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

Welcome

A conversation with <a href="#dietmar-berger">Dietmar Berger</a>, Head of Development and CMO, Sanofi; moderated by <a href="#kevin-da-silva">Kevin Da Silva</a>, Chief Editor, Nature Neuroscience

Session I: Meeting the Challenges in MS and Neuroscience Drug Development

<a href="#tanuja-chitnis">Tanuja Chitnis</a>, Professor of Neurology, Harvard Medical School; Director, Partners Pediatric Multiple Sclerosis Center, Massachusetts General Hospital 
<a href="#mark-freedman">Mark Freedman</a>, Professor of Neurology, University of Ottawa; Sr. Scientist, The Ottawa Hospital Research Institute 
<a href="#ari-green">Ari Green</a>, Chief, Division of Neuroinflammation and Glial Biology, Medical Director, Multiple Sclerosis and Neuroinflammation Center, UCSF 
<a href="#cyndi-zagieboylo">Cyndi Zagieboylo</a>, President and CEO, National MS Society 
Moderated by <a href="#mandy-jackson">Mandy Jackson</a>, Managing Editor of US Commercial News for Scrip and The Pink Sheet

Session II: Clinical Prospects for a Challenging Disease

<a href="#joachim-herz">Joachim Herz</a>, Professor, Molecular Genetics, Neuroscience, Neurology; Director, Center for Translational Neurodegeneration Research, UT Southwestern Medical Center 
<a href="#ralph-kern">Ralph Kern</a>, President and Chief Medical Officer, Brainstorm Cell Therapeutics 
<a href="#dan-lorrain">Dan Lorrain</a>, Chief Scientific Officer, Pipeline Therapeutics 
<a href="#krish-ramanathan">Krish Ramanathan</a>, Global Program Head, Neuroscience and Global Drug Development, Novartis 
Moderated by <a href="#richard-soll">Richard Soll</a>, Head of WuXi AppTec’s Boston office; Senior Advisor of Strategic Initiatives

Session III: New Directions in MS Therapies

As an industry enabler, WuXi AppTec is committed to improving the lives of patients globally. We share the conviction that we must work collaboratively to solve industry-wide challenges and bring transformative medicines to patients.
We welcome you to the conversation.

Closing

COLLABORATIONS THAT TRANSFORM Emerging Opportunities in Multiple Sclerosis and Neurosciences



10:30am – 12:30pm EDT | 4:30pm – 6:30pm CET | September 24, 2020



Can we build a roadmap to conquer Multiple Sclerosis (MS), a disease that impacts more than 2 million globally as the leading non-traumatic disability disorder in young adults? How have lessons learnt so far shed light on broader neuroscience R&D?



On September 24, join us for “Emerging Opportunities in Multiple Sclerosis and Neurosciences,” the fourth episode in our “Collaborations That Transform” webinar series.



We would like to thank <a href="https://www.biocentury.com/">BioCentury</a>, <a href="https://www.ctf.org/">Children’s Tumor Foundation</a>, <a href="https://califesciences.org/">CLSA,</a> <a href="https://www.informa.com/">Informa</a>, <a href="http://www.newenglandhemophilia.org/">New England Hemophilia Association</a> and <a href="https://www.bioindustry.org/">UK Bioindustry Association</a> for their great support of the “Collaborations That Transform” webinar series.

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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_headshot

VP and Head of Content Division, WuXi AppTec

Kevin received his BSc in pharmacology from the University of Toronto. He then obtained his PhD in neuroscience in the Department of Laboratory Medicine and Pathobiology at the University of Toronto where he did vaccine research on Alzheimer’s disease. As a post-doctoral fellow he continued his work on neurodegenerative disease at Sunnybrook Research Institute, investigating neurogenesis and cholinergic neuron degeneration in aging and Alzheimer’s disease. In 2009, he joined the journal Nature Medicine as an editor, where he handled research manuscripts related to immunology, neuroscience and stem cells. Since 2016, he has been the Chief Editor of Nature Neuroscience.


