Scientific Advisory Board

Jeff Carroll, PhD

Scientific Advisory Board Chair

Jeff Carroll is an HD family member, who himself carries the HD mutation. His mom, Cindy, died after suffering from HD, placing her six children at risk of inheriting the disease. After learning about his mom’s condition, Jeff decided to pursue a career in science in hopes of helping better understand, and someday successfully treat, HD. He conducted his PhD in the lab of long-time HD researcher Michael Hayden, at the University of British Columbia. He conducted postdoctoral research with Marcy MacDonald, at Massachusetts General Hospital in Boston, before returning home to Washington state to start his own lab. He now is an Associate Professor in the Department of Neurology at the University of Washington in Seattle. Jeff’s lab uses mouse and cellular models of HD to better understand its mechanisms, with a particular focus on empowering Huntingtin-lowering studies.

Neil Aronin, MD

Professor of Medicine and RNA Therapeutics Institute, Chief, Division of Endocrinology, Diabetes and Metabolism, The Higgins Family Professor in Neuroscience, UMassChan Medical School.

Dr. Aronin’s research focuses on developing gene silencing for therapy in Huntington’s disease, an autosomal dominant neurodegenerative disease. The laboratory uses allele selective RNAi to silence the mutant huntingtin and preserve wild type huntingtin. The research team explores potential therapies and neuronal pathophysiology attributable to the mutant huntingtin. The team also studies viral delivery of allele selective shRNAmir, with single stranded and self-complementary adeno-associated virus. Dr. Aronin’s team studies next-generation therapeutics that will be applicable to a spectrum of autosomal dominant diseases, as well as studying mechanisms of pathogenesis in Huntington’s disease: mutant huntingtin mRNA kinetics, vesicle recycling and roles of proteins that interact with mutant huntingtin.

Yvette Bordelon, MD, PhD

Dr. Yvette Bordelon received her MD and PhD degrees from the University of Pennsylvania School of Medicine in Philadelphia. Her thesis work was performed with Dr. Marie-Francoise Chesselet and involved characterizing mechanisms of cell death in an animal model of Huntington’s disease.  She completed Neurology residency training at the Massachusetts General and Brigham and Women’s Hospitals where she served as Chief Resident in her senior year and then a Movement Disorders Fellowship at Columbia University.  Dr. Bordelon then joined the faculty in the Movement Disorders Program at the David Geffen School of Medicine at UCLA where she is now a Professor in the Department of Neurology.  She has played major roles in medical education including serving as neurology clerkship director for more than 10 years and as an associate director of the neurology residency program in recent years.  Her clinical work involves the diagnosis and treatment of Movement Disorders including Huntington’s Disease, Parkinson’s disease, Essential Tremor and Atypical Parkinsonism among other neurodegenerative diseases. Her clinical research interests include the conduct of clinical trials in these disorders as well as inter-departmental collaborative studies in HD, tauopathies, PD epidemiology, and neuromodulation studies.

Jeffrey Brown, PhD

Dr. Jeffrey Brown is a seasoned biotech leader with deep experience in preclinical and IND-enabling research, with a particular focus on genetically defined disorders. Jeffrey has worked across multiple therapeutic areas, including neuropsychiatry, neurodegeneration, neuromuscular, and metabolic diseases.

He has led more than 20 preclinical programs, successfully advancing a number of these into clinical development. Throughout his career, Jeffrey has built several drug discovery platforms focused on small molecules and nucleic acid-based therapeutics across multiple organizations including Bristol-Myers Squibb, Alexion, Voyager Therapeutics, Wave Life Sciences, and Deep Genomics. Jeffrey received a Ph.D. in Pharmacology and Toxicology from the University of Utah and an eMBA from Suffolk University. Following his Ph.D., Jeffrey completed post-doctoral training at Harvard Medical School and Boston University Medical Center. Combining his drug discovery experience with his passion for helping patients affected with rare disease, Jeffrey founded Scientific Foundation LLC, an organization dedicated to helping families, patients, and biotech start-ups navigate the challenges of preclinical drug development.

