David Baron, Ph.D., received his B.A. (Biology) and Ph.D. (Anatomy) from The University of Chicago. Following his postdoctoral fellowship in pathology and pharmacology at the Medical University of South Carolina (MUSC), with the support of a National Institutes of Health Program Project grant, Dr. Baron founded the Core Structure-Function Laboratory in the Department of Pharmacology and joined the MUSC faculty with a joint appointment in the Departments of Pharmacology, and Anatomy and Cell Biology. He later joined Searle Pharmaceuticals as a research scientist, later becoming a Monsanto, then Pharmacia senior science fellow. Dr. Baron became the first Director of Toxicology at Takeda Pharmaceuticals, U.S. (Deerfield, Illinois) rising to Vice President, Nonclinical Safety Evaluation, for the United States and Europe. He has been a grant reviewer for the National Cancer Institute, given numerous invited seminars and has served on several national scientific boards (International Consortium for Innovation and Quality in Pharmaceutical Development, International Serious Adverse Event Consortium). Dr. Baron, who has polycystic kidney disease, became the Chief Scientific Officer for the Polycystic Kidney Disease (PKD) Foundation in 2015. During his career he has focused on the structural correlates of electrolyte and water transport across epithelia, and the safety and pharmacology of pioglitazone while at Takeda, a type 2 diabetes drug now in a pilot clinical trial for the treatment of PKD through the support of the PKD Foundation and the Food and Drug Administration (FDA). He will begin a 3-year term as a member of the board of the Kidney Health Initiative January 2017.
Peter Coffey, D.Phil., is the director of the London Project to Cure Blindness and a professor of cellular therapy and visual sciences at the Institute of Ophthamology, University College London (UCL). His achievements include the launch of the London Project to Cure Blindness, which aims to develop a stem cell therapy for the majority of all types of age-related macular degeneration; seminal work on retinal transplantation; and the development of a cell-based therapy for a currently untreatable form of macular degeneration, age-related macular degeneration (also called dry AMD). He is the principal author and coauthor of two landmark papers demonstrating the use of human cells to halt visual deterioration in models of dry AMD. Also, Dr. Coffey has a laboratory at the University of California, Santa Barbara (UCSB), and is co-director of UCSB’s Center for the Study of Macular Degeneration. He is the director of translation at UCSB’s Center for Stem Cell Biology and Engineering and is a member of the Neuroscience Research Institute.
Dr. Coffey has received many honors and awards, including the prestigious Estelle Doheny Living Tribute Award in 2009, Retinitis Pigmentosa International’s Vision Award in 2009, the California Institute for Regenerative Medicine (CIRM) Leadership Award in 2010, and recently the New York Stem Cell Foundation’s Robertson Award for translation stem cell work. CIRM reviewers characterized Dr. Coffey’s work as “truly innovative, novel, ambitious and important . . . highly significant, with a potential to revolutionize the field.” He is engaged in public service endeavors to explain stem cell research to the lay public, including talks to the British Parliament and the Vatican. Dr. Coffey received his D.Phil. degree at Oxford University and was a member of the faculty at Oxford and later the University of Sheffield, as lecturer and senior lecturer, before joining the faculty at UCL as head of the Ocular Biology and Therapeutics Research Department.
George Q. Daley, M.D., Ph.D., is the dean designate of Harvard Medical School and the Robert A. Stranahan Professor of Pediatrics and a professor of biological chemistry and molecular pharmacology at Harvard Medical School. He is also the director of the Stem Cell Transplantation Program at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and a Howard Hughes Medical Institute investigator.
Dr. Daley is a world-renowned expert on stem cells, cancer, and blood disorders. He received his bachelor’s degree, magna cum laude, from Harvard University (1982), a doctorate in biology from the Massachusetts Institute of Technology (1989), where he worked with Nobel
laureate David Baltimore, and his medical degree from Harvard Medical School (1991), where he was only the 12th individual in the school’s history to receive the degree summa cum laude.
Dr. Daley pursued clinical training in internal medicine at Massachusetts General Hospital, where he served as chief resident (1994–1995), and a clinical fellowship in hematology/oncology at Brigham and Women’s Hospital and Boston Children’s.