Kevin Da Silva

Chief Editor, Nature Neuroscience

Dietmar Berger is Chief Medical Officer and Head of Global Development at Sanofi. In this role, Dr. Berger leads global development science, strategy and operations for the company’s portfolio in Immunology, Hematology, Oncology, Rare Diseases, and Neurology, and oversees pharmacovigilance and medical centers of excellence. Formerly, he was Senior Vice President of Global Product Development, Clinical Science Hematology/Oncology at Genentech/Roche, and Head of Research and Development at Atara, focusing on development of T cell immunotherapies. Prior to that, he held positions of increasing responsibility at Bayer and at Amgen.

Dr. Berger completed his medical training in Freiburg (Germany), Basel (Switzerland) and Chicago (United States) and holds an M.D. and Ph.D. from the Albert-Ludwigs University School of Medicine. He has more than 25 years of experience in oncology research and development, including as Head of the Clinical Research Center at the University Medical Hospital, Freiburg, Germany. Dr. Berger has led academic research groups focusing on preclinical drug development, tumor models, angiogenesis, and immunotherapy in Germany, and at The Scripps Research Institute, La Jolla, CA, USA. He received the Cancer Award of the German Cancer Society for his research on angiogenesis and has authored more than 50 scientific publications and five books.

Dietmar Berger

Dietmar P. Berger

Head of Development and CMO, Sanofi

Ralph Kern MD MHSc is President and Chief Medical Officer of BrainStorm Cell Therapeutics. His industry experience includes senior executive roles at Biogen, Novartis and Genzyme. Prior to joining industry, he was head of the post graduate academic neurology program at the University of Toronto. He completed neurology training at McGill University and the University College London, fellowship training in neuromuscular disease, and the MHSc program in Health Administration from the Institute of Health Policy Management and Evaluation at the Dalla Lana School of Public Health at the University of Toronto.


Ralph Kern

President and Chief Medical Officer, Brainstorm Cell Therapeutics

Joachim Herz is a Professor of Molecular Genetics, Neuroscience, Neurology and Neurotherapeutics and the Director of the Center for Translational Neurodegeneration Research at the University of Texas, Southwestern Medical Center. He graduated from Medical School in Heidelberg, Germany in 1983. After residencies in Germany and the U.K. he joined the European Molecular Biology Laboratory (EMBL) as a postdoctoral fellow, where he began his work on LDL receptor-related proteins and their diverse biological functions in development, the vasculature and the peripheral and central nervous system. His current research focuses on the role of LDL receptor-related proteins as ApoE receptors and modulators of synaptic neurotransmission in the pathogenesis of late-onset Alzheimer’s disease and as immunomodulators at the vascular/neurovascular interface. He also investigates the cell biological basis of a form of frontotemporal lobar degeneration that is caused by haploinsufficiency in progranulin and explores therapeutic approaches to correct this genetic deficiency.

Joachim Herz

Paulo Fontoura

Professor, Molecular Genetics, Neuroscience, Neurology; Director, Center for Translational Neurodegeneration Research, UT Southwestern Medical Center

Dr. Ari Green is a neurologist and neuro-ophthalmologist. He is Chief of the Division of Neuroimmunology and Glial Biology where he leads a program of more than 20 faculty members as they search for treatments for MS and other neuroinflammatory and glia related degenerative disorders of the Central Nervous System. dr. Green also medical director of the UCSF MS and Neuroinflammation Center, Co-founder and Co-director of the Small Molecule Program got Remyelination and Director of the Neurodiagnostic Center.
 
Green’s research focuses on developing tests that help bring reparative treatments to patients with MS and halt neurodegeneration in patients with diseases that cause neurological injury. His work aims to advance treatments that would enable recovery after injury to the myelin sheath — the protective layer around nerve fibers in the brain and spinal cord.

