Winning the War on Huntington’s Disease

EVENT RECAP
May 13, 2021
Events

In honoring Huntington’s Disease Awareness Month, WuXi AppTec is proud to host the “Winning the War on Huntington’s Disease” webinar to raise awareness and increase understanding of this rare and devastating, hereditary neurodegenerative disease.

This complimentary webinar is the sixth in WuXi AppTec’s ongoing “Collaborations That Transform” series, where we will convene drug developers, physicians, patients, and advocates to critically examine the state of treatment and care, and how to forge ahead with resilience and collaborative new approaches despite recent setbacks.

At WuXi AppTec, we deeply believe that we must work collaboratively to solve industry-wide challenges and bring transformative medicines to patients worldwide.

AGENDA

8:00 AM

Welcome

Hui Cai, Program Chair, VP and Head of Content, WuXi AppTec

8:05 AM

Huntington’s Disease: Challenges, Lessons, and Opportunities

Michael Hayden, Founder & CEO, Prilenia Therapeutics; Killam Professor at the University of British Columbia

Moderator: Michael Ward, Global Head of Life Sciences & Healthcare Thought Leadership, Clarivate

8:30 AM

The Evolving Landscape of Care in Huntington’s Disease

Karen Anderson, Professor, Psychiatry & Neurology, Georgetown University; Director, HD Care, Education and Research Center

Blair Leavitt, Professor, Department of Medical Genetics and Senior Scientist, Centre for Molecular Medicine and Therapeutics, UBC

Svein Olaf Osen, President, International Huntington Association

Kristen Powers, Executive Producer, Twitch

Moderator: Richard Soll, Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec

9:20 AM

New Modalities and Technologies Driving Progress in Huntington’s Disease

Irina Antonijevic, CMO, Triplet Therapeutics

Ricardo Dolmetsch, President, Research & Development, uniQure

Aimee Jackson, CSO, Atalanta Therapeutics

Moderator: Dave Madge, VP, Research Service Division, WuXi AppTec

10:00 AM

Closing

SPEAKERS

Hui Cai

Program Chair, VP and Head of Content, WuXi AppTec

Dr. Hui Cai joined WuXi AppTec in 2009 as Vice President of Business Development, and is currently Vice President and Head of Content Division.

Prior to WuXi, Dr. Cai spent 10 years at Johnson & Johnson Pharmaceutical Research and Development leading multiple drug discovery programs in the therapeutic areas of inflammation and autoimmune diseases. She is a co-author and co-inventor to over 50 scientific publications and issued or pending patents. Dr. Cai is a Councilor of the American Chemical Society (ACS), a member of BayHelix, and a member of the UCSD Alumni Board. In her past capacity, she served as a Commissioner at the City of San Diego Science and Technology Commission, Chair of SABPA, and President of SDCA. Dr. Cai received her BS and MS in Chemistry from Peking University, PhD from The Scripps Research Institute, and MBA from UCSD Rady School of Management as a DLA Piper – Athena Scholar.

Michael Hayden

Founder & CEO, Prilenia Therapeutics; Killam Professor at the University of British Columbia

Dr. Hayden is an accomplished scientist and physician. He was the President of Global R&D and Chief Scientific Officer at Teva from 2012-2017. During this time approximately 35 new products were approved in major markets with many for diseases of the CNS. In 2015 Teva R&D was recognized as one of the 10 most exciting innovators in Pharma by IDEA Pharma and in 2017 Teva R&D ranked top of the industry for CNS development and clinical trial success rate by Pharma Intelligence. He is also a Killam Professor at the University of British Columbia.

Dr. Hayden was named one of the 50 Canadians born in the 20th century who have changed the world. Dr. Hayden is the co-founder of five biotechnology companies including: Prilenia, NeuroVir Therapeutics Inc., Xenon Pharmaceuticals Inc., Aspreva Pharmaceuticals Corp and 89bio. He currently sits on different public and private boards of biotechnologies companies.