Sue Browne, PhD

Dr. Sue Browne is VP of Biology, Head of Preclinical Biology and Head of Biomarkers and Precision Medicine at Passage Bio, Philadelphia PA. Passage Bio is a genetics medicine company developing transformative therapies for people with CNS disorders, including a preclinical program for Huntington’s disease gene therapy.

Dr. Browne received a BSc in Pharmacology from the University of Aberdeen and a PhD in Neuroscience from the University of Glasgow, in Scotland. She first became involved in HD research in 1993 when she moved to the States for her post-doctoral research, joining Dr. Flint Beal’s group at Massachusetts General Hospital. Her research focus, understanding the role of energy metabolism dysfunction in HD and developing translational therapeutic approaches, continued when she joined the Neuroscience Program Faculty at Cornell University’s Weill Medical College. Dr. Browne later moved to industry to pursue drug development for HD and other CNS disorders, first at Merck Research Labs. and Teva Pharmaceuticals, and now at Passage Bio.

Sue’s post-doctoral training began with the award of an HDSA Research Fellowship, and was further supported by other HDSA collaborative grants over the years. She cites her HDSA Fellowship as the most transformative event in her career, allowing her to benefit from a fantastic training in the HD field, enabling her to meet so many within the HD family, and to collaborate with HD scientists around the world. As a result, she was thrilled to become part of the HDSA SAB in 2015 and have the opportunity to help others gain a similar jump-start to their careers in HD research.

Tanya Garcia, PhD

Dr. Tanya P. Garcia is an Associate Professor of Biostatistics at the Gillings School of Public Health, University of North Carolina at Chapel Hill. She has a long-standing interest in interdisciplinary collaborations, beginning as a graduate student and continuing through her current collaborations where she develops practical methods that address challenges in neurodegenerative disease research and advance the statistical theory underlying those methods. She received the Huntington’s Disease Society of America Human Biology Project Fellowship in 2013-2015 which directly led to a K01 from the NIH National Institute of Neurological Disorders and Stroke. Her research focuses on extracting maximal information from large, highly correlated data structures, and innovating new prediction models and variable selection techniques to discover more accurate ways to model motor, cognitive, and functional impairments linked to Huntington disease.

Michelle Gray, PhD

Dr. Michelle Gray is currently an Assistant Professor in the Department of Neurology at the University of Alabama at Birmingham. She is the Dixon Scholar in Neuroscience, focused on the study of Huntington’s Disease. Dr. Gray earned her Ph.D. from the Ohio State University and performed post-doctoral training at the University of California, Los Angeles.

Dr. Gray is a laboratory scientist who has worked on Huntington’s Disease for the last 10 years. She has been an invited speaker and presenter of her work at multiple Huntington’s Disease focused meetings including those sponsored by the Hereditary Disease Foundation and CHDI,Inc. In addition, she has presented updates on Huntington’s Disease to the Alabama Academy of Neurology and participated in Huntington’s Disease Education Day sponsored by the HDSA Center for Excellence at the University of Alabama at Birmingham.

Madaline Harrison, MD

Dr. Madaline Harrison is a Professor Emerita in the Department of Neurology at the University of Virginia. Her clinical focus has been on evaluation and management of a movement disorders including Huntington’s disease, Parkinson’s disease, dystonia, tremor, Tourette’s syndrome, and related conditions.  Her initial research interest was in localization and function of dopamine receptor subtypes.  More recently, she has been involved in clinical research, including therapeutic trials in Parkinson’s and Huntington’s disease.  Dr. harrison has been an active member of the Huntington Study Group and has served on several HSG committees. She is currently a member of the Steering Committee for the virtual UHDRS study.  Dr. Harrison directed the Parkinson’s Disease and Movement Disorders Division in the UVA Department of Neurology from 2008-2020.  In 1996, she founded the UVA Huntington’s Disease Program, an interdisciplinary program that provides interdisciplinary clinical services well as genetic counseling, and served as director until 2022.  She remains active on the UVA Center of Excellence Operations Committee.  

