inherited platelet disorders.

President, ISTH

Milan Italy

Flora Peyvandi MD, PhD is full professor of Internal Medicine at the University of Milan, Italy and director of the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center of the Milan Policlinic Hospital, Italy. Her research started with molecular and biochemical characterization of coagulation disorders aiming at the development of novel treatment for the affected patients. Her research continued with the understanding of the microvascular dysfunctions in order to implement/develop and standardize innovative diagnostic and therapeutic strategies. Since many years, Dr. Peyvandi has been participating in clinical, educational and research activities of different European and International scientific societies and patients’ organizations. She is currently the President of the International Society of Thrombosis and Haemostasis and Past-President of the Executive Committee of the European Association for Haemophilia and Allied Disorders. She also serves as advisor on the European Agency of Medicine and on the Technical Health Committee of the Italian Ministry of Health. Her clinical, teaching and research experience combined with her life in different countries, led her to familiarize with patient management, scientific projects and educational systems of different parts of the world.

Health columnist, The Globe and Mail

Vancouver, Canada

André Picard is the health columnist at The Globe and Mail, Canada’s national newspaper, where he has been a staff writer since 1987. He is also the author of six bestselling books, including “The Gift of Death: Confronting Canada’s Tainted Blood Tragedy.” The book was also the basis of the mini-series "Unspeakable," about the tainted blood scandal. Mr. Picard has received much acclaim for his writing, including the Centennial Prize of the Pan-American Organization, awarded to top health journalist in the Americas. His work has also been recognized by a number of health advocacy groups, including being named Canada’s first “Public Health Hero” by the Canadian Public Health Association, and as a “Champion of Mental Health” by the Canadian Alliance on Mental Illness and Mental Health. He was also a recipient of the Queen Elizabeth II Diamond Jubilee Medal for his dedication to improving healthcare. Mr. Picard is a graduate of the University of Ottawa and Carleton University. He has received honorary doctorates from eight universities, including the University of Toronto and the University of British Columbia. He lives in Vancouver.

Vice-President, Medical, WFH

United States

Dr. Glenn Pierce currently serves on the WFH as VP Medical and WFH USA Board of Directors and NHF (US) Medical and Scientific Advisory Council. He is an Entrepreneur-in-residence at Third Rock Ventures, and a biotech consultant in the gene therapy and hematological areas.

Professor Pediatric Hematology/Oncology, Michigan Medicine Hemophilia/Coagulation

Ann Arbor, United States

Dr Steven Pipe is a Professor and the Laurence A. Boxer Research Professor of Pediatrics and Professor of Pathology at the University of Michigan, Ann Arbor, Michigan, USA. He is the medical director of the Pediatric Hemophilia and Coagulation Disorders Program and medical director of the Special Coagulation Laboratory. His clinical interests include bleeding and thrombotic disorders and congenital vascular anomalies. Dr Pipe also directs a basic research lab investigating coagulation factor VIII and the molecular mechanisms of hemophilia A. He has been actively involved in clinical trials with novel therapeutics for hemophilia including gene therapy. He was the 2015 recipient of the Leadership in Research Award from the National Hemophilia Foundation. He has served on the Board of Directors for the Hemostasis and Thrombosis Research Society, as Chair of the Board of Directors for the American Thrombosis and Hemostasis Network and most recently as Chair of the Medical and Scientific Advisory Committee to the National Bleeding Disorders Foundation.