Author of approximately 900 peer-reviewed publications and invited submissions, Dr. Hayden has focused his research primarily on translational medicine, including genetics of diabetes, lipoprotein disorders, Huntington disease, predictive, personalized medicine and drug development. Dr. Hayden and his research group have identified 10 disease-causing genes which includes the identification of the major gene underlying high-density lipoprotein (HDL) in humans. Dr. Hayden also identified the first mutations underlying Lipoprotein Lipase (LPL) Deficiency and developed gene therapy approaches to treat this condition resulting in the first approved gene therapy product (Glybera) in a major market. Dr. Hayden is the most cited author in the world on ABCA1, and Huntington Disease.

Dr. Hayden is the recipient of numerous prestigious honours and awards. He was inducted into the Canadian Medical Hall of Fame in 2017. He was named one of PharmaVoice’s “100 of the Most Inspiring People” (2015); awarded an Honorary Doctorate of Science from University of Alberta (2009) and by the University of Gottingen (2014); the Luminary award by the Personalized Medicine World Conference (2014); the Diamond Jubilee Medal (2012), on behalf of HRH Queen Elisabeth II, in recognition of his significant contributions and achievements; the Margolese National Brain Disorder Prize (2011), awarded to Canadians who have made outstanding contributions to the treatment, amelioration, or cure of brain diseases; the Killam Prize by the Canada Council of the Arts (2011), in recognition of his outstanding career achievements; and the Canada Gairdner Wightman award (2011), recognizing him as a physician-scientist who has demonstrated outstanding leadership in medicine and medical science. Dr. Hayden has also been awarded the Order of Canada (2011), and the Order of British Columbia (2010). He was named Canada’s Health Researcher of the Year by CIHR (NIH of Canada) in 2008, and he received the Prix Galien in 2007, which recognizes the outstanding contribution of a researcher to Canadian pharmaceutical research.

Dr. Hayden is committed to empowering others. In addition to mentoring over 100 graduate students and postdocs, he is also a TED mentor.

Michael Ward

Global Head of Life Sciences & Healthcare Thought Leadership, Clarivate

As both a journalist and analyst, Mike has been writing, analyzing and commenting on the life sciences industry for more than 35 years. He has held editorial and content leadership roles at various publications and organizations across the industry. In 2010, he was named European Mediscience Commentator of the Year and has been on the short list on other occasions in the past decade. In 2018, he was fourth on Labiotech.EU’s list of “Biotech Leaders You Should Know in the UK’s Golden Triangle.”

Karen Anderson

Professor, Psychiatry & Neurology, Georgetown University; Director, HD Care, Education and Research Center

Karen E. Anderson, MD, is a tenured Professor of Psychiatry & Neurology at Georgetown University. She was recruited to Georgetown in 2013 to direct the Huntington's Disease Care, Education and Research Center at Georgetown MedStar Hospital, which is also a Huntington's Disease Society of American Center of Excellence. Prior to that, she was the Director of the University of Maryland Huntington's Disease Clinic, which she founded in 2001, and a clinician at the Baltimore VA Medical Center in the Operation Enduring Freedom/Operation Iraqi Freedom Traumatic Brain Injury Clinic. Dr. Anderson’s clinical and research interests include treatment of behavioral symptoms in patients with Huntington Disease (HD), and other movement disorders such as Parkinson’s disease. She has a lead role in development of expert consensus treatment guidelines for emotional symptoms in people with HD. She conducts clinical trials for new drug therapies in dementia and movement disorders, including HD, and has had a leadership role in several HD clinical trials.

Dr. Anderson earned her undergraduate and medical degrees from the University of Chicago. She completed her internship at Columbia Presbyterian Medical Center and her residency and postdoctoral research training in psychiatry at Columbia University and the New York State Psychiatric Institute where she began her work in HD at the Gertrude Sergievsky Center. She has subspecialty certification in Neuropsychiatry and Behavioral Neurology.