He was a founding member of the executive committee of the Harvard Stem Cell Institute and served as president of the International Society for Stem Cell Research from 2007 to 2008 and as its clerk from 2012 to 2015. He anchored the special task forces that produced the society’s guidelines for stem cell research (2006) and clinical translation (2008) and their subsequent revisions and updates (2016).
Dr. Daley’s research uses mouse and human disease models to unravel the mechanisms that underlie various cancers and blood disorders. Important contributions from the Daley laboratory include the creation of customized stem cells to treat a genetic immune deficiency in mice, the differentiation of germ cells from embryonic stem cells, the generation of disease-specific pluripotent stem cells by direct reprogramming of human skin and blood cells, and demonstration of the role of the LIN28/let7 signaling pathway in the development of cancer.
Previously, Dr. Daley’s work demonstrated the central role of the BCR/ABL protein in the development of human chronic myeloid leukemia (CML), a finding that provided the critical target validation for the development of Gleevec, a highly successful treatment for this disease.
Dr. Daley has been elected to the National Academy of Medicine, the American Society for Clinical Investigation, the American Association of Physicians, the American Pediatric Societies, the American Academy of Arts and Sciences, and the American Association for the Advancement of Science.
Dr. Daley was an inaugural winner of the National Institutes of Health Director’s Pioneer Award for highly innovative research and has received the Judson Daland Prize from the American Philosophical Society for achievement in patient-oriented research, the E. Mead Johnson Award from the American Pediatric Society for contributions to stem cell research, and the E. Donnall Thomas Prize of the American Society of Hematology for advances in human-induced pluripotent stem cells.
Jennifer Fields, M.P.H., a North Carolina native, was diagnosed at the age of 2 months with sickle cell anemia (HgbSS) disease. Ms. Fields possesses a broad background in health and human services and nonprofit operations, including strategic development and grant management, totaling more than 14 years of experience. She obtained a master of public health degree from East Carolina University and is presently working as a consultant with the Sickle Cell Treatment Demonstration Program at Sickle Cell Disease Foundation California and the Sickle Cell Disease Newborn Screening Program at the Sickle Cell Disease Association of America in Baltimore, Maryland. Ms. Fields’s work has included managing several federal awards, including awards from the National Institutes of Health, Health Resources and Services Administration, Centers for Disease Control and Prevention, and the Federal Emergency Management Agency at the Department of Homeland Security, totally several millions of dollars. Ms. Fields now resides in Raleigh, North Carolina, with her 4-year-old son, Roman.
Patricia Furlong is the founding president and chief executive officer of Parent Project Muscular Dystrophy (PPMD), the largest nonprofit organization in the United States solely focused on Duchenne muscular dystrophy (Duchenne). Its mission is to end Duchenne. It accelerates research, raises its voice in Washington, demands optimal care for all young men, and educates the global community.
Duchenne is the most common fatal, genetic childhood disorder, and it affects approximately 1 out of every 4,600 boys each year worldwide. It currently has no cure.
When doctors diagnosed her two sons, Christopher and Patrick, with Duchenne in 1984, Ms. Furlong did not accept “there’s no hope and little help” as an answer. She immersed herself in Duchenne, working to understand the pathology of the disorder, the extent of research investment, and the mechanisms for optimal care. Her sons lost their battle with Duchenne in their teenage years, but she continues to fight—in their honor and for all families affected by Duchenne.
In 1994, Ms. Furlong, together with other parents of young men with Duchenne, founded PPMD to change the course of Duchenne and, ultimately, to find a cure. Today, she continues to lead the organization and is considered one of the foremost authorities on Duchenne in the world.
Brigitte Gomperts, M.D., is a physician-scientist at the University of California, Los Angeles (UCLA), in the Departments of Pediatrics and Pulmonary Medicine. She earned her medical degree from the University of Witwatersrand in Johannesburg, South Africa, and trained in general pediatrics and pediatric hematology/oncology at Washington University in St. Louis, Missouri. She is an associate vice chief of education in the Department of Pediatrics, the vice chief of research for the Pediatric Hematology-Oncology Division, and a co-director of the Jonsson Comprehensive Cancer Center Cancer Stem Cell and Biology Program at UCLA. She is also affiliated with the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA and the Molecular Biology Institute.