Director, Hemophilia Treatment Center

United States

I am an Associate Member in the Department of Hematology and Director of the Pediatric Hemophilia Treatment Center at St. Jude Children’s Research Hospital. I have secondary appointments as at University of Tennessee and St. Jude Pathology. I trained in Pediatrics, Pediatric Nephrology and Transplantation Immunology, and Pediatric Hematology/Oncology at the Universities of Heidelberg and Ulm in Germany, and at State University of New York, Stony Brook, and Children’s Hospital Oakland in the US. I trained in laboratory research for four years at Children’s Hospital Oakland Research Institute. I now have more than 25 years clinical experience in pediatric hematology. At St. Jude I work in clinical care, teaching and clinical research. From 2011-2018 I was Director of the Division of Clinical Hematology. I also have built and received accreditation for the St. Jude HTC. Our comprehensive HTC sees about 400 patients yearly. In research, I have been PI/Co-PI of the pivotal, first successful phase I/II clinical trial of AAV-mediated gene therapy for hemophilia B between St. Jude and University College London (UCL) and the UCL/St. Jude FVIII gene therapy phase I/II trial. I am developing a trial of AAV-mediated gene therapy for hemophilia B to establish safety and efficacy data across multiple patient cohorts globally, including from lower-income and higher-income regions, with the goal to accelerate widespread access. Within this novel program we also assess the educational needs of patients and staff, the cost-effectiveness, and the operational needs related to hemophilia gene therapy. I am also collaborating with ATHN, NBDF, WFH, ASGCT and the Regional HTC Network in clinical outcomes research or the development of clinical guidelines, research blueprints, or educational offerings.

Chief Medical Officer, ICER

Boston, United States

Dr. Rind is Chief Medical Officer of the Institute for Clinical and Economic Review. He is responsible for leading teams conducting analyses and critical appraisals of medical evidence across a wide range of clinical interventions including ICER reviews of therapies for hemophilia. Dr. Rind has expertise in evidence-based medicine and is a member of the international GRADE Working Group. Before joining ICER, Dr. Rind was Vice President, Editorial and Evidence-Based Medicine at UpTodate and was responsible for training UpToDate’s physician editors in clinical epidemiology, biostatistics, and evidence-based medicine. He continues to work with UpToDate as a section editor for general medicine. Dr. Rind received a B.A. in chemistry from Yale University and his M.D. from the University of Rochester. He completed his residency in internal medicine at the University of Massachusetts Medical Center and then did a joint fellowship in general internal medicine and clinical computing at Beth Israel Hospital and Harvard Medical School. He received a Master of Science in health policy and management from the Harvard School of Public Health. Dr. Rind continues to see patients as an academic primary care physician working with internal medicine residents in the outpatient practice at Beth Israel Deaconess Medical Center.

Chief Operating Officer, National Bleeding Disorders Foundation

Gisbonia, United States

Dawn has been a leader in the bleeding disorders community for many years. As a parent of a son with hemophilia, she founded the Rocky Mountain Hemophilia & Bleeding Disorders Association in Bozeman, Montana, served as its Executive Director, and began their Family Camp. After moving to Pittsburgh, Pennsylvania, she served as a board member for the Hemophilia Center of Western Pennsylvania for six years and then as Executive Director of the Western Pennsylvania Chapter. She has been with the National Bleeding Disorders Foundation (formerly named National Hemophilia Foundation) for 15 years and serves as Chief Operating Officer. Dawn serves on the Board of Directors for the World Federation of Hemophilia and World Federation of Hemophilia USA. Dawn proudly chairs the Women and Girls with Bleeding Disorders and the Hemophilia Organization Twin Committees for WFH. She holds a Bachelor of Science from Montana State University in Bozeman.

Honorary President, Association Française des Hémophiles

France

Thomas Sannié is the former President of the Association française des hémophiles (2012-2018) and Honorary President since. He is chairman of the Afath program in collaboration with the World Federation of Hemophilia to develop access to care and patient’s organization in French speaking African countries. He is a former lawyer. Today, he is the manager of projects of patients’ partnership in care in Paris region. He has been working for more than 10 years on educational program for and with patients with chronicle diseases and rare bleeding disorders. He has engaged in several pilot programs with universities to train young medical students. He is member of the Patient engagement committee of the French High Autority of health. Thomas Sannié is a former member of the WFH Board (2010-2014) and recepient in 2018 of the WFH International Prize Franck Schnabel. He is also member of WFH several committees.