Blair Leavitt

Professor, Department of Medical Genetics and Senior Scientist, Centre for Molecular Medicine and Therapeutics, UBC

Dr. Leavitt is a Huntington’s disease (HD) neurologist. He currently holds positions as a Senior Scientist at the Centre for Molecular Medicine and Therapeutics, and as a Professor in the Department of Medical Genetics at the University of British Columbia. Dr. Leavitt is a long-standing member and former Co-Chair of the Huntington Study Group, an established HD clinical trial investigator, and the Director of Research at the UBC Centre for HD in Vancouver. He has an ongoing clinical research program in neurogenetics with a focus on HD. A laboratory scientist as well as a practicing neurologist, his laboratory is dedicated to developing and testing new treatments for genetic diseases of the brain and spinal cord. Using genetically-modified mouse models of human disease as his primary tool, Dr. Leavitt’s research focuses on developing new therapies for devastating neurodegenerative diseases such as HD, FTD and ALS. His research also includes research programs searching for clinical biomarkers in HD and identifying new genetic causes of ataxia, epilepsy, neurodevelopmental disorders, and autism. He is the co-founder and CEO of Incisive Genetics Inc. a biotech company developing lipid nanoparticle delivery systems for gene editing applications. Dr. Leavitt is a founding Co-Editor-in-Chief of the Journal of Huntington’s Disease.

Svein Olaf Osen

President, International Huntington Association

Svein has a background in entrepreneurship, having started a number of companies in several countries. Svein currently works at Anzyz Technologies, focusing on Artificial Intelligence. He joined the Huntington´s Disease community in 2008, first as a member of the board in Norway due to the disease´s presence in his wife´s family.
Six years ago, Svein joined the European Huntington Association as a board member. Svein has two daughters, Maiken and Janikke with his wife Astri, who is also involved in the Huntington´s community and is the president of the European Huntington´s Association. His engagement started when he met Michael Hayden in Denmark. In this meeting, Svein was inspired to start working to find a drug that was effective for the disease. His vision for the International Huntington Association is that all the different Huntington´s Associations can be one strong global voice, and that we can collaborate across borders and different cultures, in order to build a strong network that helps both patients and families that are affected by HD.

Kristen Powers

Executive Producer, Twitch

Kristen Powers grew up as her mother experienced the devastation of Huntington's disease. After her mother passed away when Kristen was 17 years old, Kristen decided to get tested for the disease herself. Because of the stigma and misconceptions surrounding both the disease and genetic testing, Kristen made Twitch, a documentary that follows her genetic testing journey as an 18-year-old. Kristen, now 27, lives in North Carolina and is an executive director of a local non-profit.

Richard Soll

Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec

Dr. Richard Soll is presently Senior Advisor, Strategic Initiatives for the Research Service Division at WuXi AppTec and Head of the WuXi office in Boston, Massachusetts. He has held various leadership roles at WuXi as Senior Vice President including head of the medicinal chemistry unit known as the International Discovery Service Unit, business development, and corporate alliances.

Dr. Soll’s contributions led to the discovery of the HCV NS5A inhibitor elbasvir (a component of Zepatier®) in the Merck-WuXi partnership, the JAK2 inhibitor fedratinib at TargeGen which formed the basis of the $7B partnership between Celgene and Impact Biomedicines, and more than 10 other clinical stage drugs throughout his career.

Dr. Soll is currently a board member at Simcha Therapeutics, an board observer at the Accelerator Life Science Parnters, and an advisory board member with the Blavatnik Center for Drug Discovery at Tel Aviv University, the Children’s Tumor Foundation, and the Pistoia Alliance.

Dr. Soll was CSO / VP of R&D at TargeGen and was VP of Chemistry at 3-Dimensional Pharmaceuticals. He started his career at Ayerst Research Labs and was trained as a synthetic chemist at Dartmouth and Harvard.