Beth Hoffman, PhD

Dr. Beth Hoffman is the Founder and CEO at Origami Therapeutics, Inc. At Origami, Dr. Hoffman is leveraging lessons learned during 20 years of CNS drug discovery to create a novel, precision medicine approach to treating neurodegenerative diseases by “fixing broken proteins”. Dr. Hoffman served on the Board of Directors at HDSA’s San Diego Chapter and currently serves on the Scientific Advisory Board of the Tau Consortium. Dr. Hoffman received an AB in Molecular Biology from Wellesley College, a PhD in Cell Biology from the Johns Hopkins University and was Chief of the Molecular Pharmacology Unit at the National Institute of Mental Health in Bethesda, Maryland.

Kim Kegel-Gleason, PhD

Dr. Kegel-Gleason studies the normal and altered function of huntingtin (HTT), the protein mutated in Huntington Disease (HD) using cell biology and biochemical approaches including proteomic and lipidomic analysis. Her early work revealed that autophagy and the lysosomal system were activated with HTT accumulation in an HD cell system. More recently, she has investigated the structural features necessary for HTT’s membrane/lipid association and discovered a specific preference to bind to phosphatidylinositol phosphates (PIPs). These PIPs are lipids messengers that orchestrate cortical actin remodeling and signal from the plasma membrane. Dr. Kegel-Gleason uses human stem cells and mouse models to study effects of normal and mutant HTT lowering on protein and lipid changes in brain. 

Ali Khoshnan

Dr. Ali Khoshnan is an Associate Professor of Research in Neuroscience and Physiology at the Zilkha Neurogenetic Institute, Keck School of Medicine, University of Southern California. He received his Ph.D. in Virology from the University of Texas Health Science Center in San Antonio in 1995. Dr. Khoshnan has since made significant contributions to the field of neuroscience, with a particular focus on Huntington’s disease (HD). He began his research on the molecular pathogenesis of HD as a postdoctoral scholar with the late Professor Paul Patterson at the California Institute of Technology in 2000-2002 and continued as a senior investigator from 2002-2022. His work during this period led to the development of novel intrabodies with therapeutic and diagnostic applications, the identification of signaling pathways that underlie neuroinflammation in HD, the production of novel panels of antibodies to toxic conformations of mutant huntingtin protein, and the discovery of a “prion-like” mechanism for the amplification of neurotoxic species in HD. Dr. Khoshnan also established animal models to investigate the impact of gut-brain axis on the progression of HD and to develop gut-based therapeutics. He joined the Zilkha Neurogenetic Institute in November 2022 to continue his research on HD.

Dr. Holly Kordasiewicz

Dr. Holly Kordasiewicz is Vice President of Neurology Research at Ionis Pharmaceuticals, a company that specializes in RNA therapeutics. Dr. Kordasiewicz heads a team focused on identifying antisense oligonucleotide therapeutics for currently untreatable neurodegenerative diseases, including drugs now in clinical trials for ALS, Alzheimer’s disease, Parkinson’s disease and Huntington’s disease. Dr. Kordasiewicz joined Ionis 8 years ago after working on the preclinical validation for Ionis’ Huntington’s disease program as a post-doctoral fellow in the laboratory of Dr. Don Cleveland at the University of California at San Diego. Dr. Kordasiewicz began her work on understanding neurodegenerative diseases at the University of Minnesota, where she received her Ph.D. in Neuroscience.

Seung P. Kwak, Ph.D.

Dr. Kwak is Director of Molecular Systems Biology overseeing the HD human genetics portfolio CHDI Foundation. Taking advantage of a large cohort of HD participants in the Enroll-HD program at CHDI, he aims to discover genetic modifiers of HD as means to develop therapeutic strategies, and apply the knowledge gained from modifiers towards patient stratification in future clinical trials. Dr. Kwak has spent 17 years at CHDI overseeing various preclinical programs. Before coming to CHDI, he worked 10 years at Wyeth Pharmaceuticals as team Leader of early phase programs in chronic pain therapeutic area. Seung received a BA in Psychobiology from Oberlin College and a PhD in Neuroscience from University of Michigan. He joined CHDI in 2006.

