Advances in Rare Diseases: Hemophilia

Glenn Pierce is currently an Independent Consultant and Entrepreneur-in-residence at Third Rock Ventures. He serves on the World Federation of Hemophilia (WFH), WFH USA Board of Directors, the National Hemophilia Foundation (NHF) Medical and Scientific Advisory Council (MASAC). He is also a director of Voyager Therapeutics and Global Blood Therapeutics, two publicly held biotechnology companies, and the Chief Medical Officer of Ambys Medicines, a privately held liver regeneration company he co-founded through Third Rock Ventures in 2018.
Glenn retired from Biogen in 2014 as senior vice president of Haematology, Cell and Gene Therapies. He had overall R&D responsibility for haemophilia and hemoglobinopathies and led the research and clinical development of the first extended half-life FVIII and FIX Fc fusions as Chief Medical Officer since joining the company in 2009.
Glenn received an MD and a PhD in Immunology, both from Case Western Reserve University in Cleveland, Ohio. He did his postgraduate training in pathology and haematology research at Washington University in St. Louis, Missouri.
He has 30 years’ experience in biotechnology research and development, including Amgen, Selective Genetics, Avigen, Inspiration, Bayer and Biogen in the therapeutic areas of tissue regeneration and haematology. At Amgen he founded the neurobiology and experimental pathology departments. He is an author of more than 150 scientific papers and has been awarded over 15 patents in various areas of drug delivery, tissue engineering, medical devices, protein engineering, and viral vectors. Dr. Pierce served on the Medical and Scientific Advisory Council, the Board of Directors and was President of the NHF variously for over 20 years. He founded NHF’s annual/biannual gene therapy and new technologies workshop in 1996 to further collaboration and advancements in bleeding disorders research.
Dr Pierce lived with severe haemophilia A until 2008, when he received a liver transplant. He now spends a substantial amount of time on WFH development and humanitarian aid efforts in the developing world. He is based in San Francisco and La Jolla, California.

Dr. Hui Cai joined WuXi AppTec in 2009 as Vice President of Business Development, and is currently Vice President and Head of Content Division.
Prior to WuXi, Dr. Cai spent 10 years at Johnson & Johnson Pharmaceutical Research and Development leading multiple drug discovery programs in the therapeutic areas of inflammation and autoimmune diseases. She is a co-author and co-inventor to over 50 scientific publications and issued or pending patents. Dr. Cai is a Councilor of the American Chemical Society (ACS), a member of BayHelix, and a member of the UCSD Alumni Board. In her past capacity, she served as a Commissioner at the City of San Diego Science and Technology Commission, Chair of SABPA, and President of SDCA. Dr. Cai received her BS and MS in Chemistry from Peking University, PhD from The Scripps Research Institute, and MBA from UCSD Rady School of Management as a DLA Piper – Athena Scholar.

Dr. Federico Mingozzi is the chief scientific officer at Spark Therapeutics, bringing two decades of experience in gene therapy, immunology, as well as biochemistry and molecular biology in both industry and academic settings.
Federico began his scientific career studying the genetic bases of bleeding disorders. At the Children’s Hospital Philadelphia (CHOP) he conducted pioneering studies on liver gene transfer with adeno-associated virus (AAV) vectors and immunology. Federico was involved in several first-in-human clinical studies of gene therapy based on the AAV vector platform while serving as the director of translational research the Center for Cellular and Molecular Therapeutics, at CHOP. He also led studies aimed at the characterization of human immune responses to AAV vectors and the development of strategies to modulate vector immunogenicity.
He then joined the French National Institute of Health and Medical Research (INSERM) as Research Director and Genethon, a leading French nonprofit R&D organization focused on gene therapy for rare diseases, as Team Leader. There, he spearheaded the development of in vivo gene therapies for inherited diseases. His work continued to focus on the characterization of human immune responses to AAV vectors and on the development of strategies to overcome immune responses in gene transfer.
Federico currently serves as faculty at the Pierre and Marie Curie University in Paris, France, and Universitat Autonoma de Barcelona, Spain. He received his bachelor’s degree in biology and his Ph.D. in biochemistry and molecular biology from the University of Ferrara in Italy, and his M.B.A. from Drexel University. Throughout his distinguished career, Federico has received several awards and has contributed to more than 90 publications, including seminal findings in the field of AAV gene therapy.

John Pasi has a clinical practice that spans both adults and children and covers all aspects of haemostasis and thrombosis. His particular research interests are new bioengineered therapies for haemophilia, novel therapies for haemophilia including gene therapy and RNAi technologies. He is closely involved in the design and development of trials for therapies in phase 1-4 programmes. He has been a part of the development of national policy / guideline formulation for haemostatic and thrombotic disorders and training initiatives such as residential academies, preceptorships and online education / training. Previously an advisor to the Parliamentary Health Select Committee, he is a member of NHS England Clinical Reference Group (GRG) for Inherited Bleeding Disorders, and co-chair of the ISTH Scientific and Standardization Committee on factor VIII, factor IX and rare coagulation disorders and the WFH Gene Therapy Roundtable.

