Scientific Advisory Board

SCIENTIFIC ADVISORY BOARD ANNE B. YOUNG, MD, PHD CHAIR, SCIENTIFIC ADVISORY Board Chair Emerita, Department of Neurology Massachusetts General Hospital Distinguished Julieanne Dorn Professor of Neurology Harvard Medical School Royal College of Physicians, London American SCIENTIFICAcademy of Arts and Sciences ADVISORYNational Academy of MedicineBOARD ith funding from the Hereditary Disease Foundation and the National Institutes of Health, Anne WYoung has been involved in Huntington’s disease research for four decades. Anne participated in the Hereditary Disease Foundation’s Venezuela HD Project from 1981 until 2002 when the team could no longer return because of then Venezuelan President Hugo Chavez’s restrictions. In Venezuela, Anne focused on making accurate diagnoses, drawing blood for DNA, taking skin biopsies and helping collect tissue samples generously donated by the Venezuelan HD family members. Of the 20,000 neurological exams performed, Anne did many of them. Anne trained and mentored medical students and residents who joined the team. Anne and her late husband John B. (“Jack”) Penney, Jr. tested new models of how the basal ganglia controls movements. They based their theories on data from animal and postmortem human brain samples. They discovered, through clever experiments, how the basal ganglia are affected in Huntington’s and Parkinson’s diseases. The basal ganglia controls movement, reward, emotions and memory. Anne and Jack’s model suggested the design of therapies that would help both diseases. Anne was recruited in 1991 to Harvard Medical School and Massachusetts General Hospital as the hospital’s first female head of a department. She founded and designed the MassGeneral Institute for Neurodegenerative Diseases (MIND) in 2001 to accelerate the discovery of new and effective therapies for these disorders. Anne is a driving force in the Hereditary Disease Foundation’s Scientific Advisory Board which she chairs. She also plays a key role on the Foundation’s Board of Directors as its Vice Chair. She received the HDF’s Leslie Gehry Brenner Prize for Innovation in Science in 2016. 1 SCIENTIFIC ADVISORY BOARD I met patients with HD when I was doing my PhD and was “ incredibly moved. My passion to find an effective treatment for Huntington’s disease drives everything I do. From “the beginning I have understood the importance of the Hereditary Disease Foundation and its critical role in advancing research towards therapies for HD. Sarah J. Tabrizi, FRCP, PhD, FMedSci University College London Institute of Neurology 1 SCIENTIFIC ADVISORY BOARD SCIENTIFIC ADVISORY BOARD Anne B. Young, MD, PhD — Chair Diane E. Merry, PhD The Hereditary Disease Foundation’s Scientific Advisory Board is composed of distinguished scientists from Massachusetts General Hospital Thomas Jefferson University around the world. The Board sets the scientific priorities for the Foundation, reviews grant and fellowship Harvard Medical School Richard I. Morimoto, PhD applications and selects the most innovative and promising research projects for funding. Leslie M. Thompson, PhD — Vice Chair Northwestern University University of California, Irvine A. Jennifer Morton, PhD, ScD, FRSB ANNE B. YOUNG, MD, PHD Gillian P. Bates, FMedSci, PhD, FRS University of Cambridge University College London (UCL) Institute of Neurology CHAIR Richard C. Mulligan, PhD C. Frank Bennett, PhD Harvard Medical School SCIENTIFIC ADVISORY Board Ionis Pharmaceuticals Harry T. Orr, PhD Chair Emerita, Department of Neurology Yvette Bordelon, MD, PhD University of Minnesota David Geffen School of Medicine at UCLA Massachusetts General Hospital Henry L. Paulson, MD, PhD Distinguished Julieanne Dorn Professor of Neurology Alan Buckler, PhD University of Michigan Triplet Therapeutics, Inc. Harvard Medical School Christopher E. Pearson, PhD Royal College of Physicians, London Marie-Françoise Chesselet, MD, PhD The Hospital for Sick Children David Geffen School of Medicine at UCLA University of Toronto, Canada American Academy of Arts and Sciences National Academy of Medicine Beverly L. Davidson, PhD Bernard M. Ravina, MD Children’s Hospital of Philadelphia Praxis Precision Medicine ith funding from the Hereditary Disease Foundation and the National Institutes of Health, Anne University of Pennsylvania University of Rochester School of Medicine WYoung has been involved in Huntington’s disease research for four decades. Anne participated in the Steven Finkbeiner, MD, PhD Lynn A. Raymond, MD, PhD, FRCPC Hereditary Disease Foundation’s Venezuela HD Project from 1981 until 2002 when the team could no longer Taube/Koret Center for Neurodegenerative Disease University of British Columbia, Canada return because of then Venezuelan President Hugo Chavez’s restrictions. In Venezuela, Anne focused