About Us:

Scientific Advisory Board


About Us: Scientific Advisory Board

Marc Tessier-Lavigne, PhD | Chairman of the Board

Dr. Marc Tessier-Lavigne is the President of Stanford University. He was formerly the President of Rockefeller University. A world leader in the study of brain development, Dr. Tessier-Lavigne has pioneered the identification of the molecules that direct the formation of connections among nerve cells to establish neuronal circuits in the mammalian brain and spinal cord. He has also been at the forefront of discoveries on many other brain wiring proteins and receptor mechanisms. The mechanisms he has identified are important for understanding how the human brain forms during normal development, and provide essential tools to assist regeneration of nerve connections following trauma or injury, such as paralyzing injuries to the spinal cord.

Dr. Tessier-Lavigne was born in Trenton, Canada, and received a BSci in Physics from McGill University, and a BA in Philosophy and Physiology from Oxford University, where he was a Rhodes Scholar. He obtained his PhD in Physiology from University College London, and performed postdoctoral work at the MRC Developmental Neurobiology Unit in London and at Columbia University, where he was a Markey Scholar. From 1991-2001 Dr. Tessier-Lavigne was on the faculty at the University of California, San Francisco, and from 2001-2003 he served as the Susan B. Ford Professor in the School of Humanities and Sciences and Professor of Biological Sciences at Stanford University. He was also an investigator with the Howard Hughes Medical Institute from 1994-2003.

In 2003, Dr. Tessier-Lavigne joined Genentech, Inc., where he held the position of Executive Vice President, Research Drug Discovery and directed disease research and drug discovery in cancer, immune disorders, infectious diseases, and neurodegenerative diseases. He was also a member of Genentech’s Extended Executive Committee and its Early Stage Portfolio Committee, which oversees all experimental medicines from the early development stage to the end of Phase II proof-of-concept studies in humans.

Dr. Tessier-Lavigne serves on the editorial boards of several scientific journals, including the Board of Reviewing Editors of Science magazine, and he is co-editor in chief of Current Opinion in Neurobiology. He also serves on the Scientific Advisory Boards of a number of foundations and research organizations. In 2000, Dr. Tessier-Lavigne co-founded a biotechnology company, Renovis, Inc., which focuses on developing therapies for neurological and psychiatric disorders. He is currently Chair of the Renovis Scientific Advisory Board. Previously, he was a scientific advisor to Exelixis, Inc.

Dr. Tessier-Lavigne is the recipient or co-recipient of a number of awards, including the Young Investigator Award of the Society for Neuroscience (USA), the Charles Judson Herrick Award of the American Association of Anatomists, the Ameritec Prize for contributions towards a cure for paralysis, the Fondation Ibsen Prize in Neuronal Plasticity, the Viktor Hamburger Award of the International Society for Developmental Neuroscience, and the Wakeman Award for regeneration research.

In recognition of his scientific contributions, Dr. Tessier-Lavigne has been elected a Fellow of the Royal Society (London), a Fellow of the Royal Society of Canada, and a Fellow of the American Association for the Advancement of Science. In 1999, he was named a Canadian “Leader for the 21st Century” by Time magazine, Canada.

Douglas G. Cole, MD | Co-Chair

Dr. Douglas Cole is a General Partner at Flagship Ventures. He obtained post-graduate training in medicine at the Johns Hopkins Hospital in Baltimore, MD and in neurology at the Massachusetts General Hospital in Boston, MA. In 1992, Dr. Cole was appointed Instructor in Neurology at Harvard Medical School and an Assistant in Neurology at Massachusetts General Hospital. He established a research program investigating the mechanistic basis of neuronal signaling events and plasticity in neuro-psychiatric disorders with the support of the NIH and several non-profit research foundations. Dr. Cole holds an AB magna cum laude in English from Dartmouth College and an MD from the University of Pennsylvania School of Medicine.

In 1996, Dr. Cole joined Cytotherapeutics, in Providence, RI. As Medical Director, he oversaw various research and clinical activities related to the company’s cell-based therapeutic technologies. In 1997, he was appointed Program Executive at Vertex Pharmaceuticals, Inc., in Cambridge, MA, where he led a multidisciplinary program that conducted preclinical development through Phase II studies in tissue protection and repair, oversaw international research collaboration, and was responsible for identifying strategic market and technology opportunities in multiple arenas.

