RFA 10-05: CIRM Disease Team Therapy Development Awards Review Meeting | May 23 – 25, 2011 Scientific and Medical Research

Funding Working Group (Grants Review Working Group) SPECIALIST ROSTER Specialist Reviewer Gyula Acsadi Merry Lee Bain Edward Berger Michael Boulton Robert Paul Johnson Henry Kaplan Douglas Kerr Pamela Robey Mauricio Rojas Anne Rosser Josh Rubin John Wagner Andrew Whitelaw Affiliation The Children‟s Hospital of Michigan NDA Partners LLC NIH, NIAID University of Florida Harvard University Kentucky Lions Eye Center Biogen Idec NIH, NIDCR University of Pittsburgh Medical Center Cardiff University Washington University in St. Louis University of Minnesota University of Bristol

SPECIALIST BIOS Gyula Acsadi, M.D., Ph.D. Dr. Acsadi is the Clinical Division Chief of Pediatric Neurology and Co-Director of the Muscular Dystrophy Association Clinic at The Children‟s Hospital of Michigan and Associate Professor in the Department of Pediatrics and Neurology at Wayne State University. He received his M.D. and completed a Neurophysiology Fellowship and a Residency in Pediatrics at the Medical University of Pecs, Hungary. He received his Ph.D. in Molecular Genetics from the Hungarian Academy of Sciences. He also completed a Residency in Pediatric Neurology at The Children‟s Hospital of Michigan. Dr. Acsadi has held the position of Visiting Scientist in the Department of Pediatrics in the Waisman Center at the University of Wisconsin-Madison where he studied gene therapy, and the position of Visiting Assistant Professor in the Neuromuscular Research program at the Montreal Neurological Institute at McGill University before moving to The Children‟s Hospital of Michigan. He is Board certified by the American Board of Psychiatry & Neurology as a neurologist with special qualifications in child neurology. Throughout Dr. Acsadi‟s professional career, his clinical interest has been in pediatric neurological diseases in general but with main emphasis in researching and treating neuromuscular disease including muscular dystrophies and motor neuron diseases. This interest has led to his involvement in clinical trials for spinal muscular atrophy (SMA). He has participated in designing and conducting these trials and served as site PI in multicenter trials. Recently, he was responsible for putting together the “Pediatric Pilot Project” for the National Institutes of Health (NIH) Rare Diseases Clinical Research Consortia (RDCRC) sponsored “Inherited neuropathy consortium” grant. During and shortly after completing medical school, Dr. Acsadi was interested in behavioral and electrophysiological studies of the limbic system. With the advancements of the molecular genetic era, he was involved in early gene therapy work for muscular dystrophy that led to a publication as first author in Nature. Subsequently, Dr. Acsadi‟s research interests have focused on viral and non-viral gene transfer in animal models for muscular dystrophy and amyotrophic lateral sclerosis (ALS). During the past 5-6 years, he has been working on the molecular mechanisms of disease process of spinal muscular atrophy (SMA) using cell culture model systems. Dr. Acsadi is a member of the Child Neurology Society of USA and the American Academy of Neurology. He has been listed in Best Doctors in America and Top Doctors. He has published over 40 articles in peer-referred journals. Dr. Acsadi is active in scholarly review for granting agencies, including the NIH, and for scientific peerrefereed journals. His research has been supported by several funding agencies including the NIH, the Muscular Dystrophy Association, Project Cure SMA, and Families of SMA. Merry Lee Bain, MS Ms. Bain is Member at NDA Partners LLC, a unique international consultancy with a single focus – to dramatically improve the success rate and development speed of our clients' biopharmaceutical and medical device discoveries. Working primarily with
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investor-driven emerging companies, NDA Partners LLC helps their clients make the critical decisions with respect to their product development investments, recognize and understand the scientific and regulatory issues that will impact their programs, avoid the common pitfalls that have derailed other companies and drug development programs, and design and implement optimal product development and regulatory programs. Dr. Bain is a biomedical engineer with more than 25 years of regulatory affairs experience in the medical device industry. She has played an integral role in the development of a wide variety of medical devices, including orthopedic implants, vascular catheter-based technology, tissue-based devices, and combination products. Her primary focus has been on steering new products through the FDA approval process, including the design and management of preclinical and clinical studies, and writing and overseeing FDA submissions. Prior to joining NDA Partners, she held executive officer positions at Cook Medical companies, and worked with the FDA as an industry representative on a number of task forces for developing FDA guidelines and policies. Edward A. Berger, Ph.D. Dr. Berger is Chief of the Molecular Structure Section in the National Institutes of Allergy and Infectious