President and CEO, Institute for Policy Advancement Ltd

Mark Skinner, Washington, DC, is President/CEO of the Institute for Policy Advancement Ltd, specializing in patient-centered outcomes research. He is an Assistant Professor in the Department of Health Research Methods, Evidence and Impact at McMaster. He has previously led both national and international patient organizations including the World Federation of Hemophilia and National Hemophilia Foundation where he currently serves on their Medical and Scientific Advisory Council. He is the principal investigator for the Patient Reported Outcomes Burdens and Experiences (PROBE) study, a global research project to enhance the direct patient voice in healthcare decision-making. He holds numerous roles as an advisor on critical blood safety and supply matters including having served on the US Health and Human Services Advisory Committee on Blood and Tissue Safety and Availability. He serves on the board of the Institute for Clinical and Economic Review (ICER), the National Organization for Rare Disorders (NORD) and formerly the Patient Centered Outcomes Research Institute (PCORI) Advisory Panel on Rare Disease, where he was an inaugural member. Previously, he was Vice President State Programs at the American Insurance Association and Administrative Assistant/Chief of Staff to the Speaker of the Kansas House of Representatives. He holds degrees in Public and Business Administration from Kansas State University and JD from Washburn University School of Law.

Professor, Christian Medical College

India

Alok Srivastava, MD, FRACP, FRCPA, FRCP Christian Medical College, Vellore, India Alok Srivastava is a Professor of Medicine in the Department of Haematology, and head of the Centre for Stem Cell Research, a unit of inStem, Bengaluru, at the Christian Medical College (CMC), Vellore in India. Dr. Srivastava has been involved with the management of patients with bleeding disorders for over 25 years. His group has worked extensively on developing management protcols for hemophilia and other inherited bleeding disorders including outcomes assessment which address the needs in developing countries. He promoted the concept of lower dose prophylaxis as an alternative to episodic factor replacement therapy. A major focus of his group's current research include assessment of novel therapies in different clinical trials as well as developing gene therapy for hemophilia using both AAV and lentiviral approaches, with the latter being evaluated in a first in human clinical trial. He leads the World Federation of Hemophilia designated International Hemophilia Training Center at CMC, Vellore. Dr. Srivastava is President of the Indian Association for Haemophilia & Allied Disorders. He is also the chair of the steering committee of the Association for Haemophilia & Allied Disorders – Asia-Pacific. He was the chair of the FVIII/IX subcommittee of the Scientific and Standardization Committee (SSC), International Society of Thrombosis and Haemostasis (ISTH) from 2006-2010. He was on the board of the WFH from 2002-2014 and served as the Vice-President (Medical) from 2012-2014. He has led the writing group for all three editions of the WFH guidelines for the management of hemophilia.

Director of the Department of Haemostasis & Transfusion, CHRU Lille

Lille, France

Sophie Susen is professor of Hematology at Lille medical school Lille (France) and heads the department of Hemostasis and Transfusion in Lille University Hospital. She has been a member of the expert-group for blood products at the French regulatory agency. Her basic research activity focuses mainly in the role of VWF and blood flow via different approaches. She is the national coordinator of the French National reference center on von Willebrand disease and the national network fore rare bleeding disorders. She heads the research team "Cardiac pathologies, blood flow abnormalities and Haemostasis" at Inserm.

Global Medical Lead - Hemophilia, Pfizer

Greater Toronto Area, Canada

Lisa Wilcox, PhD is the Global Medical Affairs lead for Hemophilia at Pfizer. In this role she leads the global medical affairs team and medical strategy for the Hemophilia portfolio, including approved and pipeline products. Lisa joined the pharmaceutical industry in 2004 with Wyeth Canada, transitioning to Pfizer in 2009. Since that time she has held multiple roles in Medical Affairs across therapeutic areas that span Oncology, Rare Diseases and Internal Medicine.