Her research focuses on the repair and regeneration of the lungs and how the normal repair mechanisms go awry in lung diseases. Her lab is using novel human three-dimensional models to understand repair and regeneration in the proximal and distal lung and has expertise in adult stem cell models of lung diseases and patient-specific induced pluripotent stem cell lung disease modeling and high-throughput drug screening. The ultimate goal is to use this knowledge to develop novel targeted therapies and prevention strategies for lung diseases. Major areas of interest include lung fibrosis, mucociliary clearance, chronic obstructive pulmonary disease, and premalignant lesions with stepwise progression to lung cancer. Because she is a physician-scientist, her lab is particularly interested in translational research that will result in new therapies for lung diseases.
Benjamin D. Humphreys, M.D., Ph.D., is the Joseph Friedman Associate Professor in Renal Diseases and the chief of the Division of Nephrology at Washington University School of Medicine in St. Louis. Prior to joining Washington University in St. Louis, Dr. Humphreys was the director of the Harvard Stem Cell Institute Kidney Program and an associate professor of medicine at Harvard Medical School and Brigham and Women’s Hospital. Dr. Humphreys is a member of the American Society of Clinical Investigation and an established investigator of the American Heart Association. He is the recipient of the National Kidney Foundation Young Investigator Award and the American Society of Nephrology Gottschalk Research Scholar Award. His National Institutes of Health–funded laboratory focuses on adult kidney injury and repair. The laboratory has special expertise in genetic mouse models of kidney disease, stem cell biology, and the generation of kidney organoids from human
pluripotent stem cells, and it employs these approaches to identify new treatments for patients suffering from kidney disease. Dr. Humphreys earned his bachelor of arts degree from Harvard College and his medical and doctor of philosophy degrees from Case Western Reserve University. He completed a residency in internal medicine at Massachusetts General Hospital and a fellowship in nephrology at Brigham and Women’s Hospital in Boston. He has authored more than 100 publications and multiple book chapters, and he holds 5 patents.
Harry L. Malech, M.D., is the chief of the Laboratory of Host Defenses and the Genetic Immunotherapy Section in the National Institute of Allergy and Infectious Diseases of the National Institutes of Health (NIH). At NIH, Dr. Malech cares for and studies patients who have a variety of inherited immune deficiencies, with a long-term focus on children and young adults with chronic granulomatous disease (CGD) or X-linked severe combined immune deficiency (XSCID). His clinical service has research programs of study of allogeneic hematopoietic stem cell transplant and autologous stem cell ex vivo gene therapy for CGD, SCID, and other immune deficiencies. Laboratory research is focused on achieving efficient genetic correction of patient hematopoietic stem cells. Related work includes studies of the generation of induced pluripotent stem cells from patients with CGD and XSCID and the use of gene targeting methods to genetically correct induced pluripotent stem cells or hematopoietic stem cells. Dr. Malech is an elected member of the Association of American Physicians and the American Society for Clinical Investigation. He is a recent past president for 2014–2015 of the American Society of Gene & Cell Therapy.
Eduardo Marbán, M.D., Ph.D., is an international leader in cardiology and a pioneering heart researcher. His 30-plus years of experience in patient care and research have led to key discoveries in gene and stem cell therapies for heart disease. Those discoveries have formed the basis for multiple startup companies.
Dr. Marbán attended public schools through high school and later Wilkes College, where he earned a B.S. in mathematics. Thereafter, Dr. Marbán completed a combined M.D./Ph.D. program at Yale University. Postgraduate training took him to the Osler Medical Service at the Johns Hopkins University, where he eventually spent 25 productive years. During his tenure there, he served in a variety of academic and research positions, including chief of cardiology.