Diseases (NIAID) Laboratory of Viral Diseases in the National Institutes of Health (NIH). His laboratory has had a long-standing interest in how enveloped viruses enter target cells and seeks to unravel the basic mechanisms of membrane fusion mediated by the interactions of viral envelope glycoproteins with their target cell receptors and to apply this knowledge to the development of novel strategies to treat and prevent virus infection. Dr. Berger‟s laboratory has developed specialized expression and reporter gene technologies to study membrane fusion mediated by viral glycoproteins and to screen cDNA libraries for essential cellular receptors. These approaches enabled his laboratory to discover the first HIV coreceptor, fusin (subsequently renamed CXCR4), followed by the second major coreceptor, CCR5. These findings led Dr. Berger to devise a novel genetically engineered protein called sCD4-17b that potently neutralizes HIV. Dr. Berger is currently exploring use of sCD4-17b as a topical microbicide to block sexual transmission of HIV, including genetically modifying Lactobacillus species in the healthy vaginal tract to produce HIV-blocking proteins. He is also developing Env-targeted immunotoxins as an approach to eliminate the reservoirs of HIV-infected cells that persist despite potent antiretroviral therapy. Dr. Berger has numerous awards including the following: Breakthrough of the Year, Science 1996; AAAS-Newcomb Cleveland Prize; Highly Cited Researchers, ISI; 100 Great Experiments by Great Scientists; Kenneth Fong/Clontech Award; Novartis-Drew Award for Biomedical Science; Damon Runyon-Walter Winchell Foundation Most Prominent Alumni; AMA/NIAID Nathan Davis Award; NIH Outstanding Contributions to Education of Postbaccalaureate Trainees; Norman P Salzman Memorial Mentor Award in Virology, Honorable Mention. Dr. Berger has published extensively throughout his career in numerous high impact journals including Nature, Science, Immunity, Journal of

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Virology, Proceedings of the National Academy of Sciences, Journal of Biological Chemistry, and Cell. His professional activities include the following: Institute of Human Virology, Executive Committee, Scientific Advisory Board; NIAID Technology Evaluation Advisory Committee; NIH Office of AIDS Research, Coordinating Committee on Etiology & Pathogenesis; HHMI/NIH Program Advisory Committee; NIAID Promotion and Tenure Committee; CONRAD CICCR Global Microbicide Project, Scientific Advisory Group; NIH Virology Interest Group, Founding Steering Committee Chairperson; XII International Congress of Virology, Paris, International Scientific Committee, 2002; Keystone Symposium on HIV Pathogenesis, Lead Organizer, 2001. Michael Boulton, Ph.D. Dr. Boulton is Professor in the Department of Anatomy and Cell Biology at the University of Florida. He received his B.S. degree in Microbiology with Chemistry at the University of Reading, and his Ph.D. from Polytechnic of Central London. He has recently moved to the University of Florida from the University of Texas Medical Branch where he was Director of the AMD Center. Dr. Boulton has a distinguished international record for his investigations into the pathophysiology of the retina. Dr. Boulton‟s research group has two main research areas: age-related changes in the retina and retinal neovascularization. Dr Boulton has a large number of research projects in progress which include nanotechnology to eliminate lipofuscin from the aged eye; investing the contribution of mitochondrial dysfunction and autophagy dysregulation to age-related macular degeneration; hematopoietic stem cell repair of the damaged retina; targeting -secretase in the regulation of angiogenesis; investigating the role of bone marrow-derived progenitor cells in vascular repair; and developing new strategies for the regulation of angiogenesis. Dr. Boulton has over 200 publications and has a long history of research support including two current National Institute of Health (NIH) supported R01 grants. He has given over 40 plenary lectures at international conferences. He currently sits on several of review committees and is a scientific advisor to a number of other agencies. Dr Boulton is on the editorial board of five international journals and plays a major role in the organization and development of a number of professional societies. He has also made a significant contribution to teaching graduate and undergraduate students and has played a major part in the organization and development of graduate education. Robert Paul Johnson, M.D. Dr. Johnson is Clinical Associate in Medicine at Massachusetts General Hospital in the Infectious Disease Unit, Associate Professor in the Division of Immunology at Harvard University, and Adjunct Associate Professor at the University of Massachusetts Medical School. He received his B.S. degree Psychology summa cum laude from Duke University and his M.D. from Harvard Medical School. Dr. Johnson completed his internship at Yale New Haven Hospital and was Assistant and then Chief Resident during his residency. Dr. Johnson was Clinical and Research Fellow in Infectious Disease under Dr. Bruce Walker at the Infectious Disease unit at Massachusetts General Hospital.

