Speakers: Dr. Len Valentino, Dr. Barbara Konkle, Dr. Glenn Pierce, Dr. Jill Johnsen, Mark Skinner, JD., and Shellye Horowitz.
Join us to discover the latest updates on "My Life, Our Future." This collaborative effort seeks to advance understanding of hemophilia by developing the MLOF Research Repository, a scientific resource. Combining and analyzing genetic and clinical data through this database may allow researchers to solve unmet needs in patients with hemophilia, including understanding inhibitor development and bleeding severity or aiding in identifying new therapeutic targets.
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Dr. Len Va
lentino brings more than 35 years of clinical and research experience related to bleeding disorders to the role, aligning with the board’s strategy of a more research-focused agenda. Prior to his most recent work with Spark Therapeutics, a biotech startup, he founded and led the Hemophilia and Thrombophilia Center at Rush University Medical Center in Chicago, where he successfully balanced a $10 million budget to support research grants, research, and clinical teams – keeping the patient and their families as his core focus. Additionally, he has held leadership roles with key industry groups. In these roles, Dr. Valentino was responsible for managing the global medical teams that successfully led strategic planning for multiple worldwide hemophilia product launches.
He earned his undergraduate and medical degrees from Creighton University and Creighton University School of Medicine. He then completed the University of Illinois at Chicago’s Pediatric Medicine Residency before completing a fellowship in pediatric hematology-oncology at the David Geffen School of Medicine at UCLA. Dr. Valentino remains an active member of multiple professional organizations, including The American Society of Hematology, the International Society of Thrombosis and Hemostasis, the Medical Affairs Professional Society, and The Hemophilia and Thrombosis Research Society.
Dr. Barbara Konkle is a hematologist at the Washington Center for Bleeding Disorders, Professor of Medicine Emeritus in Hematology-Oncology at the University of Washington, and the Medical Director of the Bleeding Disorders Laboratory at Bloodworks NW, all in Seattle. She currently serves on the Board of Directors of the World Federation of Hemophilia and Co-Chairs the Steering Committee for the WFH Gene Therapy Registry. She is also a founding Board member of the Foundation for Women and Girls with Blood Disorders. She has decades of experience in the diagnosis and treatment of bleeding disorders and led the US ‘My Life Our Future’ project, which genotyped over 11,000 patients and potential genetic carriers of hemophilia. Her research includes studies of outcomes in hemophilia and von Willebrand disease, including in women, and trials of new therapies for bleeding disorders. She has published extensively in these areas.
Dr. Glenn Pierce has more than 35 years of experience in bench-to-bedside research and development with several public and private biopharmaceutical companies, including Biogen, Bayer, Inspiration, Avigen, Selective Genetics, and Amgen. At Biogen, Dr. Pierce served as senior vice president of hematology, cell and gene therapies, with overall R&D responsibility for hemophilia and hemoglobinopathies and led the research and clinical development of the first extended half-life FVIII (Factor VIII) and FIX (Factor IX) Fc fusions. He co-founded Ambys Medicines, a cell and gene therapy liver regeneration start-up, where he served as chief medical officer and most recently served as interim CSO of Voyager Therapeutics, a neurodegenerative gene therapy company, where he serves on the board of directors.
Dr. Pierce currently serves as vice president of medical with the World Federation of Hemophilia (WFH). He serves on the U.S. medical and scientific advisory council (MASAC), and previously served on the board of directors and as board president of the National Bleeding Disorders Foundation (NBDF) (previously NHF). Dr. Pierce spearheaded the initiation of the expanded WFH humanitarian aid program, providing clotting factor distribution to over 25,000 people with hemophilia in 74 lower-income countries. He also co-developed the My Life Our Future (MLOF) program in the U.S. to genotype more than 10,000 individuals in the U.S. bleeding disorder community. He currently consults for a number of next-generation gene therapy companies in hemophilia and other therapeutic areas.
Dr. Jill Johnsen is a physician scientist with expertise in classical hematology. Her research program is dedicated to improving the diagnosis and care of patients with blood disorders through advancement of our basic understanding of the underlying biology and through the translation of new knowledge and laboratory innovations to improve clinical testing. She is particularly interested in better understanding the impact of DNA variation on bleeding and how bleeding uniquely impacts females. Her research leverages new technologies, including targeted and whole genome next generation DNA sequencing, multi-omics, long-read sequencing, and new and novel molecular methods including multiplex assays of variant effects (MAVEs). Dr. Johnsen was part of the MLOF U.S. national hemophilia genotyping effort, and she now maintains the MLOF Biorepository and co-leads the MLOF Research Repository with Dr. Konkle.
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 at the Department of Health Research Methods, Evidence and Impact at McMaster University. He has previously led both national and international patient organizations, including the World Federation of Hemophilia and the National Hemophilia Foundation. 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 serving 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 of State Programs at the American Insurance Association and Administrative Assistant/Chief of Staff to the Speaker of the Kansas House of Representatives. He holds a degree in public and business administration from Kansas State University and a JD from Washburn University School of Law.
G Shellye Horowitz, MA, PPS, has strong ties to the bleeding disorders community with six traceable generations of hemophilia A in her family. Shellye inherited hemophilia from her father. Her advocacy work includes an emphasis on early diagnosis and care for females with hemophilia and strengthening diagnosis and care for all people with mild bleeding disorders. Shellye currently works for the University of Washington as a Program Coordinator in the Johnsen Lab.