In his research career, Dr. Marbán, a cellular electrophysiologist by training, has pursued questions of relevance to heart disease (ischemia, heart failure, and arrhythmias). The Marbán laboratory elucidated the fundamental pathogenesis of myocardial stunning, pioneered the concept of gene therapy to alter electrical excitability, and created the first de novo biological pacemaker as an alternative to electronic pacemakers. He first became interested in stem cells in 2002, building on his work on biological pacemakers. Since 2004 the lab has been intensively studying cardiac progenitor cells, in particular, their origins and their therapeutic potential. The basic work has come full circle in that Dr. Marbán’s cardiac-derived cell products form the basis for four grant-funded clinical trials, one of which has been completed (CADUCEUS) and the other three ongoing (ALLSTAR, DYNAMIC, and HOPE-Duchenne). The CADUCEUS trial was the first to show that cell therapy can repair “irreversible” tissue damage caused by heart attacks, ushering in the concept of therapeutic regeneration in humans.
In 2007 Dr. Marbán became the founding director of the Cedars-Sinai Heart Institute, a multidisciplinary entity that brings together adult and pediatric cardiologists, cardiac surgeons, imaging specialists, and researchers to foster discovery and enhance patient care. The institute is built on a long tradition of excellence and innovation at Cedars-Sinai, including the invention of the Swan–Ganz catheter. The Cedars-Sinai Heart Institute, ranked as the top heart program in the western United States, performs more heart transplants annually than any other institution worldwide.
Among the many honors Dr. Marbán has received are the Basic Research Prize of the American Heart Association (AHA), the Research Achievement Award of the International Society for Heart Research, the Gill Heart Institute Award, and the distinguished scientist awards of the AHA and the American College of Cardiology.
Anthony Oro, M.D., Ph.D., is a professor of dermatology, associate director of the Center for Definitive and Curative Medicine, a member of the Institute for Stem Cell Biology and Regenerative Medicine and the Stanford Cancer Institute at Stanford University, and the Cancer Biology and Stem Cell graduate student programs. He trained in the medical scientist program at the Salk Institute under Ron Evans lab, working on developmental functions of novel orphan nuclear receptors in model systems. During dermatology residency/fellowship in Matthew Scott’s lab at Stanford, he helped solidify the link between the hedgehog path-
way and human cancer. In his own lab in the Program in Epithelial Biology at Stanford, Dr. Oro has extended the original studies focusing on the role of skin stem cells to understand in tissue regeneration and carcinogenesis. He has a long-standing interest in the mechanisms of hedgehog signaling in hair follicle regeneration, and in the pathogenesis of the most common human tumor, basal cell carcinoma of the skin. As a practicing physician, he led the clinical trials developing the first human hedgehog pathway inhibitor in skin cancer. His recent focus is on tumor evolution and novel resistance-associated signaling pathways. His interest in the mechanisms of human skin development and early ectodermal differentiation has led to the development of Therapeutic Reprogramming, the use of in vitro human skin differentiation protocols and genome editing tools to produce clinical grade, corrected, autologous human skin from patient-specific induced pluripotent (iPS) cells. He is focusing his efforts to treat the blistering disease Epidermolysis bullosa. Dr. Oro has received numerous awards including the Marion Sulzberger Memorial and Montagna Awards, and is a member of the American Society for Clinical Investigation. Dr. Oro has 18 patents and published more than 70 peer-reviewed articles, commentaries and chapters.
Anthony Ratcliffe, Ph.D., is president and CEO of Synthasome, Inc. Dr. Ratcliffe obtained his B.Sc. in biochemistry in 1977, and Ph.D. in immunology in 1980, from the University of Birmingham, United Kingdom. He then joined The Kennedy Institute for Rheumatology, London as a research scientist, and in 1987 he moved to Columbia University, New York, as associate professor in the Department of Orthopaedic Surgery. In 1996 he joined Advanced Tissue Sciences in La Jolla, where he served as vice president for Research until 2002. Dr. Ratcliffe has focused his work on musculoskeletal research, tissue engineering, and product development. He has had a number of external leadership positions, including serving as a member of the Board of Directors of the Orthopaedic Research Society, Study Sections for the National Institutes of Health, co-chairman of the Grant Review Committee for the Orthopaedic Research and Education Foundation, co-chairman of the Tissue Engineering Committee for the American Society for Testing and Materials, and has published more than 100 papers.