Marcy MacDonald, PhD

Professor of Neurology, Harvard Medical School and the Massachusetts General Hospital, Boston, MA

Dr. Marcy MacDonald obtained her Ph.D. in Medical Biophysics from the University of Toronto in 1980 and is currently a Professor of Neurology (Genetics) at Harvard Medical School and the Massachusetts General Hospital, where she is a founding member of the Center for Human Genetic Research.

Dr. MacDonald became involved in Huntington’s disease research in 1985, joining Dr. James Gusella in an international collaboration that identified the Huntington’s disease gene in 1993, improving the diagnosis and management of the disorder. Her research pioneers the use of genetic mouse models and genetic studies in human samples to contribute knowledge essential to the development of effective therapies for Huntington’s disease.

Dr. MacDonald has served the HDSA as co-chair of the Grants and Fellowship Committee (1998-2003). She is the Chair of the Medical and Scientific Advisory Committee (2008-2011) and for the more than two decades has spoken about Huntington’s disease research at HDSA events for local chapters and at the annual convention.

Harry Orr, PhD

Tulloch Professor of Genetics, Department of Laboratory Medicine and Pathology,Director, Institute of Translational Neuroscience, University of Minnesota

Harry Orr received his Ph.D. from Washington University and completed his postdoctoral fellowship at Harvard. Dr. Orr’s research explores genes that play a role in neuron deterioration. This offers important implications for developing gene therapy and other types of treatments for patients with neurodegenerative diseases. Orr discovered the genetic basis for spinocerebellar ataxia type 1, an inherited and ultimately fatal movement disorder, like HD SCA1 is due to a polyglutamine expansion. Orr directs the Institute of Translational Neuroscience, created to translate laboratory research discoveries into clinical trials of new therapies.

Amber Southwell, PhD

Dr. Amber L. Southwell, Assistant Professor, Burnett School of Biomedical Sciences, Division of Neuroscience, has been working in preclinical therapy development for Huntington disease (HD) since 2002. In 2009 she earned her PhD at the California Institute of Technology working with Dr. Paul Patterson to develop an intrabody gene therapy for HD. From 2009-2016, she did postdoctoral research at the University of British Columbia with Dr. Michael Hayden where she developed several novel mouse models of HD, a selective mutant huntingtin gene silencing therapy, and a CSF biomarker for brain huntingtin. She began her laboratory research group at the University of Central Florida in January 2017, where she continues these studies as well as new research on modifiers of disease onset. 

Leslie Thompson, PhD

University of California at Irvine Professor, Psychiatry & Human Behavior

Emily Troncoso

Emily Troncoso is the Chief Compliance Officer for Navy Federal Credit Union. She manages all regulatory compliance matters relating to Navy Federal products, services, and members. Prior to this role, she was a Deputy General Counsel with Navy Federal and a litigation attorney with WilmerHale, a Washington law firm. She received her undergraduate and law degrees from Harvard University, and she served as a judicial clerk for Hon. Richard J. Leon of the U.S. District Court for the District of Columbia.

Emily is originally from outside Philadelphia. She now lives in northern Virginia with her husband and two children. Her late mother and grandmother lived with Huntington’s disease. Previously, Emily has served as a board member for the D.C. Metro chapter of Huntington’s Disease Society of America and the Friends of Georgetown University HD Center.

Hilary Wilkinson, PhD

Hilary Wilkinson is a drug discovery and development expert with experience in leading in vitro and in vivo pharmacology teams at both large pharmaceutical and small biotechnology companies. She contributed to the advancement of programs targeting oncology, immunology, bone, cardiovascular, muscle, and central nervous system disorders and guided projects from target validation until Phase II demonstration of clinical effect. Hilary has expertise in varied therapeutic approaches such as small molecule, therapeutic proteins, AAV and oligonucleotides as well as multiple delivery methods including inhaled, transdermal and oral. She has worked previously at Merck Research Laboratories, CHDI and Eternity Bioscience, Inc. Hilary rejoined CHDI to advance the clinical validation of candidate wet biomarkers.

Hilary Wilkinson has a BS in Life Sciences from MIT and a PhD in Molecular Biology from Princeton University. She studied invertebrate avermectin-sensitive glutamate-gated chloride channels as a World Health Organization Post-Doctoral Fellow. Hilary rejoined CHDI in 2022.