SCIEnTIFIC ADvISoRy BoARD

MICHAEL S. LEvInE, PHD

Vice Chancellor for Academic Personnel

Distinguished Professor of Psychiatry and Biobehavioral Sciences

David Geffen School of Medicine at UCLA

Dr. Michael Levine’s laboratory focuses on the neurophysiological mechanisms underlying neurodegenerative disorders. His multi-disciplinary work carries implications for disorders such as Huntington’s and Parkinson’s diseases and pediatric epilepsy. He has published over 250 peer-reviewed research reports as well as more than 30 book chapters.

Dr. Levine is a fellow of the American Association for the Advancement of Science and received the National Association for Research on Schizophrenia and Depression’s Distinguished Investigator Award in 1999. He has served as the Department of Psychiatry and Biobehavioral Sciences’ Associate Chair for Academic Affairs, the Special Assistant to two Vice Chancellors for Academic Personnel, Chair of the Undergraduate Interdepartmental Program in Neuroscience, Chair of the Interdepartmental PhD Program in Neuroscience, and Associate Director of the Intellectual and Developmental Disabilities Research Center. He presently also serves as the Vice Chancellor for Academic Personnel at UCLA.

JEFFREy D. Long, PHD Professor

Departments of Psychiatry and Biostatistics

University of Iowa

Dr. Jeffrey Long is a data scientist who has been working in Huntington’s disease for over 12 years with emphasis on characterizing progression. Dr. Long helped develop the Huntington’s Disease Integrated Staging System (HD-ISS), which is a new biologically based and data-supported staging system for HD. He also developed the normed prognostic index for HD (PIN), and helped develop the CAG-age product, both of which can be used for characterizing progression and enrichment of treatment populations for clinical trials. Dr. Long has analyzed imaging, biofluid, and clinical data for several large observational studies in HD, including PREDICT-HD, TRACK-HD, and Enroll-HD. He actively works with sponsors and non-profits to assist in the planning of clinical trials for disease-modifying therapies.

JoHn MAzzIoTTA, MD, PHD

Vice Chancellor, UCLA Health Sciences

CEO, UCLA Health

Royal College of Physicians, London

National Academy of Medicine

Dr. John Mazziotta is recognized as one of the world’s foremost experts on brain imaging. He also was the principal investigator of the International Consortium for Brain Mapping, leading a world-wide effort to create the first atlas of the human brain, including behavioral, demographic, imaging and genetic data.

Dr. Mazziotta has published more than 260 research papers and eight texts. He has received numerous awards and honors, including the Oldendorf Award from the American Society of Neuroimaging, the S. Weir Mitchell Award and the Wartenberg Prize of the American Academy of Neurology, and the Von Hevesy Prize from the International Society of Nuclear Medicine. He has been Vice Chancellor of UCLA Health Sciences and CEO of UCLA Health since 2015.

RICHARD I. MoRIMoTo, PHD

Bill and Gayle Cook Professor of Biology

Department of Molecular Biosciences

Director, Rice Institute for Biomedical Research

Northwestern University

American Academy of Arts and Sciences

Dr. Richard Morimoto studies cell stress responses that detect damaged proteins that occur in aging and neurodegenerative diseases including Huntington’s, Alzheimer’s, Parkinson’s and ALS. His laboratory focuses on how proteins misfold and the role of genetic pathways that detect and prevent misfolding, and the use of small molecules to delay or prevent protein aggregation and disease.

Dr. Morimoto has received numerous awards including the National Institutes of Health MERIT Award twice and the Huntington’s Award for Excellence in Medicine from the Huntington’s Disease Society of America. He is a co-founder— together with Andrew Dillin and fellow HDF SAB member Jeffery Kelly—of Proteostasis Therapeutics, Inc., a company which aims to discover small molecule therapeutics for diseases of protein conformation.

A. JEnnIFER MoRTon, PHD, SCD, FRSB

Professor of Neurobiology

University of Cambridge

Professorial Fellow

Director of Studies in Medicine and Veterinary Medicine

Newnham College, Cambridge

A. Jennifer Morton has been working on Huntington’s disease ever since she set up her laboratory at the University of Cambridge in 1991. She is interested in understanding the relationship between neurodegeneration and the neurological symptoms in HD. She is particularly interested in sleep, circadian rhythms and cognitive decline in HD. With funding from the Hereditary Disease Foundation, The Wellcome Trust, and CHDI Foundation, she has characterized the behavioral profile of a number of mouse models of HD.

