<a href="#hui-cai">Hui Cai</a>, Program Chair, VP and Head of Content, WuXi AppTec

Welcome

<a href="#michael-hayden">Michael Hayden</a>, Founder &amp; CEO, Prilenia Therapeutics; Killam Professor at the University of British Columbia
Moderator: <a href="#michael-ward">Michael Ward</a>, Global Head of Life Sciences &amp; Healthcare Thought Leadership, Clarivate

Huntington’s Disease: Challenges, Lessons, and Opportunities 

<a href="#karen-anderson">Karen Anderson</a>, Professor, Psychiatry &amp; Neurology, Georgetown University; Director, HD Care, Education and Research Center
<a href="#blair-leavitt">Blair Leavitt</a>, Professor, Department of Medical Genetics and Senior Scientist, Centre for Molecular Medicine and Therapeutics, UBC
<a href="#svein-olaf-osen">Svein Olaf Osen</a>, President, International Huntington Association
<a href="#kristen-powers">Kristen Powers</a>, Executive Producer, Twitch
Moderator: <a href="#richard-soll">Richard Soll</a>, Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec

The Evolving Landscape of Care in Huntington’s Disease 

<a href="#irina-antonijevic">Irina Antonijevic</a>, CMO, Triplet Therapeutics
<a href="#ricardo dolmetsch">Ricardo Dolmetsch</a>, President, Research &amp; Development, uniQure
<a href="#aimee-jackson">Aimee Jackson</a>, CSO, Atalanta Therapeutics
Moderator: <a href="#dave-madge">Dave Madge</a>, VP, Research Service Division, WuXi AppTec

New Modalities and Technologies Driving Progress in Huntington’s Disease  

Closing

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.

Final_Huntington’s Disease Banner

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.

Hui Cai

hui cai

Program Chair, VP and Head of Content, WuXi AppTec 

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 Hayden

Dr. Hayden-3

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

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.”

Michael Ward

Mike headshot

Global Head of Life Sciences & Healthcare Thought Leadership, Clarivate

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.

Karen Anderson

ANDERSON_KAREN_Headshot-1 (1) (1)

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

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.

Blair Leavitt

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

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.

Svein Olaf Osen

President, International Huntington Association

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.

Kristen Powers

KP_2018_2

Executive Producer, Twitch

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.

Richard Soll

Rich Roll-

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

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.

Irina Antonijevic

TTX website_Irina_headshot

CMO, Triplet Therapeutics 

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.

Ricardo Dolmetsch

President, Research & Development, uniQure 

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. 

Aimee Jackson

Aimee Headshot 2020

CSO, Atalanta Therapeutics

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.

Dave Madge

DMadge

VP, Research Service Division, WuXi AppTec

Events

Featured

Winning the War on Huntington’s Disease

<a href="#john-crowley">John Crowley</a>, Chairman &amp; CEO, Amicus Therapeutics
Moderator: <a href="#simone-fishburn">Simone Fishburn</a>, VP &amp; Editor in Chief, BioCentury

Racing against Time in Rare Diseases 

<a href="#tiffany-house">Tiffany House</a>, Chairman &amp; Board Member, International Pompe Association
<a href="#priya-kishnani">Priya Kishnani</a>, C.L. and Su Chen Professor of Pediatrics, Division Chief, Medical Genetics, Duke University Medical Center
<a href="#brad-crittenden">Brad Crittenden</a>, Executive Director, Canadian Association of Pompe
<a href="#jordi-diaz-manera">Jordi Díaz-Manera</a>, Professor of Neuromuscular Disorders, Translational Medicine and Genetics Honorary Consultant in Clinical Genetics, Newcastle University
Moderator:  <a href="#richard-soll">Richard Soll</a>, Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec

Closing the Gap in Pompe Disease:  Where Are We Heading 

<a href="#barry-byrne">Barry Byrne</a>, Professor and Associate Chair, Director, Powell Gene Therapy Center, Department of Pediatrics, University of Florida
<a href="#edward-conner">Edward Conner</a>, Site Lead and Division Head of Gene Therapy Medical &amp; Development, Astellas Gene Therapies
<a href="#geoff-mackay">Geoff MacKay</a>, President &amp; CEO, AVROBIO
Moderator: <a href="#david-chang">David Chang</a>, CEO, WuXi Advanced Therapies, WuXi AppTec