President & Chief Operating Officer, Be Biopharma

Cambridge, United States

Krishnan Viswanadhan, Pharm.D. President & Chief Operating Officer Be Biopharma Krishnan Viswanadhan, Pharm.D., president and chief operating officer of Be Biopharma, is a biopharmaceutical executive with over 20 years of broad cross-functional experience in advancing new medicines for patients with serious and life-threatening diseases. Krishnan joined Be Bio as president and COO in 2021 and is responsible in driving all operational aspects of the business to unleash the power of engineered B cell medicines as therapeutics for patients with serious diseases. Prior to Be Bio, Krishnan was senior vice president, global cell therapy franchise lead at Bristol Myers Squibb (BMS) where he was responsible for overseeing the integrated cell therapy franchise strategy across the enterprise including building core capabilities including key investments to support long term growth. He oversaw the teams responsible for the development, approval and life cycle management of Breyanzi, a CD19 CAR-T, and Abecma, the first BCMA CAR T therapy. Prior to BMS, Krishnan was vice president, business development and global alliances, at Celgene Corporation, where he was responsible for managing the portfolio of partnerships and equity investments. Prior to Celgene, Krishnan had roles in large and small companies in regulatory strategy. Krishnan is a registered pharmacist and received his Pharm.D. from Rutgers University and holds an MBA degree from Cornell University. Krishnan currently serves the board as a non-executive director of JW Therapeutics, a leading cell therapy company in China, and as an independent director at Cargo Therapeutics.

Assistant Professor, Thrombosis and Atherosclerosis Research Institute (TaARI) - McMaster University

Hamilton, Canada

Dr. Davide Matino is an Assistant Professor of Medicine at McMaster University in Hamilton, Ontario, Canada. Dr. Matino obtained his medical degree at the University of Perugia and completed his residency training at the University of Rome “Tor Vergata”. He completed his Clinical Fellowship in Bleeding disorders at McMaster University where he also received his MSc in Health Research Methodology. Prior to joining faculty at McMaster University in 2020 he was working at the University of Perugia as Assistant Professor in the Department of Experimental Medicine, leading a research project focused on the study of the immune response to FVIII in hemophilia, funded by the Italian Ministry of Health. He is actively engaged in the care of patients with bleeding and thrombotic disorders and contributes to education in the field of bleeding disorders locally, nationally and internationally. He is an active clinical trial investigator involved in numerous trials in the field of thrombosis and haemostasis with a specific focus on novel therapies for hemophilia. Dr Matino also directs a basic research lab investigating coagulation factor VIII and factor IX and the molecular mechanisms of hemophilia A and B. His work has received several awards and has been presented both nationally and internationally at the International Society for Thrombosis and Haemostasis, American Society of Hematology, and other scientific meetings.

Head of Thrombosis and Hemostasis Centre, Institute of Hematology and Blood Diseases Hospital

Tianjin, China

Dr. Renchi Yang is Professor and Head of Thrombosis and Hemostasis Center at Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences (CAMS) and Peking Union Medical College (PUMC). Dr. Yang contributes extensively to clinical research and is involved in many clinical trials. He has published over 100 peer-reviewed publications and book chapters on bleeding disorders. He is Steer Committee member of East Asia Hemophilia Forum and the Association for Hemophilia and Allied Disorders-Asia-Pacific (AHAD-AP). He is Vice Chairman of the Thrombosis and Hemostasis Society, China Medicine Education Association. He is also Standing Committee member of the Rare Diseases Society, Chinese Research Hospital Association. He is Chairman of Hemophilia Committee, China Alliance for Rare Diseases. He is Director of the National Hemophilia Registry Center, Chairman of the Hemophilia Treatment Center Collaborative Network of China, Chairman of National Hemophilia Cooperative Group. He is currently the translated edition (Chinese) editor of Haemophilia, the official journal of the World Federation of Hemophilia (WFH).