For the past 10 years, Dr. Morton has also been working with HD sheep. She believes that, although it is an unconventional model, there is much to be gained from understanding the behavioral pathology in this large-brained diurnal model of HD. Her lab’s goal is to identify quantifiable measures of behavior that can be used to test the efficacy of novel therapies for HD.

RICHARD C. MuLLIgAn, PHD

Director, Harvard Gene Therapy Initiative

Laboratory of Molecular Medicine

Mallinckrodt Professor of Genetics

Children’s Hospital

Harvard Medical School

Dr. Richard Mulligan is an internationally recognized pioneer in the development of new technologies for transferring genes into mammalian cells. Scientists use the specialized tools created in his laboratory to unravel basic questions about human development and to devise new therapies for the treatment of both inherited and acquired diseases.

Dr. Mulligan is the Mallinckrodt Professor of Genetics at Harvard Medical School and Director of the Harvard Gene Therapy Initiative, an integrated effort of basic scientists and clinical investigators at Harvard University and its affiliated hospitals directed towards the pre-clinical and clinical evaluation of novel gene-based therapies for inherited and acquired diseases. His honors include the MacArthur Foundation Prize, the Rhodes Memorial Award of the American Association for Cancer Research, the ASMB-Amgen Award, and the Nagai Foundation International Prize.

HARRy T. oRR,

PHD

James Schindler and Bob Allison Ataxia Chair in Translational Research Professor, Department of Laboratory Medicine and Pathology Director, Institute for Translational Neuroscience

University of Minnesota

National Academy of Medicine

Dr. Harry Orr’s laboratory at the University of Minnesota has a long-standing and productive National Institute of Neurological Disorders and Stroke-supported research program on the use of genetics, biochemical, and behavioral approaches in the study of neurodegeneration with a focus on the human disease spinocerebellar ataxia type 1 (SCA1). In collaboration with Dr. Huda Zoghbi at Baylor University, they cloned the gene affected in SCA1- the first genetically defined ataxia. They then established the first transgenic mouse model of a polyglutamine disease. This model is the center of continued studies on the normal function of the gene product, ataxin-1, as well as the SCA1 pathogenic process. The work is among the first studies to indicate that regions outside of the polyQ tract are critical for disease. Identification of molecular pathways involved in neurodegenerative disease can be significant in development of efficacious targets for intervention. A current focus is on two signaling pathways, each having distinct but seminal roles in SCA1. Dr. Orr received the 2022 Kavli Prize in Neuroscience for his pioneering genetic research on SCA1.

Neurons of mouse brain

Megan S. Keiser, Davidson Lab (CHOP)

HEnRy L. PAuLSon, MD, PHD

Lucile Groff Professor in Neurology Director, Michigan Alzheimer’s Disease Research Center

University of Michigan Health System

National Academy of Medicine

Dr. Henry Paulson explores the reasons why the aging brain degenerates in various neurodegenerative diseases. His research focuses on Huntington’s disease, spinocerebellar ataxia type 3 and several other inherited ataxias, as well as Alzheimer’s disease and related protein conformational disorders.

Dr. Paulson pursues both basic studies of disease mechanisms and translational studies in hopes that his research will lead to therapies for these fatal diseases. His leadership roles include directing the Michigan Alzheimer’s Disease Center and codirecting the Michigan Neuroscience Institute.

CHRISToPHER E. PEARSon, PHD

Senior Scientist

Program of Genetics & Genome Biology

The Hospital for Sick Children (SickKids)

Full-Professor

Department of Molecular Genetics, University of Toronto

Canada Research Chair in Disease-Associated Genome Instability

Dr. Christopher Pearson studies the mechanisms of disease-causing repeat expansions. His lifetime goal is to treat repeat diseases by arresting or reversing somatic expansions so as to arrest or reverse disease progression. His lab uses molecular, cellular and mouse models, and patient tissues of Huntington’s disease, myotonic dystrophy, spinocerebellar ataxias, and C9orf72-associated amyotrophic lateral sclerosis (ALS) to focus on DNA repair, DNA damage, epigenetics, and unusual DNA structures formed by the repeats. Recent advances include identifying repeat expansions associated with autism as well as identifying the first small-molecule to induce contractions of the expanded CAG repeat in brains of HD mice.

Dr. Pearson was a Scholar of the Medical Research Council of Canada, Member of the Canadian Genetic Disease Network, and is a Canada Research Chair in Disease-Associated Genome Instability. He serves on scientific advisory boards for numerous venture capital companies and foundations. He is Associate Editor for several scientific journals.