Irina Antonijevic

CMO, Triplet Therapeutics

Irina is a neurologist and psychiatrist, board certified in Germany, with a PhD in neurophysiology. During her residency, in Germany and the US, Irina has treated many patients with various movement disorders, including HD. In 2001, Irina transitioned from academia to industry with the goal to develop new and better therapeutics for neuropsychiatric disorders. Her drug development efforts have focused on translational medicine and early development, mostly in neuroscience, but including ophthalmology and immunology. Most development programs focused on rare genetic diseases. In 2009/2010 Irina was Medical Director at CHDI. Since then, Irina has remained committed to develop new therapeutics for HD, working in global pharma, such as Sanofi Genzyme, as well as small biotech, such as Wave Life Sciences and now Triplet Therapeutics, investigating different modalities, including small molecules, biologics as well as nucleic acid therapeutics for HD and other rare neurogenetic disorders.

Ricardo Dolmetsch

President, Research & Development, uniQure

Dr. Ricardo Dolmetsch joined uniQure as President, Research & Development in September 2020. He is responsible for uniQure’s gene therapy research activities, as well as nonclinical development, process development, analytical development and vector development.
Dr. Dolmetsch joins uniQure from Novartis Institutes for Biomedical Research (NIBR), the research arm of Novartis AG (NYSE: NVS), where he served as the Global Head of Neuroscience since 2013. He has led the development of treatments for neurodegenerative and neuropsychiatric diseases, including rare genetically defined disorders. During his tenure, Dr. Dolmetsch played an instrumental role in Novartis’ acquisition of AveXis in 2018 and the successful approval of Zolgensma, a commercial gene therapy for patients with spinal muscular atrophy Type 1. Prior to NIBR, Dr. Dolmetsch was a Professor at Stanford Medical School and a Senior Director at the Allen Institute for Brain Science in Seattle, Washington.
He obtained his bachelor’s degree with honors from Brown University, earned his Ph.D. in neuroscience from Stanford University and conducted his postdoctoral training at Harvard University Medical School and Children’s Hospital Boston.

Aimee Jackson

CSO, Atalanta Therapeutics

Aimee has worked in the field of RNA interference and oligonucleotide therapeutics for 20 years. Aimee most recently served as the Vice President of Research for miRagen Therapeutics, where she advanced 3 microRNA programs from initial concept to First-in-Human clinical trials in less than 5 years. These programs encompassed diverse therapeutic areas and comprised both microRNA mimics and inhibitors. All three programs successfully completed Phase 1 clinical trials, and two of these programs have advanced to Phase 2 clinical trials, including a fibrosis program for which Aimee served as the Product Development Team Lead. Prior to joining miRagen, she led preclinical research at Regulus Therapeutics and Merck/Rosetta, where she served as Molecular Profiling Lead for the development of siRNA-based therapeutics. Known for identifying siRNA off-target effects, she played a lead role in the identification of siRNA design aspects for enhanced activity and specificity.

Aimee is known for being passionate about cutting-edge and innovative approaches to technology and medicine to improve patients’ lives.

Dave Madge

VP, Research Service Division, WuXi AppTec

Dr Dave Madge is Vice President of the Research Service Division in WuXi AppTec. In this role Dave leads business development in Europe and Israel, alongside a portfolio of strategic initiatives across multiple therapeutic areas and modalities. Prior to joining WuXi AppTec, in 2014, Dave was VP, Research, for the ion channel drug discovery company Xention Ltd, in Cambridge, UK, developing new molecules for cardiovascular and respiratory disorders. Before Xention Dave was based at University College London, as part of an integrated biomedical research group, and was responsible for developing new therapeutic discovery projects into funded biotech companies. Dave has a PhD in Medicinal Chemistry from Imperial College, London, and started his career at The Wellcome Foundation in the medicinal chemistry team.

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