Realizing the Promise of Gene Therapy in Pompe 

Closing 

Dear Colleagues and Friends,
In commemorating International Pompe Day on April 15, we are proud to host a special webinar, “Progress in Halting Pompe Disease,” to raise awareness of not only the disease but of ongoing efforts for better care and cure. This is the fifth complimentary webinar in the rare disease series, where we convene physicians, drug developers, patients, and advocates to critically examine the current state of rare diseases that impact more than 300 million patients worldwide.
You are welcome to watch the on-demand video on <a href="https://www.youtube.com/channel/UCHbCFUWVaMBCOpyBXf-Sd6g">WuXi AppTec YouTube page</a>.
Below are our past events in the “Collaborations that Transform” series.
2020:
<a href="https://wxpress.wuxiapptec.com/events/webinar-nf1-nf2/" target="_blank" rel="noopener noreferrer">Neurofibromatosis</a>
<a href="https://wxpress.wuxiapptec.com/events/webinar-hemophilia-2/" target="_blank" rel="noopener noreferrer">Advances in Rare Diseases: Hemophilia</a>
<a href="https://wxpress.wuxiapptec.com/events/webinar-sma-2/" target="_blank" rel="noopener noreferrer">Spinal Muscular Atrophy (SMA)</a>
<a href="https://wxpress.wuxiapptec.com/events/webinar-ms-2/" target="_blank" rel="noopener noreferrer">Emerging Opportunities in Multiple Sclerosis and Neuroscience</a>
2021
<a href="https://wxpress.wuxiapptec.com/events/premiering-sunday-wuxi-rare-disease-awareness-concert/" target="_blank" rel="noopener noreferrer">WuXi Rare Disease Awareness Concert on International Rare Disease Day 2021</a>

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John F. Crowley is our Chairman and CEO. John’s involvement with biotechnology stems from the 1998 diagnosis of two of his children with Pompe disease—a severe and often fatal neuromuscular disorder. In his drive to find a cure for them, he left his position at Bristol-Myers Squibb and became an entrepreneur as the Co-founder, President and CEO of Novazyme Pharmaceuticals, a biotech start-up conducting research on a new experimental treatment for Pompe disease (which he credits as ultimately saving his children’s lives). In 2001, Novazyme was acquired by Genzyme Corporation and John continued to play a lead role in the development of a drug for Pompe disease as Senior Vice President, Genzyme Therapeutics.

John and his family have been profiled on the front page of The Wall Street Journal and are the subjects of a book by Pulitzer prize-winning journalist Geeta Anand, “The Cure: How a Father Raised $100 Million-And Bucked the Medical Establishment-In a Quest to Save His Children.” The major motion picture, Extraordinary Measures, starring Brendan Fraser and Harrison Ford, is inspired by the Crowley family journey. John is the author of a personal memoir: Chasing Miracles: The Crowley Family Journey of Strength, Hope, and Joy.

John is also a commissioned officer in the U.S. Navy Reserve, assigned to the United States Special Operations Command and is a veteran of the global war on terrorism, with service in Afghanistan. He graduated with a B.S. in Foreign Service from Georgetown University, and earned a J.D. from the University of Notre Dame Law School and an M.B.A. from Harvard. The Crowley family was the recipient of the 2011 Family Exemplar Award from the University of Notre Dame. He is also a member of the University Council on Science & Technology at Notre Dame. Currently, John serves as a member of the Intellia Therapeutics, Inc. Board of Directors. He is the former National Chairman of the Make-A-Wish Foundation of America and is a founding Board member of the Global Genes Project. John is a Henry Crown Fellow at the Aspen Institute.


John Crowley

john-crowley

Chairman & CEO, Amicus Therapeutics

Simone serves as Vice President and Editor in Chief, bringing experience from both industry and academia in translational science, where she focused for over 15 years on the scientific, commercial and strategic considerations for advancing laboratory discoveries to products for patients.