Green has led a team dedicated to bringing advanced imaging and electrophysiological measures of the visual system and brain to the clinic. His team pioneered early-stage testing of promising new treatments for optic neuritis, MS and other diseases of the central nervous system. In his laboratory, Green supervises postdoctoral fellows and students on projects related to this work.

Green earned his medical degree at Duke University School of Medicine, where he was a Howard Hughes Medical Institute Predoctoral Fellow for two years. He then completed his residency in neurology at UCSF, where he spent time as chief resident, followed by UCSF fellowships in neuroimmunology and neuro-ophthalmology. In 2005 the American Academy of Neurology and National Multiple Sclerosis Society named Green their first joint clinical research fellow.

Green is a Full Professor at UCSF and holds the title of Debbie and Andy Rachleff Distinguished Professor of Neurology.  He was a prior Harry Weaver Neuroscience Scholar. He is a member of the American Academy of Neurology, Association for Research in Vision and Ophthalmology, North American Neuro-Ophthalmology Society and Association for Clinical and Translational Science. He is also associate editor of JAMA Neurology.

Ari Green

Ari J Green

Chief, Division of Neuroinflammation and Glial Biology, Medical Director, Multiple Sclerosis and Neuroinflammation Center, UCSF

Mark Freedman is Professor of Medicine (Neurology) at the University of Ottawa, Senior Scientist at the Ottawa Hospital Research Institute and Director of the Multiple Sclerosis Research Unit at the Ottawa Hospital-General Campus.

His extensive research includes molecular neurochemistry, cellular immunology, and clinical studies in MS. His basic science interest concerns immune mechanisms of damage in MS, with a particular interest in the role of the innate immune system such as gamma-delta T-cells. His main clinical interests are cell-based therapies for MS. He was the lead investigator of the Canadian Bone Marrow Transplant Study in MS and he co-heads an international study of mesenchymal stem cells for the treatment of MS. He is the current President- Elect of ACTRIMS.

Mark Freedman

Mark S. Freedman

Professor of Neurology, University of Ottawa; Sr. Scientist, The Ottawa Hospital Research Institute

Dr. Tanuja Chitnis a Professor of Neurology at Harvard Medical School and Senior Scientist within the Ann Romney Center for Neurologic Diseases at BWH where she created the Translational Neuroimmunology Research Center focused on bringing bench discoveries to clinical trials for multiple sclerosis and related diseases. She a board-certified neurologist specializing in multiple sclerosis (MS) with a dual appointment at the Brigham and Women’s Hospital (BWH) and Massachusetts General Hospital (MGH) in Boston, Massachusetts, USA. In addition, she serves as the Director of the CLIMB Natural History of MS study at the Partners MS Center, which is located within the Neurosciences Center at BWH. CLIMB follows over 2400 MS patients longitudinally. Here she oversees a team of analysts and postdoctoral fellows working to identify biomarkers precision treatment in MS patients, and immune mechanisms of MS risk and disease course. Her interest in children with MS led her to start the Partners Pediatric MS Center at MassGeneral Hospital for Children where she serves as the Director. Between 2010-2018, she served as the elected Chair of the International Pediatric MS Study Group, where she has led several initiatives in the study of MS in children including the launch of the first clinical trials in this population, leading to the first FDA-approved therapy for this disease. She has authored over 250 publications and reviews related to MS and demyelinating disorders, including the New England Journal of Medicine, Neurology and Journal of Neuroscience. She serves on the advisory board and steering committee of several MS-related organizations and studies including ACTRIMS, U.S. Network of Pediatric MS Centers, and the National MS Society. She receives grant funding from the Department of Defense, NIH, National MS Society, Guthy Jackson Charitable Foundation, and is the co-PI of a large study of SysteMS biology in MS sponsored by Verily. She is the recipient of several awards including the Joseph Martin Award for Clinical Research in 2019 from the Scientific Advisory Council at MGH, and the 2018 Milestones Award from the National MS Society.