Michel Sadelain, M.D., has made major contributions to the generation and optimization of CAR T cells to treat cancer as well as to the devel-
opment of stem cell therapies for blood disorders. Dr. Sadelain’s work has focused on developing novel strategies to extend the survival of CAR T cells in the body and to enable T cells with increased potency to overcome the resistance imposed by tumor and other cells in the tumor microenvironment. In 2002 his group was the first to report the design of “second-generation” CARs that, in addition to a binding domain outside of the T cell and a signaling domain inside, included a costimulatory domain designed to promote cell proliferation and survival. These advances provided a broad platform to enhance CAR T cell therapy, leading directly to the development of new CAR T cell therapies that are showing increasing efficacy in patients. Building on proof-of-principle experimentation in mice bearing CD19+ malignancies, the Memorial Sloan Kettering Cancer Center (MSKCC) team led by Dr. Sadelain has recently obtained dramatic clinical responses in adult patients with acute lymphoblastic leukemia.
Dr. Sadelain is the founding director of the Center for Cell Engineering and the head of the Gene Transfer and Gene Expression Laboratory at MSKCC, where he holds the Stephen and Barbara Friedman Chair. Dr. Sadelain is also a member of the departments of medicine and pediatrics at Memorial Hospital and the molecular pharmacology and chemistry program of the Sloan Kettering Institute.
Dr. Sadelain received his M.D. from the University of Paris, France, in 1984 and his Ph.D. from the University of Alberta, Canada, in 1989. After completing a clinical residency at the Centre Hospitalier Universitaire Saint-Antoine in Paris, Dr. Sadelain carried out a postdoctoral fellowship with Richard Mulligan, Ph.D., at the Whitehead Institute for Biomedical Research at the Massachusetts Institute of Technology, before joining MSKCC in 1994 as an assistant member. Dr. Sadelain is a member of the American Society of Hematology, the American Society of Human Genetics, and the American Society of Cell and Gene Therapy, where he served on the board of directors from 2004 to 2007, and he is an elected member of the American Society for Clinical Investigation. He has authored more than 150 scientific papers and book chapters.
Deepak Srivastava, M.D., is the director of the Gladstone Institute of Cardiovascular Disease and the Roddenberry Stem Cell Center at Gladstone and is also a professor at the University of California, San Francisco (UCSF), Medical Center. Dr. Srivastava received his B.S. from Rice University and his M.D. from The University of Texas, and he
trained in pediatrics at UCSF and in pediatric cardiology at Harvard Medical School.
Dr. Srivastava’s laboratory discovered genetic bases for cardiac septal and valve defects and revealed complex signaling, transcriptional, and translational networks that regulate progenitor cells to adopt a cardiac cell fate and subsequently fashion a functioning heart. He has leveraged this knowledge to reprogram fibroblasts directly into cardiomyocyte-like cells for regenerative purposes. Dr. Srivastava is a member of the American Academy of Arts and Sciences and the National Academy of Medicine.
Lorenz Studer, M.D., is the director of the Center for Stem Cell Biology and a member of the Developmental Biology Program at Memorial Sloan Kettering. A native of Switzerland, he received his M.D. and doctorate degree from the University of Bern where he co-developed the first cell-based therapy for Parkinson's disease in the country. He subsequently trained as postdoctoral fellow with Ron McKay at the National Institutes of Health pioneering the therapeutic application of neural stem cell–derived neurons in models of neurodegeneration. In his laboratory, he has established techniques that can turn human pluripotent stem cells into many of the diverse cell types of the nervous system. He was also among the first to realize the potential of patient-specific stem cell in modeling human disease and in drug discovery. Furthermore, he is currently leading a multidisciplinary consortium to pursue the first clinical application of human stem cell–derived dopamine neurons for the treatment of Parkinson’s disease. Dr. Studer’s work has been recognized by numerous awards including the Boyer Young Investigator Award and the Annemarie Opprecht Award.