on making Gladstone Institutes accurate diagnoses, drawing blood for DNA, taking skin biopsies and helping collect tissue samples generously H. Diana Rosas, MD University of California, San Francisco Massachusetts General Hospital donated by the Venezuelan HD family members. Of the 20,000 neurological exams performed, Anne did many Kenneth H. Fischbeck, MD Harvard Medical School of them. Anne trained and mentored medical students and residents who joined the team. National Institute of Neurological Disorders and Joan A. Steffan, PhD Stroke, National Institutes of Health University of California, Irvine Anne and her late husband John B. (“Jack”) Penney, Jr. tested new models of how the basal ganglia controls Michelle Gray, PhD movements. They based their theories on data from animal and postmortem human brain samples. They Sarah J. Tabrizi, FRCP, PhD, FMedSci University of Alabama at Birmingham discovered, through clever experiments, how the basal ganglia are affected in Huntington’s and Parkinson’s University College London (UCL) Institute of Neurology diseases. The basal ganglia controls movement, reward, emotions and memory. Anne and Jack’s model Myriam Heiman, PhD Leslie P. Weiner, MD suggested the design of therapies that would help both diseases. Massachusetts Institute of Technology University of Southern California Broad Institute of MIT and Harvard Nancy S. Wexler, PhD Anne was recruited in 1991 to Harvard Medical School and Massachusetts General Hospital as the hospital’s H. Robert Horvitz, PhD Columbia University first female head of a department. She founded and designed the MassGeneral Institute for Neurodegenerative Massachusetts Institute of Technology Hereditary Disease Foundation Diseases (MIND) in 2001 to accelerate the discovery of new and effective therapies for these disorders. Anne 2002 Nobel laureate is a driving force in the Hereditary Disease Foundation’s Scientific Advisory Board which she chairs. She also Ai Yamamoto, PhD David E. Housman, PhD Columbia University plays a key role on the Foundation’s Board of Directors as its Vice Chair. She received the HDF’s Leslie Gehry Massachusetts Institute of Technology Brenner Prize for Innovation in Science in 2016. X. William Yang, MD, PhD Jeffery W. Kelly, PhD David Geffen School of Medicine at UCLA The Skaggs Institute for Chemical Biology The Scripps Research Institute Andrew S. Yoo, PhD Washington University School of Medicine Michael S. Levine, PhD David Geffen School of Medicine at UCLA Scott Zeitlin, PhD University of Virginia School of Medicine John Mazziotta, MD, PhD UCLA Health Sciences 3 SCIENTIFIC ADVISORY BOARD SCIENTIFIC ADVISORY BOARD LESLIE M. THOMPSON, PHD FRANK BENNETT, PHD VICE CHAIR SCIENTIFIC ADVISORY Board Chief Scientific Officer Ionis Pharmaceuticals Donald Bren and Chancellor’s Professor Department of Psychiatry and Human Behavior Department of Neurobiology and Behavior University of California, Irvine eslie Thompson has studied Huntington’s disease for most of her scientific career and was a member of the rank Bennett is responsible for preclinical antisense drug discovery and antisense technology research. He Linternational consortium that identified the causative gene for HD in 1993. Leslie is involved in understanding Fis one of the founding members of Ionis Pharmaceuticals. Frank has been involved in the development of how the HD mutation causes dysfunction of neurons and other cell types in the brain with the goal of identifying antisense oligonucleotides as therapeutic agents, including research on the application of oligonucleotides druggable targets. She is also looking at how modifications of the huntingtin (HTT) protein and other cellular for inflammatory, neurodegenerative diseases and cancer, oligonucleotide delivery, pharmacokinetics molecules are influenced by the mutation. and medicinal chemistry. He has published more than 230 papers in the field of antisense research and Leslie worked with a group of investigators to establish the HD patient-derived iPS cell consortium (induced development and has more than 175 issued U.S. patents. pluripotent stem cells). She is using stem cells to study disease and modifiers of disease onset through multi- Prior to joining Ionis, Frank was Associate Senior Investigator in the Department of Molecular Pharmacology institutional collaborations and Big Data approaches. Several potential therapeutic approaches have been at SmithKline and French Laboratories, currently, GlaxoSmithKline. He received his PhD in Pharmacology identified over the years and she is currently evaluating the use of human neural stem cells as a candidate HD from Baylor College of Medicine, Houston, Texas and his BS degree in Pharmacy from the University of New therapeutic. She is an

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