Tanuja Chitnis

tanuja chitnis

Professor of Neurology, Harvard Medical School

Cyndi Zagieboylo became president and CEO of the National MS Society in 2011. Her Society career started in 1985 and she has worked with every CEO of the organization including founder, Sylvia Lawry. Ensuring people affected by MS can live their best lives while providing leadership on a global basis to reach a world free of MS is her life’s work.

Cyndi serves on the Society’s National Board of Directors and on the MS International Federation Board of Directors. She is a founding member and executive committee chair of the International Progressive MS Alliance which was launched in 2013. The Alliance expedites the development of therapies for progressive MS and currently includes membership from 18 countries and 6 industry partners.

She is passionate about building the MS movement, amplifying the voices of people affected by MS to influence lawmakers, and ensuring people affected by MS know that they have a supportive partner in their life journeys through a nationwide MS Navigator service.

Cyndi received her bachelor’s degree in rehabilitation counseling and psychology from Springfield College, followed by a master’s degree in social psychology from the University of Connecticut.

Cyndi Zagieboylo

Cynthia Zagieboylo

President and CEO, National MS Society

Mandy Jackson is Managing Editor, US Commercial News, for Scrip and The Pink Sheet. Mandy reports on daily biopharma developments, writes feature stories, produces the Finance Watch column and oversees Emerging Company Profiles. She covers finance, start-ups, dealmaking, clinical trial results, commercial competition and corporate strategy.

Mandy regularly interviews everyone from big pharma CEOs to biotech start-up founders, enhancing her expertise on industry trends and market dynamics. She also is interested in drug pricing and novel reimbursement strategies, new treatments in areas of true unmet need, diversity in the biopharma industry and novel approaches to drug development. She has been a business reporter since 2000, covering biopharma, biotech law and commercial real estate.

Mandy Jackson

Managing Editor of US Commercial News for Scrip and The Pink Sheet

Krishnan (Krish) Ramanathan, is currently Global Program Head in Neuroscience in the Novartis Development Unit. His responsibility includes taking medicines in late stage of research in neuroscience and developing them for regulatory submission and commercial launch. He was responsible for overseeing the registration of Kesimpta®, recently approved by US FDA for adults with relapsing multiple sclerosis.

Krish has an academic background in Biomedical Engineering. He completed his degrees in Master of Science in Engineering and a Ph.D. at the Johns Hopkins University in Baltimore, USA. He specialized in the area of neuroscience and studied some of the fundamental molecular mechanisms underlying the process of “hearing” different frequencies of sound.

Since obtaining his Ph.D. Krish has spent majority of his time working in the Pharmaceuticals and Biotechnology area. He has covered different disease areas in Pharmaceuticals, different functional roles and different geographies. He started his professional career with McKinsey & Company based in Chicago, then moved onto GlaxoSmithKline based in Philadelphia, and had a brief stint in Nuvelo, based in San Carlos, before joining Novartis in Cambridge USA.

Prior to his current role in Novartis, Krish spent 3 years based in Moscow, Russia, where he was the Chief Scientific Officer for the Russian affiliate of Novartis Pharma AG. His previous experiences include therapeutic areas in cardio-metabolic diseases, immunology, dermatology, and infectious diseases. Krish is passionate about translating complex science into stories that everybody can appreciate.

Krish Ramanathan

Global Program Head, Neuroscience and Global Drug Development, Novartis

Daniel is a founding executive team member of Pipeline Therapeutics where he currently serves as Chief Scientific Officer. Pipeline is creating a portfolio of first-in-class small molecule therapeutics with the goal of restoring function through remyelination, synaptogenesis, and axonal repair. Previously, he was VP of Biology at Inception Sciences — a Versant Ventures discovery engine — where he led all aspects of biology and non-clinical pharmacology, including the acquisition of the remyelination company Inception 5 by Roche pharmaceuticals. Prior to joining Inception, Dan was Senior Director of Pharmacology at Amira Pharmaceuticals and contributed to the discovery of several clinical stage small molecule therapeutics to treat inflammation and fibrosis. Notably, he led the efforts of the LPA1R program that was acquired by BMS. Prior to that, he was a Research Fellow at Merck where he contributed to early CNS drug discovery. He was a postdoctoral fellow at the University of Chicago and received a PhD in Behavioral Neuroscience from the University of Buffalo.