Sally Temple, Ph.D., is the co-founder and scientific director of the Neural Stem Cell Institute, located in Rensselaer, New York. Dr. Temple’s group is focused on studies of neural stem cells and on using this knowledge to develop therapies for central nervous system disorders.
Dr. Temple trained at Cambridge University and University College London with Dr. Martin Raff, FRS. In 1989, Dr. Temple discovered that the embryonic mammalian brain contained a rare, multipotent stem cell that could be grown in tissue culture, producing both neurons and glia. Since then, her group has continued to make pioneering contributions to the field of neural stem cell research, identifying cell-intrinsic and extracellular niche factors that participate in their self-renewal and differentia-
tion into diverse cell types. Using patient-derived neural, retinal pigment epithelial, and induced pluripotent stem cells, her research group is building novel models to study disease mechanisms of age-related neurodegeneration, with the aim of identifying new targets to slow or stop the disease process. In recognition of her work, Dr. Temple has received the Royal Society Stothert Research Fellowship, the Javits National Institutes of Health merit award, the MacArthur award and the Ellison investigator award. Dr. Temple is currently the president of the International Society for Stem Cell Research.
Ann Tsukamoto, Ph.D., has been working in the stem cell field for almost 27 years. Her most recent position was executive vice president for Scientific and Strategic Alliances at StemCells, Inc. During her 18 year tenure at StemCells, Dr. Tsukamoto led the scientific team that discovered the human central nervous system stem cell (HuCNS-SC®) and a second candidate stem cell for the liver and that transitioned the human neural stem cell into early clinical development in all three components of the CNS: brain, spinal cord, and eye. The biological potential and activity of these HuCNS-SC® cells was demonstrated in some patients and reflected results seen in preclinical rodents studies. The many challenges of developing a cell therapy in a small biotech firm led to the closure of StemCells, Inc., in August 2016.
Prior to her time at StemCells, Inc., Dr. Tsukamoto worked at the first stem cell company, SyStemix, Inc., where she co-discovered the human hematopoietic stem cell (hHSC) and played a leading role in the launch of the clinical research program for this cell. The purified hHSC was shown to be cancer-free when isolated from the cancer-contaminated hematopoietic mobilized blood of patients with disseminated cancer, and it successfully regenerated the patients’ blood-forming system after myeloablative chemotherapy. Dr. Tsukamoto is an inventor on seven issued U.S. patents, of which six are related to the human hematopoietic stem cell. She received her Ph.D. in microbiology and immunology at the University of California, Los Angeles, and did her postdoctoral work with Dr. Harold Varmus at the University of California, San Francisco, where she worked on the wnt-1 gene and developed a transgenic model for breast cancer. Wnt-1 was later discovered to be a key player in the stem cell self-renewal pathway.
Fyodor Urnov, Ph.D., is an associate director at the Altius Institute for Biomedical Sciences. Prior to joining Altius in August 2016, Dr Urnov
was the vice president of discovery and translational research at Sangamo BioSciences, where he most recently led an effort to expand genome editing and targeted gene regulation technologies to new disease indications, including beta-thalassemia and sickle cell anemia. As a co-developer of genome editing, Dr. Urnov led the company’s research and development efforts to deploy genome editing for crop trait engineering in partnership with Dow AgroSciences, and he co-managed Sangamo’s partnership with Sigma-Aldrich for the generation of engineered cell lines for manufacturing, transgenic animals, and research reagents.
He is an author of more than 70 scientific publications and an inventor on more than 100 issued and pending U.S. patents related to genome editing and targeted gene regulation technology. Dr. Urnov is also an adjunct professor in the department of Molecular and Cell Biology at the University of California, Berkeley, where he teaches upper-division undergraduate and graduate classes in the life sciences as well as Biology for Voters, a class for students who are not majoring in the sciences. Pri- or to joining Sangamo, Dr. Urnov was a postdoctoral fellow at the National Institutes of Health in the laboratory of Alan P. Wolffe, where he trained in the study of chromatin-based genome regulatory processes in metazoa. Dr. Urnov received a B.Sc. in biology from Moscow State University and his Ph.D. in biology from Brown University, where he studied chromatin-based integration of genome control in the laboratory of Susan A. Gerbi.