Dr. Cole has led Flagship’s investments in CombinatoRx and Alinea Pharmaceuticals. He co-founded Ensemble Discovery with Noubar Afeyan and Professor David Liu of Harvard University, and he served as CEO of the company in its initial stages. He currently serves on the Board of Directors of CombinatoRx, Ensemble Discovery, Aveo Pharmaceuticals, and CGI Pharmaceuticals, and is an Observer on the Board of Directors of Alinea. He has been actively involved with Renovis Hypnion, and Genstruct.

Ivan Lieberburg, PhD, MD | Co-Chair

Dr. Ivan Lieberburg is currently a member of the Tavistock Group, a private equity firm, where he concentrates on health care and life sciences investment opportunities. Prior to joining Tavistock in May 2009, Dr. Lieberburg was employed at Elan for 22 years, where he held a number of senior management positions, including Executive Vice President and Chief Medical Officer, Chief Scientific Officer, head of the Corporate Office of Technology, and Senior Vice President of Research. He has been involved in the basic research, development, and/or marketing of a number of Elan’s products, including Zanaflex (tizanidine), Diastat (rectal diazepam), Mysoline (primidone), Zonegran (zonisamide), Myobloc (botulinum toxin, serotype-B), Permax (pergolide), Skelaxin (metaxolone), Tysabri (natalizumab), Myocet (liposomal doxorubicin), Abelcet (liposomal amphotericin B), Frova (frovatriptan), Prialt (intrathecal ziconitide), Azactam (aztreonam), and Maxipime (cefepime), as well as numerous preclinical programs including Elan’s Alzheimer’s immunotherapy program, which is currently in Phase III clinical trials. Prior to joining ƒlan (formerly Athena Neurosciences, Inc.) in 1987, Dr. Lieberburg held faculty positions at Albert Einstein School of Medicine and Mt. Sinai School of Medicine.

Dr. Lieberburg received an AB in Biology from Cornell University, a PhD in Neurobiology from The Rockefeller University and an MD from the University of Miami School of Medicine. He performed his postdoctoral research at The Rockefeller University and his medical residency and postdoctoral fellowship at the University of California, San Francisco, where he is presently a Clinical Professor of Medicine and an active attending on the in-patient medical service. Dr. Lieberburg has authored over 100 scientific publications and numerous patents, and he has been named to a number of honors including Rockefeller University Fellow, Public Health Corps Scholar, National Research Service Award, Kaiser Teaching Award, Hartford Foundation Scholar, and McKnight Fellow in Neuroscience. He is board certified in internal medicine and endocrinology/metabolism. Dr. Lieberburg is a director of Adamas Pharmaceuticals, and he sits on the scientific advisory boards of Adamas and Limerick BioPharma.

Steven J. Burden, PhD

Dr. Steven Burden is a Coordinator in the Molecular Neurobiology Program at the Helen and Martin S. Kimmel Center for Biology and Medicine at the Skirball Institute of Biomolecular Medicine and a Professor in the Department of Pharmacology at NYU Medical School. Dr. Burden received his PhD from the University of Wisconsin at Madison in 1977. He trained as a postdoctoral fellow with Dr. U. J. McMahan at Stanford Medical School, with Dr. M. C. Raff at University College London, and was awarded a Paul Cohen Fellowship from the Muscular Dystrophy Association. From 1980-1984, he served as an Assistant Professor in the Department of Cell Biology and Anatomy at Harvard Medical School. In 1984, Dr. Burden moved to the Biology Department at M.I.T., where he was the Thomas and Virginia Cabot Career Development Professor and a recipient of a Jacob Javits merit award from NINDS. In 1995, he moved to the Skirball Institute.

Dr. Burden is a developmental neurobiologist, who has worked on the cellular and molecular mechanisms that control the formation of neuromuscular synapses. His work has also defined pathways, controlled by synaptic factors and muscle fiber electrical activity, which regulate gene expression in skeletal muscle.

Darryl C. De Vivo, MD

Dr. Darryl C. De Vivo is the Sidney Carter Professor of Neurology, Professor of Pediatrics, and Director Emeritus (1979-2000) of the Pediatric Neurology Service at Columbia University Medical Center in New York City. He is also Associate Chairman (Neurology) for Pediatric Neurosciences and Developmental Neurobiology, Founding Director of the Colleen Giblin Research Laboratories for Pediatric Neurology, Director of the Pediatric Neuromuscular Disease Program, and Co-Director of the Columbia University Center for Motor Neuron Biology and Disease.