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Dr. Johnson has a longstanding interest in immune regulation, lymphocyte biology, and infection with SIV and HIV, respectively, as a means for developing more effective and less toxic therapies for the treatment of AIDS. He is focused on the development of novel gene therapy vectors that block HIV replication into HSCs for use in cell-based therapies for treating AIDS; employing animal models for studying vaccination strategies against SIV/HIV; in vivo testing of genetically-modified HSCs; and to develop methods to mobilize HSCs from bone marrow; and developing new strategies for creating HIVresistant stable T cell populations that can restore immune function in AIDS patients. Dr. Johnson has been a member of the Scientific Advisory Board at Cytomatrix, Member of the Immunology and Virology Programs at Harvard Medical School, Chairman of the Division of Immunology at the New England Primate Research Center, and Director of the Developmental Core at the Harvard University Center for AIDS Research. Dr. Johnson is an active member of the retroviral professional community. Select activities include: Member of the Scientific Program Committee for the 8 th, 9th, 11th, and 13th Conference on Retroviruses and Opportunistic Infections, and Conference Organizer for the Keystone Conference on AIDS Vaccines. Dr. Johnson is an active reviewer and referee. Currently, he serving on the AIDS Study Section, is the Co-Chair on the HIV Vaccine Trials Network Nonhuman Primate Early State Investigator Program, and is the Protocol Co-Chair for the HIV vaccine Trials Network Protocol 084 for the National Institute of Allergy and Infectious Disease (NIAID) at the National Institutes of Health (NIH). He is currently on the editorial board at the Journal of Medical Primatology, AIDS Research and Human Retroviruses, Journal of Virology, Current Molecular Medicine, Virulence, and Nature Communications. Henry J. Kaplan, M.D., F.A.C.S. Dr. Kaplan is the Evans Professor of Ophthalmology; Professor in the Department of Microbiology and Immunology; and Chairman of the Department of Ophthalmology & Visual Sciences at the University of Louisville School of Medicine and Director of the Kentucky Lions Eye Center, Louisville. He received his A.B. from Columbia University and his M.D. from Cornell Medical School and is Fellow, American College of Surgeons (F.A.C.S.). Dr. Kaplan completed an Internship in Medicine at Lakeside Hospital, The University Hospitals of Cleveland, Case-Western Reserve University in Cleveland; a Surgical Residency at Bellevue Hospital, New York University Medical Center; a National Institutes of Health (NIH) Research Fellowship in Immunology in the Department of Cell Biology at the University of Texas Southwestern Medical School in Dallas under the mentorship of J. Wayne Streilein and Ruppert Billingham; an Ophthalmology Residency at the University of Iowa Hospitals and Clinics in Iowa City; and a Retina-Vitreous Fellowship at the Medical College of Wisconsin in Milwaukee under the tutelage of Thomas M. Aaberg. Dr. Kaplan has served as an Assistant Professor in the Department of Cell Biology at the University of Texas Southwestern Medical School; an Associate Professor then Professor and Director of Research in the Department of Ophthalmology at Emory University School of Medicine; and Professor

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and Chairman of the Department of Ophthalmology and Visual Sciences at the Washington University School of Medicine in St. Louis. Dr. Kaplan has a novel background as a clinician scientist with scientific expertise in immunology and clinical expertise in vitreo-retinal diseases. His postdoctoral training launched his interest in autoimmune diseases of the eye and led to the discovery of anterior chamber-associated immune deviation (ACAID). His clinical interest in uveitis has led to his research in ocular immunology and autoimmune diseases, which has been funded by the National Eye Institute (NEI) for the past 30 years. His interest in retinal diseases has involved the study of the pathogenesis and treatment of both agerelated macular degeneration and hereditary retinal diseases. Many novel observations and insights were made in collaboration with different colleagues, including the first successful submacular surgery to recover central vision in the presumed ocular histoplasmosis syndrome (POHS); the development of techniques to harvest and transplant sheets of RPE cells into the subretinal space of man and other species; the first clinical trial of human allogenic RPE cell transplantation in patients with exudative age-related macular degeneration (AMD) in the United States; the role of senescent Bruch‟s membrane in RPE attachment and differentiation; and the study of RPE cell differentiation and dedifferentiation in vitro. Dr. Kaplan is also involved in research focused on characterizing the Pro23His rhodopsin mutation model of retinitis pigmentosa in the miniature swine. This model will then be used to study the ability of swine induced pluripotent stem cells (iPSCs) to regenerate photoreceptors destroyed in this disease. Dr. Kaplan‟s research has been supported by the NIH, and he has participated in peer review at the NIH including serving as a member and then Chairman of the Visual Disorders Study Section at the NEI. He has previously served as a co-editor and then Editor-in-Chief of Ocular Immunology and Inflammation and as member of the American Uveitis Society of which he was President from 1997 – 1999. Doug Kerr, M.D. Ph.D. Dr. Kerr is the Medical Director of Neurology and Global Research and Development at Biogen Idec. He received his Ph.D. from Thomas Jefferson University and his M.D. from Jefferson Medical. He is a Board Certified neurologist and neuroscientist who spent the previous 14 years at The Johns Hopkins University before joining Biogen in January of 2010. He was Associate Professor of Neurology in The Johns Hopkins School of Medicine and in the Department of Molecular Microbiology and Immunology in the Johns Hopkins Bloomberg School of Public Health and at the Kennedy Krieger Institute. Dr. Kerr founded and directed the Johns Hopkins Project RESTORE, a multidisciplinary effort dedicated to advancement of treatments for autoimmune neurologic disorders. In 2006, Dr. Kerr was awarded the Derek Denny-Brown Young Neurological Scholar Award from the American Neurological Association as one who “has achieved a significant stature in neurological research and whose promise of continuing major contributions to the field of neurology is anticipated.” He has testified before the US Congress and the UK House of Lords on stem cell prospects in neurologic disorders.

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He has over 120 peer-reviewed publications in the medical literature, mainly dealing with spinal cord motor neuron disorders and autoimmune neurologic disorders. Dr. Kerr currently directs clinical development for programs for the autoimmune spinal cord and optic nerve disorder neuromyelitis optica, and motor neuron disorders.

Pamela Gehron Robey, Ph.D. Dr. Gehron Robey is Chief of the Craniofacial and Skeletal Disease Branch (CSDB) and Chief of the Skeletal Biology Section and Acting Chief of Skeletal Clinical Studies within the CSDB at the National Institute of Dental and Craniofacial Research (NIDCR) in the National Institutes of Health (NIH). She received her B.A. degree from Susquehanna University and her M.S. degree and her Ph.D. from Catholic University of America. Dr. Robey‟s research interest is in cellular biochemistry of bone formation. Her current efforts are directed towards taking recent advances in the area of bone cell biology and factorology and developing clinical applications for increased understanding and therapeutic treatment of brittle bone diseases. Dr. Robey is currently the chair of the Hard Tissue Disorders Special Interest Group on the NIH campus. In addition, she is very active member of the American Society of Bone and Mineral and on the Science Advisory Board of the National Osteoporosis Foundation. Dr. Robey has published numerous articles in journals including PLoS One, Stem Cell Research, Cell Stem Cell, Journal of Biological Chemistry, and Nature Medicine among others and is an inventor on several patents. Mauricio Rojas, M.D. Dr. Rojas is Professor in the Center for Translational Research in the Lung-Division of Pulmonary, Allergy and Critical Care Medicine at Emory University. He received his M.D. in 1987 from Universidad Nacional de Colombia Medical School in Santafe de Bogota, Colombia. After Research Trainee and Research Associate positions in Colombia were he became the sub-director of National Institute of Immunology, Dr. Rojas had his postdoctoral training as a Fellow in the Department