Since joining BioCentury in 2013, Simone has written and edited on innovation from idea to patient, most recently serving as Executive Editor. Previously, she was a director of translational research at Nektar Therapeutics and a senior managing consultant at Exponent Inc., where she specialized in projects for the biopharma industry. Simone serves as an adviser on multiple university translational programs, including initiatives at the University of California and Stanford University. Simone is also active in programs to advance women's careers; she is a board member of Women In Bio and served as its President in 2013-14.

Simone is a Fulbright scholar and performed a post-doc at the University of California San Francisco. She holds a Ph.D. in Molecular Pharmacology from the Weizmann Institute of Science, and an M.A. and B.A. in Pharmacology from Cambridge University.

Simone Fishburn

Simone_Fishburn_8x10

VP & Editor in Chief, BioCentury

Tiffany House is presently President, Acid Maltase Deficiency Association (AMDA), Inc., San Antonio, Texas; a non-profit organization for patients with Pompe Disease.  Spearheads annual agenda, communicates with industry, current on latest advancements in treatment and patient care, and attends various scientific/patient conferences.

Patient Representative/Voting Member of Four Advisory Committees (AC), Food and Drug Administration (FDA), Washington, D.C.; serves as the “voice of the patient.”  Active team member reviewing Lumizyme (Pompe treatment).  Committed to email correspondence, teleconferences, and in-person meetings in D.C.  Lumizyme AC Voting Member, evaluated application with recommendations, October 2008.  Patient Representative Role guest speaker, FDA Patient Representative workshops, 2009 and 2010.

Patient Representative, Niemann-Pick Type C and NAGS Deficiency AC, January 2010; Patient Representative, Direct-to-Consumer Genetic Testing AC, March 2011.

Chairman/Board Member, International Pompe Association (IPA), Baarn, the Netherlands; an international Pompe patient support organization. The mission of the IPA is to “Campaign for early diagnosis and effective, affordable and safe therapies.  Strive to provide information and support to all patients, their families and others with interests in Pompe Disease.”  Board Member, participates in regular conference calls with Board and industry, annual meetings, and face-to-face meetings with industry and scientific/medical experts.

Tiffany received her Juris Doctorate (Suma Cum Laude) in 2011 at St. Mary’s University School of Law, San Antonio, Texas and an M.A. in English, 2007 and B.A. in English, 2005 (Thesis: Pompe’s Disease: My Experiences and the Search for a Cure) at the University of Texas at San Antonio, Texas.

Tiffany House

Tiffany House Headshot 2013

Chairman & Board Member, International Pompe Association

Priya S. Kishnani, MD, is chief of the Division of Medical Genetics, Department of Pediatrics, at Duke University Medical Center in Durham, North Carolina, and director of its YT and Alice Chen Center for Genomic Research. She holds certification from the American Board of Medical Genetics and the American Board of Biochemical Genetics. She began her medical education in Bombay, India, followed by a residency and 2 fellowship programs at Duke University. Dr Kishnani’s primary focus has been the translation of laboratory science into the clinical arena. 

She has a long-standing research and clinical interest in Pompe disease, and was the lead investigator for the pivotal trials of Pompe disease and responsible for the clinical translation from the bench, which led to FDA approval of Myozyme™ (2006)/Lumizyme™ (2010) as the first treatments for Pompe disease. Dr Kishnani continues to investigate the natural history of Pompe disease, uncovering several long-term complications, among them speech and swallowing dysfunction, cardiac arrhythmias, vascular involvement, sleep and gait disturbances, CNS involvement and small fiber neuropathy. She and the Duke team played an integral role in the nomination and approval for the addition of Pompe disease to the RUSP (Recommended Uniform Screening Panel) for newborn screening in the United States. 