Dr. De Vivo was a Neurology Director and past President of the American Board of Psychiatry and Neurology (1992-1999), Secretary of the American Academy of Neurology (1993-1997), past President of the Child Neurology Society (1989-1991), and a member of the National Advisory Council Neurological Diseases and Stroke at the National Institutes of Health (1997-2001). He completed postgraduate training in medicine, pediatrics, and neurology at the Massachusetts Memorial Hospital, the Massachusetts General Hospital in Boston, the National Institutes of Health in Bethesda, and Washington University School of Medicine in St Louis.

He has published nearly 400 original articles and reviews, lectures extensively in the U.S. and abroad, serves on several editorial boards and advisory committees, and is an ongoing associate editor for Rudolph’s Textbook of Pediatrics. Publications directly focused on Spinal Muscular Atrophy include: “Familial adult-onset proximal spinal muscular atrophy in a family with type II hyperlipoproteinemia,” “Neurological and neuromuscular disorders” in The Columbia University College of Physicians and Surgeons Complete Guide to Early Child Care, “The Neuromuscular System” in Rudolph AM (Ed.) Textbook of Pediatrics, and “Mitochondrial myopathy simulating spinal muscular atrophy.” Dr. De Vivo was also a co-editor, along with Drs. Jones and Darras, for the textbook Neuromuscular Disorders of Infancy, Childhood, and Adolescence: A Clinician’s Approach, which has become the standard reference in the field.

Dr. De Vivo did his undergraduate work at Amherst College and received his MD from the University of Virginia School of Medicine.

Geoffrey Duyk, MD, PhD

Dr. Geoffrey Duyk is the founder and Managing Partner of Circularis LP. Prior to Circularis, Dr. Duyk was a partner at TPG Growth Biotech. Dr. Duyk also previously served on the Board of Directors and was President of Research & Development at Exelixis where he led a 550+ person group focused on the discovery and development of small molecule therapeutics. Before joining Exelixis, Dr. Duyk was one of the founding scientific staff at Millenium Pharmaceuticals. As Vice President of Genomics at Millennium, he was responsible for building and leading the informatics, automation, DNA sequencing, and genotyping groups as well as the mouse and human genetics group. Prior to his tenure at Millennium, Dr. Duyk was an Assistant Professor at Harvard Medical School in the Department of Genetics and Assistant Investigator of the Howard Hughes Medical Institute. While at Harvard Medical School, Dr. Duyk was a Co-Principal Investigator in the NIH funded Cooperative Human Linkage Center. Dr. Duyk has been and continues to be a member of numerous NIH panels and oversight committees focused on the planning and execution of the human genome project.

Dr. Duyk holds a PhD and MD from Case Western Reserve University and completed his medical and fellowship training at University of California, San Francisco. While at UCSF, Dr. Duyk was a fellow of the Lucille P. Markey Foundation and was awarded a post-doctoral fellowship from the Howard Hughes Medical Institute.

Kenneth H. Fischbeck, MD

Dr. Kenneth Fischbeck serves as Chief of the Neurogenetics Branch of the National Institute of Neurological Disorders and Stroke at the NIH. Before assuming this position, Dr. Fischbeck held various teaching positions at the University of Pennsylvania. Most recently Dr. Fischbeck served as a Professor of Neurology for the University of Pennsylvania.

Dr. Fischbeck holds a specialty certification from the American Board of Psychiatry and Neurology, and he is a member of the American Academy of Neurology, the American Society of Human Genetics, and the American Neurological Association.

Additionally, Dr. Fischbeck serves as chair of the Scientific Review Committee of the Les Turner ALS Foundation and the Steering Committee of the Huntington’s Disease Society of America. He also sits on the Scientific Advisory Committee for the National Neurofibromatosis Foundation, the Scientific Advisory Board for the Families of SMA, the Cure Huntington’s Disease Initiative Planning Committee for the Hereditary Disease Foundation, the Science Committee for the American Academy of Neurology, the Scientific Advisory Committee for the Children’s Research Institute at the Children’s National Medical Center, the Scientific Advisory Board for the Heredity Disease Foundation, and the Scientific Review Board for the Kennedy’s Disease Association.

Dr. Fischbeck is also a member of the editorial boards at Neurobiology of DiseaseAdvances in Neurology, and Muscle & Nerve.

Dr. Fischbeck’s work has earned him a list of honors including recognition in Best Doctors in America, the Cotzias Award from the American Academy of Neurology, and, most recently, election to the Institute of Medicine, National Academy of Sciences. He was also given the Award for Excellence in Teaching by the University of Pennsylvania Medical School Class of 1999 and received the school’s Dean’s Award for Excellence in Basic Science Teaching in 1994.

He has been invited to participate in lectures and symposia nationwide and contributed to nearly 150 publications and 22 book chapters.