of Microbiology and Immunology at Vanderbilt University School of Medicine in Nashville, Tennessee. After his Fellowship, Dr. Rojas became a Research Instructor in the Department of Medicine, Division of Rheumatology and Clinical Immunology at Vanderbilt University, were his main focus was the study of the intracellular signal of B cell receptor. In 2002, Dr. Rojas joined Emory University where he has been involved in the development of novel therapies for lung disease, which includes the use of gene therapy, protein therapy and cell therapy. Dr. Rojas‟s current research is on lung injury; a clinical syndrome characterized by acute onset of respiratory dysfunction and precipitated by a variety of lung insults. His research focuses on development of new technologies for treatment of lung diseases. His laboratory has developed a technique, called membrane translocating sequence (MTS) technology, which can deliver large functional protein domains or complete proteins into a broad range of cell types. Using this technology, the Rojas lab has

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developed an NF-B inhibitor that, when used in vivo, has been demonstrated to reduce the systemic effect of endotoxins. The second research focus in the Rojas lab is in regenerative medicine where he hypothesizes that acute or chronically injured lungs can be repair by inducing mobilization, homing and differentiation of adult bone marrow (BM)-derived stem cells into the lung. To study this novel concept, the Rojas lab uses murine and porcine models and multiple approaches, including molecular biology, proteomics, genomics, lung physiology and pathology. Recently, Dr. Rojas initiated a new research area in regenerative medicine and aging, focusing these research efforts on alterations of the immune system and cell mediated regeneration associated with aging that can have implications in new therapies for chronic lung diseases like pulmonary fibrosis. During his postdoctoral training at Vanderbilt, Dr Rojas„ research was focused in the study of intracellular signals involved in the action of several growth factors. Part of his work was focus in develop a new group in vivo inhibitors of protein-protein interactions. This work results in the development of a novel technology to deliver protein into the cell. Dr Rojas holds the patent of this technology: US patent number 6,248,558 (6/19/01) and US patent number 6,432,680 (8/13/02). Results of Dr. Rojas‟ research efforts have been published in numerous scientific journals including Parasite Immunology, Vaccine, The Journal of Immunology; Lancet; Biochemical and Biophysics Research Communication; Nature Biotechnology; Journal of Biological Chemistry; American Journal of Physiology Lung Cellular and Molecular Physiology; American Journal of Respiratory Cell; Molecular Biology; and American Journal Respiratory and Critical Care Medicine. In addition, Dr Rojas serves as reviewer for several journals including: TiPS; Critical Care Medicine; American Journal of Respiratory and Critical Care Medicine; American Journal of Physiology Lung Cellular and Molecular Physiology; and Inflammation Research. Anne Rosser, M.D. Ph.D. Dr. Rosser is Chair of Clinical Neuroscience in Cardiff and co-direct the Cardiff Brain Repair Group (BRG) and is an honorary consultant neurologist at the University of Wales Hospital and the clinical lead for the South Wales Huntington‟s disease service. She earned her First class honors M.D. with a third year specialism in neuroanatomy in the accelerated preclinical course at Cambridge University, UK. Before continuing her medical training, Dr. Rosser received a Ph.D. in systems neuroscience studying neuroendocrine olfactory reflex under the supervision of Professor Barry Keverne. Following her Ph.D., Dr. Rosser was first trained in medicine in Cambridge and London and then in neurology at the National Hospital for Neurology, Queen Square, London, and in Cambridge. Dr. Rosser then secured a Medical Research Council Clinician Scientist Fellowship to work at the Brain Repair Centre in Cambridge University where she established a stem cell group focusing on fetal neural progenitor cells and took the lead for the Cambridge Huntington‟s disease (HD) clinic where she was the co-PI on an MRC-supported study of Neural Transplantation in HD. In 2001, she moved to Cardiff, initially on a Lister Institute Clinician Scientist Fellowship.