Priya Kishnani

C.L. and Su Chen Professor of Pediatrics, Division Chief, Medical Genetics, Duke University Medical Center

Brad Crittenden is Executive Director and past president of Canadian Association of Pompe (CAP). He previously worked for 15 years doing research for major forest products companies, where he took process and product improvements from concept to implementation. He has also been a freelance web designer, working in project areas like venture capital, education, local sports and patient advocacy.
In 2006, after four years of testing, Brad was diagnosed with Pompe disease. It was a surprise but, in retrospect, not surprising as he can remember symptoms even back as a child. He sits on the Volunteer Engagement Advisory Committee for Muscular Dystrophy Canada and volunteers through the local chapter. Brad supports local charities through 100 Men Who Care, and is constantly inspired by what they do. He has served on advisory boards and in his current role looks for opportunities for CAP to partner with other organizations on common goals. Even small things can have a big impact.
Brad grew up in a small community in British Columbia enjoying the outdoors and especially the water. He now lives in the sunny Okanagan Valley of British Columbia in a small city nestled between two beautiful lakes. For many years he competed and now still coaches mixed, women’s, and 55+ dragon boat teams.

Brad Crittenden

bradcrittenden_3c

Executive Director, Canadian Association of Pompe

Professor Jordi Díaz-Manera joined Newcastle University in 2020 where he is working as Professor of Neuromuscular Disorders and as Honorary Consultant Clinical Geneticist with the Newcastle Hospitals NHS Foundation Trust.
Jordi trained as an adult neurologist at the Hospital de la Santa Creu I Sant Pau (HSCSP) in Barcelona from 2001 to 2005. In 2006 he was appointed as Clinical Associate by the Neurology Department of HSCSP. He spent two years as a fellow in the laboratory of Giulio Cossu at the Stem Cell Research Institute of the Ospedalle San Rafaelle in Milano (Italy). His PhD was focused on the development of new diagnostic tools and therapies for Dysferlin Muscular dystrophy.
Jordi has a long-standing experience in translational clinical and basic research. His current clinical research projects are focused on the characterization of large cohort of patients with muscular dystrophies, the implementation of quantitative muscle MRI for the diagnosis and follow-up of patients with muscular diseases and the development of new outcome measures for clinical trials and natural history studies.
His basic research interest focuses on the process of muscle fiber degeneration and the replacement of muscle tissue by fat and fibrosis that takes place in patients with muscular dystrophies. He is especially interested in translating outcomes from cell biology experiments to the development of new therapies, and test them in preclinical animal models of muscular dystrophies.
Jordi was the Coordinator of the Spanish Neuromuscular Research Group from 2012 to 2014 and member of the Executive Committee of the Catalan Society of Neurology from 2017 to 2019.


Jordi Díaz-Manera

JordiDiaz-photo

Professor of Neuromuscular Disorders, Translational Medicine and Genetics Honorary Consultant in Clinical Genetics, Newcastle University

Dr. Barry Byrne is a clinician scientist who is studying a variety of rare diseases with the specific goal of developing therapies for inherited muscle disease. As a pediatric cardiologist, his focus is on conditions that lead to skeletal muscle weakness and abnormalities in heart and respiratory function.
His group has made significant contributions to the understanding and treatment of Pompe disease, a type of neuromuscular disease due to glycogen storage in motor units. The research team has been developing new therapies using AAV-mediated gene therapy to restore cardiac and skeletal muscle function in DMD, Friedreich’s ataxia, Pompe, and other inherited neuromuscular diseases. His group at the Powell Center has also established a series of new methods for large-scale AAV clinical manufacturing.
Dr. Byrne is the Associate Chair of Pediatrics and Director of the University of Florida Powell Center and Child Health Research Institute. He obtained his B.S. degree from Denison University; M.D. and Ph.D. from the University of Illinois; and he completed his Pediatrics residency and cardiology fellowship as well as post-doctoral training in Biological Chemistry at the Johns Hopkins Hospital. Following his early career at Hopkins, he joined the University of Florida and is now the Earl and Christy Powell University Chair in Genetics.
In addition to his academic appointments, he serves as the Chief Medical Advisor of the Muscular Dystrophy Association and a member of the TACT committee of TREAT-NMD. He is an advisor to the Pfizer Rare Disease Therapeutic Area Advisory Committee and co-founder of AavantiBio.