Dr. Fischbeck holds an AB magna cum laude in Biochemical Sciences, an AM in Biology from Harvard University, and an MD from Johns Hopkins University.

David J. Glass, MD

Dr. David J. Glass is the Vice President of Research at Regeneron Pharmaceuticals, Inc. Prior to joining Regeneron, Dr. Glass was the Global Head of Muscle Diseases research at Novartis Institutes for Biomedical Research. A native of New York, Dr. Glass received his undergraduate degree from Columbia University and his MD from New York Medical College. After postdoctoral work at Columbia, he worked at Regeneron Pharmaceuticals for 14 years before establishing the Muscle Diseases group at Novartis.

Dr. Glass’s research has led to several key scientific findings in the area of skeletal muscle atrophy and hypertrophy. He is the co-author of more than 60 peer-reviewed articles on cell signaling mechanisms in neuromuscular disease, obesity, and cancer.

Dr. Glass is an elected member of the American Society for Biochemistry and Molecular Biology and the American Society for Clinical Investigation. He is also a Visiting Scientist at Harvard Medical School, where he teaches a course on experimental design. His book, Experimental Design for the Practicing Biologist, was well-reviewed and, in addition, he co-authored a paper that appeared in the “Leading Edge” section of the journal Cell on the philosophy of science, advocating the use of questions as opposed to hypotheses in framing experimental projects.

Edward M. Kaye, MD

Dr. Edward M. Kaye is CEO at Stoke Therapeutics. Previously, Dr. Kaye served as Senior Vice President, Chief Medical Officer, and Interim CEO of Sarepta Therapeutics (previously named AVI BioPharma) and also as Group Vice President for Clinical Development and Therapeutic Head for Lysosomal Storage Disorders and Neurodegenerative Diseases at Genzyme. Dr. Kaye held additional leadership roles in Clinical Development and Medical Affairs for over 10 years at Genzyme and developed specific experience with pediatric neuromuscular conditions. He played a leadership role in gaining Myozyme’s approval for Pompe Disease and oversaw collaborations in this field, including the development of ataluren for Duchenne Muscular Dystrophy (DMD).

Prior to joining Genzyme, Dr. Kaye was Chief of Biochemical Genetics at Children’s Hospital of Philadelphia and Associate Professor of Neurology and Pediatrics at University of Pennsylvania School of Medicine. Before this, he was Chief of Pediatric Neurology and Director of the Barnett Mitochondrial Laboratory at St. Christopher’s Hospital for Children in Philadelphia.

Dr. Kaye received his medical education and pediatric training at Loyola University Stritch School of Medicine and University Hospital. He completed his child neurology training at Boston City Hospital, Boston University, and completed his training as a neurochemical research fellow at Bedford VA Hospital, Boston University.

Dr. Kaye sits on the scientific advisory boards of CureDuchenne and CureCMD (Congenital Muscular Dystrophy).

Katherine Klinger, PhD

Dr. Katherine Klinger has been with Genzyme Corp (formerly Integrated Genetics) since 1985 and currently serves as Sr. Vice President of Genetics and Genomics. In this role, Dr. Klinger directs genomic support of corporate research and development efforts. Dr. Klinger received her BA in biology from Trinity University, San Antonio, TX and her PhD in biochemistry at the University of Texas Health Science Center. Her postdoctoral fellowship was conducted at Case Western Reserve, Cleveland, Ohio. She served as an Adjunct Assistant Professor in the Department of Molecular Biology and Microbiology at Case Western Reserve University School of Medicine from 1985-2003 and is currently an Assistant Clinical Professor in the Department of Pediatrics at the University of Connecticut School of Medicine. Dr. Klinger is ABMG certified in medical genetics and clinical genetics and is a diplomat of the American College of Medical Genetics (ACMG).

Robert E. Pacifici, PhD

Dr. Robert E. Pacifici joined CHDI, Inc. in November 2004 as the Chief Scientific Officer. CHDI, Inc. is a privately-held, not-for-profit organization that is pursuing a biotech approach to rapidly discover and develop drugs that prevent or slow Huntington Disease. Through collaborations with industrial and academic partners, CHDI, Inc. participates in all aspects of the pharmaceutical process, from high throughput screening to preclinical development.