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Dr. Rosser cares for HD patients at all stages of disease, ranging from asymptomatic to end-stage and undertakes a regular movement disorder clinic. The BRG is a group of approximately 30 post-docs, research associates, clinician researchers and technicians. Under Dr. Rosser‟s direction, the group is seeking to develop new strategies for therapy in neurdegeneration (in particular, HD, Parkinson‟s disease (PD), and stroke) based on a multidisciplinary approach in several converging areas. The laboratory encompasses research ranging from basic cell and molecular biology and cell culture to whole animal behavior in models of neurological disease. The BRG has a strong translational reputation and direct access to patient cohorts through Dr. Rosser‟s HD clinics and through collaboration with other specialist clinics in Cardiff, such as the regional young PD and multiple sclerosis clinics. A particular interest of the BRG is CNS reconstruction by cell replacement therapy. The group has a long history of experimental and, more recently, clinical involvement using primary fetal CNS cells. The next stage for CNS circuit reconstruction will be the use of renewable cell sources. Dr. Rosser‟s group studies human fetal CNS progenitors, human ES cells, and more recently human IPS cells with a view to eventual human application. Dr. Rosser has been the clinical lead for the South Wales Huntington‟s disease for the last ten years and have developed the clinic as a multidisciplinary joint research and clinical management service. The clinic supports numerous clinical studies, and she is PI on studies ranging from large observational studies such as Registry (European HD network - EHDN) and PREDICT (University of Iowa) to interventional drug studies. Dr. Rosser is currently engaged in a number of Cardiff-based studies including an interventional study of physical exercise in HD and a neural transplantation study. In addition to cell replacement in HD, Dr. Rosser is part of the new „Transneuro‟ study of neural transplantation in PD, for which our team will be responsible for cell delivery. Her associated HD clinical activities include being the chair of the EHDN scientific advisory committee, the EHDN lead for the English-speaking network, the Chair of the UK HD network, and Associate director for the Dementia and Neurodegenerative Research Network (DeNDRoN). Dr. Rosser is also actively involved teaching and education in the area of neurodegeneration and the training of physicians in the administration of clinical assessment of HD for research purposes. In 2004, Dr. Rosser was elected as a Fellow of the Royal College of Physicians. Joshua B. Rubin, M.D., Ph.D. Dr. Rubin is Co-Director of Pediatric Neuro-Oncology, Co-Leader of the Cancer and Developmental Biology program, and Associate Professor in the Department of Pediatrics at Washington University in St. Louis School of Medicine. He received his B.S. degree from Yale University and his M.Sc., his Ph.D. in Neuroscience, and his M.D. from Albert Einstein College of Medicine. Dr. Rubin completed his Residency at Children‟s Hospital at Harvard University and his Fellowship in Hematology/Oncology at Children‟s Hospital/Dana Farber Cancer Institute. Dr. Rubin‟s laboratory is interested in defining how normal brain development impacts on the genesis of pediatric brain tumors and using this knowledge to develop more efficacious and less toxic approaches to brain tumor treatment. In this work, he uses

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genetically engineered mouse models of pediatric brain tumors, primary human pediatric brain tumor isolates and intracranial xenografts, as well mouse models of normal brain development. His laboratory is focused on how primary oncogenic events, such as mutational activation of the sonic hedgehog or epidermal growth factor pathways, or loss of function of neurofibromin, alter activity in the CXCR4 pathway to promote abnormal growth. This work has identified critical roles for CXCR4 and cAMP in the regulation of brain tumorigenesis and provides the rationale for multiple preclinical and clinical studies to test the activity and toxicity of CXCR4 antagonists and cAMP elevating drugs. This overlaps with Dr. Rubin‟s other research interest, which is in the clinical development of novel therapies for brain tumors. In the multidisciplinary Pediatric Brain Tumor Program at St. Louis Children's Hospital, Dr. Rubin is working to develop and deliver innovative therapies to children with brain tumors. Dr. Rubin has his Missouri License and is Board Certified by the American Board of Pediatrics, sub-board Pediatric Hematology/Oncology. He was Alpha Omega Alpha and a former NICHD Scholar of the Child Health Research Center for Excellence in developmental biology at Washington University School of Medicine and Hyundai Scholar. John E. Wagner, M.D. Dr. John Wagner is Professor of Pediatrics; Director of the Division of HematologyOncology and Blood and Marrow Transplantation; and Co-Director of the Center for Translational Medicine at the University of Minnesota. He