Barry Byrne

BJB-1

Professor and Associate Chair, Director, Powell Gene Therapy Center, Department of Pediatrics, University of Florida

Edward Conner, M.D. is Astellas Gene Therapies Site Lead and Division Head of Gene Therapy Medical & Development and is responsible for leading the global clinical development strategy for Astellas Gene Therapies. He oversees clinical development, clinical operations, regulatory affairs, medical affairs, and patient advocacy. Dr. Conner brings more than 10 years of industry leadership experience in early and late-stage clinical development across a broad range of disease areas, including rare diseases.
Dr. Conner joined Astellas Gene Therapies (formerly Audentes Therapeutics) from Sangamo Therapeutics, Inc. where he served as Senior Vice President and Chief Medical Officer and led the clinical development of the company’s pipeline of genomic therapies. Prior to Sangamo, Ed served as Vice President, Global Clinical Development at Ultragenyx Pharmaceutical Inc. where he led the global clinical development efforts for two of the company’s rare disease product candidates. Previously, Ed served as Senior Medical Director at BioMarin Pharmaceutical Inc. where he led clinical development and regulatory interactions for its global Phase 3 program in Pompe disease, and earlier in his career Dr. Conner served as Medical Director at Genentech, Inc. where he was the clinical science team leader of two product candidates, including XOLAIR®.
Dr. Conner completed his Internal Medicine residency training at the University of Michigan and was a fellow in Clinical Immunology and Allergic Diseases at Johns Hopkins School of Medicine. He earned a B.S. in Biology, cum laude, from Duke University and his M.D. from the University of California, San Francisco.


Edward Conner

Edward_Conner

Site Lead and Division Head of Gene Therapy Medical & Development, Astellas Gene Therapies

Geoff MacKay is President and CEO of AVROBIO, Inc, a clinical stage gene therapy company focused in rare disease.  
Geoff was founding CEO of eGenesis Inc., which applies CRISPR-Cas 9 gene editing to the field of xenotransplantation.
Mr. MacKay spent 11 years as President and Chief Executive Officer at Organogenesis Inc., building the company into the leading cell-based therapy company in the world as measured by FDA approvals, patients treated and revenue.
Mr. MacKay held numerous leadership positions within Novartis AG, such as Vice-President and Business Unit Head, Transplantation and Immunology at Novartis Canada, Vice-President, Tissue-Engineering in Novartis USA, Head of Global Sales Immunology and Transplantation based in Basel, Switzerland, and Sales and Marketing Manager of Novartis Biotech Europe.
Mr. MacKay has served as Chairman of the Board of the Alliance for Regenerative Medicine (ARM), a Washington DC-based multi-stakeholder advocacy group whose mission is to advance the field of regenerative medicine.  Mr. MacKay also served as Chairman of the Board of MassBio as well as multiple biotech company directorships.
Additionally, he was an Advisory Council member for the Health Policy Commission (HPC) for the Commonwealth of Massachusetts, a state agency focused on health policy reform to reduce overall cost growth while improving patient outcomes.
Through his public and private enterprise work, Mr. MacKay is dedicated to applying innovation to reshape and improve the quality of healthcare.

Geoff MacKay

G. MacKay photo 2019 2

President & CEO, AVROBIO

Dr. David Chang has 30-year industrial experience, and is currently the CEO of Wuxi AppTec Advanced Therapies Business Unit located in Philadelphia, PA. Prior to current role, he was Corporate VP and Head of Cell Therapy Global Manufacturing, of Celgene Corporation; the Global Head of Engineering and Strategy at Roche based in Basel, Switzerland; the VP/Site Head of Roche Shanghai Technical Operations in China. Earlier in his career Dr. David Chang worked at Genentech as the Senior Director of Global Manufacturing Science and Technology, and as the Director of Process Development in its Oceanside, CA site. He was also formerly at Biogen Idec as Director of cell culture R&D, at BASF Bioresearch as a cell culture group leader, and at Schering-Plough Research Institute as a process development engineer.

Dr. David Chang obtained his Bachelor’s degree in Chemical Engineering from National Taiwan University in Taiwan, and the Master’s and PhD degrees in Biochemcial Engineering from MIT in Massachusetts, USA.

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