Richard Pezzillo is the Executive Director of the New England Hemophilia Association (NEHA). Prior to joining NEHA, Richard worked in Washington, DC on Capitol Hill as the press secretary for Senator Sheldon Whitehouse and as the Communications and Marketing Director for the Hemophilia Federation of America (HFA). Richard is the former co-chair of the National Hemophilia Foundation’s Youth Leadership Institute. He is also the recipient of the Ryan White Meritorious Service Award, and Association for Healthcare Philanthropy and Providence Business News “40 under 40” award. Richard graduated with a BS from Western Connecticut State University and currently resides in Providence, Rhode Island.

During his more than 25 years as a physician and an industry executive, Dr. Vivaldi has developed a reputation as a devoted advocate for patients.
Most recently, he served as Executive Vice President and Chief Global Therapeutic Operations Officer at Bioverativ, which was acquired in 2018 by Sanofi. Prior to Bioverativ, Dr. Vivaldi was Chief Commercial Officer at Spark Therapeutics, where he worked to commercialize the company’s gene therapeutics. He also managed Spark’s Medical Affairs and Patient Advocacy groups.
Prior to Spark, Dr. Vivaldi co-founded and served as President and CEO of Minerva Neurosciences, a clinical-stage company developing products for patients with neuropsychiatric diseases. Before co-founding Minerva, he led Genzyme’s rare disease business as President of both the rare disease business and the renal & endocrine group, as well as Senior Vice President and General Manager of Genzyme’s Latin America Group.
Dr. Vivaldi holds his medical degree from the Universidade do Rio de Janeiro Medical School. He completed a residency in endocrinology at Universidade do Estado do Rio de Janeiro and a fellowship at Mount Sinai Hospital Center in New York in the department of genetics, focusing on Gaucher disease. He also holds an MBA degree from COPPEAD, Universidade Federal do Rio de Janeiro.

Guy Young, MD, received his medical degree from the State University of New York at Stony Brook School of Medicine in Stony Brook, New York. He completed a residency in pediatrics at Schneider Children’s Hospital at Long Island Jewish Medical Center, Albert Einstein College of Medicine in New York, and a fellowship in pediatric hematology/oncology at Children’s National Medical Center, George Washington University School of Medicine in Washington, D.C.
Dr. Young is the Director of the Hemostasis and Thrombosis Center and the Clinical Coagulation Laboratory at Children’s Hospital Los Angeles and a Professor of Pediatrics at the University of Southern California’s Keck School of Medicine.
His current research activities encompass the use of thrombin generation and thromboelastography with novel hemophilia agents as well in conducting clinical trials in hemophilia both his own and those sponsored by industry.
Dr. Young has published over 130 journal articles, numerous abstracts, and several textbook chapters. He has delivered lectures, workshops, and presentations at national and international meetings. He is a past Board member and past chair of the program committee and review course committees for the American Society of Pediatric Hematology/Oncology, and also a past chair of the program committee clinical research award committee for the Hemophilia and Thrombosis Research Society. He is a past chair of the Scientific and Standardization Committee on Factor VIII, Factor IX, and Rare Bleeding Disorders of the International Society on Thrombosis and. He has been awarded the National Hemophilia Foundation Physician of the Year Award in 2013.

Dr. Richard Soll is presently Senior Advisor, Strategic Initiatives for the Research Service Division at WuXi AppTec and Head of the WuXi office in Boston, Massachusetts. He has held various leadership roles at WuXi as Senior Vice President including head of the medicinal chemistry unit known as the International Discovery Service Unit, business development, and corporate alliances.
Dr. Soll’s contributions led to the discovery of the HCV NS5A inhibitor elbasvir (a component of Zepatier®) in the Merck-WuXi partnership, the JAK2 inhibitor fedratinib at TargeGen which formed the basis of the $7B partnership between Celgene and Impact Biomedicines, and more than 10 other clinical stage drugs throughout his career.
Dr. Soll is currently a board member at Simcha Therapeutics, an board observer at the Accelerator Life Science Parnters, and an advisory board member with the Blavatnik Center for Drug Discovery at Tel Aviv University, the Children’s Tumor Foundation, and the Pistoia Alliance.
Dr. Soll was CSO / VP of R&D at TargeGen and was VP of Chemistry at 3-Dimensional Pharmaceuticals. He started his career at Ayerst Research Labs and was trained as a synthetic chemist at Dartmouth and Harvard.