currently holds two endowed chairs: the Hageboeck/Chilren‟s Cancer Research Fund Chair in Pediatric Cancer Research and the University of Minnesota McKnight Presidential Chair in Hematology and Oncology. After receiving his M.D. degree at Jefferson Medical College in 1981, he completed his internship and residency in Pediatrics at Duke University School of Medicine in 1984 and a postdoctoral fellowship in Hematology-Oncology at the Johns Hopkins School of Medicine in 1987, where he remained until joining the faculty at the University of Minnesota in 1991. Internationally recognized as an expert in the field of stem cells and umbilical cord blood transplantation, he is board certified in Pediatrics and Pediatric Hematology/Oncology. He also serves as Scientific Director of the Clinical Research Blood and Marrow Transplant Program and Director of the Clinical Trials Office at the University of Minnesota Cancer Center. Dr. Wagner's research has focused on the development of new treatment approaches for life-threatening diseases for which conventional treatments are unsatisfactory using molecular and cellular therapies. While historically, most of his work is in the setting of leukemia and bone marrow transplantation, new possibilities now exist allowing us to consider moving outside this area, such as the area of skin diseases, cardiovascular diseases, diabetes, and neurological diseases. Specific projects underway in his laboratory include investigation of hematopoietic recovery and engraftment after umbilical cord blood transplantation; prevention of graft-versus-host disease after blood and marrow transplantation; development of novel conditioning regimens; diseasespecific studies on Fanconi anemia and severe epidermolysis bullosa; and development

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of novel cellular therapies involving cardiac stem cells, regulatory T cells, dendritic cellbased vaccines for brain tumors, NK cells for anticancer therapy, and tissue repair. Dr. Wagner has served on numerous national and international scientific advisory committees including for the National Institutes of Health (NIH) - National Heart, Lung, and Blood Institute (NHLBI), National Marrow Donor Program; Society of Hematotherapy and Graft Engineering; the Food and Drug Administration (FDA) Biological Response Modifiers Advisory Committee; and the American Association of Blood Banks. He has also made significant contributions as a scientist/clinician member of the CIRM‟s Standards Working Group. In addition, he has reviewed proposals for several prestigious granting organizations including the Wellcome Trust, National Institute for Biological Sciences, Israel Science Foundation, the National Research Institute (Taiwan), and the Swiss Science Foundation. Professor Andrew Whitelaw, M.D. Dr. Whitelaw is Professor of Neonatal Medicine at the University of Bristol. He went to school in Fife and Edinburgh, and then studied natural sciences at Cambridge and medicine at St Mary‟s (now Imperial) in London. He worked in training posts in pediatrics in London, including a research fellowship in metabolism and nutrition at the Institute of Child Health. He then moved to Toronto as a neonatal fellow and realized that techniques to investigate the newborn brain were emerging. Returning to London, he worked for the MRC for 2 years, then became consultant senior lecturer at Hammersmith Hospital (Royal Postgraduate Medical School). He was a member of a group pioneering neurological assessment, cranial ultrasound, neonatal MRI, neonatal EEG and randomized trials in neonatal intensive care. In 1990, he moved to Oslo where he was Associate Professor and became Professor of Pediatrics in 1995. In 1998, he became Professor Neonatal Medicine in Bristol and Consultant Neonatologist at Southmead and St Michael‟s Hospitals. Dr. Whitelaw‟s major research interest and clinical expertise has been in the mechanisms, diagnosis and treatment of neonatal brain injury, particularly intraventricular haemorrhage and hydrocephalus in prematures and hypoxic-ischemic injury in term babies. Together with Professor Marianne Thoresen (CSSB), Dr. Whitelaw has established Bristol as an internationally recognized centre for experimental treatments for babies with brain injury and the use of models of neonatal brain injury. He has always felt that clinical trials and scientific investigation are a natural part, indeed a quality indicator, of clinical care. Dr. Whitelaw was awarded the Raymond Horton Smith Prize for the best MD thesis at Cambridge in 1978. In 1985-6 after working in Colombia for UNICEF, he did the first scientific studies and trials on skin-to-skin contact for premature infants (Kangaroo baby care) - a technique now used worldwide. In 2005-7 he was a member of the Nuffield Council on Bioethics Working Party on Critical Care Decisions in the Fetus and Newborn. Andrew was elected President of the Neonatal Society 2006-8. He has held funding from the MRC, Wellcome Trust and a number of charities including Action Medical Research and the Health Foundation.

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