Dr. Pacifici was formerly the Site Director and Chief Scientific Officer for Eli Lilly and Company’s Research Triangle Park Laboratories where his responsibilities included oversight of the company’s global screening and quantitative biology efforts. Prior to joining Lilly, Dr. Pacifici was Vice President of Discovery Technologies at Xencor, a privately held biotechnology company which specializes in developing rationally designed protein therapeutics. Dr. Pacifici spent over a decade at Amgen where he held positions of increasing responsibility, including leadership for their automation, high throughput screening, and information technologies groups. In addition to his internal responsibilities, he was instrumental in forging Amgen’s relationships with Caliper Technologies, The Automation Partnership, and the acquisition of Kinetix Pharmaceuticals

Dr. Pacifici received a BS in Biochemistry from the University of Massachusetts, Amherst and his PhD in Biochemistry from the University of Southern California. He holds an adjunct appointment at the University of Southern California’s Department of Molecular Pharmacology and is the Chair for the National Institute on Neurological Disorders and Stroke (NINDS) Spinal Muscular Atrophy Project’s Scientific Steering Committee. He currently sits on several additional external boards and advisory committees, including the Cooperative International Neuromuscular Research Group and the TREAT ALS Steering Committee.

Alfred W. Sandrock, MD, PhD

Dr. Alfred W. Sandrock, Jr. is the CEO of Voyager Therapeutics. Prior to joining Voyager, Dr. Sandrock was the Head of Research and Development at Biogen Idec, Inc. A board-certified neurologist, Dr. Sandrock leads the team that oversees clinical development in neurologic indications. He is also an Assistant Clinical Professor of Neurology at Harvard Medical School and a Clinical Associate in Neurology at Massachusetts General Hospital.

Dr. Sandrock received his MD from Harvard Medical School and his PhD in neurobiology from Harvard University. He completed an internship in medicine, a residency and chief residency in neurology, and a clinical fellowship in neuromuscular disease and clinical neurophysiology (electromyography) at Massachusetts General Hospital.

His contributions to literature include articles on the identification of molecules that promote axonal regeneration, the role of neuregulin at the neuromuscular junction, as well as multiple articles on therapeutics in multiple sclerosis.

Dr. Sandrock is currently a member of the American Academy of Neurology, the Society for Neuroscience, and the American Association of Electrodiagnostic Medicine.

Gregory L. Verdine, PhD

Dr. Gregory Verdine is the Erving Professor of Chemistry at Harvard University. Dr. Verdine also serves as the Director of the Chemical Biology Initiative and Program in Cancer Chemical Biology at the Dana-Farber Cancer Institute. His research interests lie in the emerging area of chemical biology. Dr. Verdine and his co-workers study biologic processes underlying control of gene expression and preservation of genomic integrity.

Dr. Verdine received his BS degree in 1982 from St. Joseph’s University and his PhD in 1986 from Columbia University (under Koji Nakanishi and Maria Tomasz). Following an NIH postdoctoral fellowship with Chris Walsh at M.I.T. and Harvard Medical School, he joined the Harvard University Department of Chemistry in 1988. Named Thomas D. Cabot Associate Professor in 1992, Verdine was promoted to Full Professor in 1994. In recognition of his accomplishments in teaching and research, Dr. Verdine was named Harvard College Professor in 2000. In 2002, he was named the Erving Professor of Chemistry, the longest-established endowed professorship at Harvard. He is a Full Member of the Harvard Department of Molecular and Cellular Biology, and co-directs two graduate programs, the University-wide Graduate Program in Chemical Biology (cofounded with Jon Clardy) and the Graduate Program in Molecular, Cellular and Chemical Biology. Together with Stanley J. Korsmeyer, Dr. Verdine co-founded the Harvard/Dana-Farber Program in Cancer Chemical Biology and was named its first Executive Director; he also serves as the Executive Director of the Dana-Farber Chemical Biology Initiative. He has published more than 150 original research articles.

Dr. Verdine has received numerous awards and honors, including the National Science Foundation Presidential Young Investigator Award, the Sloan Fellowship, the Searle Scholar Award, the Eli Lilly Award in Biological Chemistry, the Arthur C. Cope Scholar Award, the Nucleic Acids Award of the Royal Society of Chemistry, the Cruikshank Award of the Gordon Research Conferences, and the Nobel Laureate Signature Award of the American Chemical Society (with Anirban Banerjee). He has extensive experience advising the pharmaceutical and biotech industries, especially with regard to the development of next-generation therapeutics. Dr. Verdine currently serves as Founder and Chair of the Scientific Advisory Boards of Enanta Pharmaceuticals, Aileron Therapeutics, and Gloucester Pharmaceuticals. He has served as a regular member of the NIH Bioorganic and Natural Products Study Section, the NCI Board of Scientific Counselors, and is an advisor to the Japanese Ministry of Science and Education, and to the Max-Planck Institute for Molecular Physiology.