Dan Lorrain

Chief Scientific Officer, Pipeline Therapeutics

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

Head of WuXi AppTec’s Boston office; Senior Advisor of Strategic Initiatives

Events

Featured

Emerging Opportunities in Multiple Sclerosis and Neurosciences

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

A conversation with <a href="#paulo-fontoura">Paulo Fontoura</a>, Global Head and SVP, Neuroscience and Rare Diseases Clinical Development, Roche; moderated by <a href="#david-chang">David Chang</a>, CEO, WuXi Advanced Therapies, WuXi AppTec

Session I: Perspective on Global Development and Access to Medicines in Rare Diseases

<a href="#kristin-schneeman">Kristin Schneeman</a>, Director, FasterCures, a center of the Milken Institute 
<a href="#mencia-de-lemus">Mencía de Lemus</a>, President of SMA Europe; President of FundAME, National SMA patient organisation, Spain 
<a href="#laurent-servais">Laurent Servais</a>, Professor of Paediatric Neuromuscular Diseases, Neuromuscular Reference Center Disease, Department of Paediatrics, Liège, Belgium; MDUK Neuromuscular Center, Department of Paediatrics, University of Oxford, UK 
Moderated by <a href="#lauren-martz">Lauren Martz</a>, Senior Editor, Head of Translation &amp; Clinical Development, BioCentury

Session II: Patients at the Center

<a href="#karen-chen">Karen Chen</a>, CEO, Spinal Muscular Atrophy Foundation 
<a href="#stanley-crooke">Stanley Crooke</a>, Chairman of the Board and CEO, Ionis Pharmaceuticals; Founder, CEO and Chairman of the Board, n-Lorem Foundation 
<a href="#thomas-crawford">Thomas Crawford</a>, Professor of Neurology and Pediatrics, Johns Hopkins Hospital, Johns Hopkins Children’s Center, Kennedy Krieger Institute 
<a href="#oscar-henry-mayer">Oscar Henry Mayer</a>, Professor of Clinical Pediatrics, The Perelman School of Medicine at the University of Pennsylvania, Division of Pulmonology, Children’s Hospital of Philadelphia 
Moderated by <a href="#richard-soll">Richard Soll</a>, Head of WuXi AppTec’s Boston office; Senior Advisor of Strategic Initiatives

Session III: Translating Fundamentals into Meaningful Therapeutics

COLLABORATIONS THAT TRANSFORM 
Rare Diseases: Spinal Muscular Atrophy
10:30am – 12:30pm EDT | 4:30pm – 6:30pm CET | August 20, 2020
August is the month each year when special efforts are made to raise awareness of patients suffering from Spinal Muscular Atrophy (SMA), a crippling, degenerative neuromuscular disease. In its most severe form, SMA is the leading genetic cause of infant death. WuXi AppTec welcomes the opportunity to contribute to SMA Awareness Month this year through our next webinar, “Progress in Halting Spinal Muscular Atrophy”, on Aug. 20, 2020.
This is the third webinar in WuXi AppTec’s “Collaborations That Transform” series on rare diseases, where we convene physicians, drug developers, patients, and advocates to critically examine the current state of therapies and the promise of the next generation of treatment approaches and modalities
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<div style="clear: both;">We would like to thank <a href="https://www.biocentury.com/">BioCentury</a>, <a href="https://www.ctf.org/">Children’s Tumor Foundation</a>, <a href="https://califesciences.org/">CLSA,</a> <a href="https://www.informa.com/">Informa</a>, <a href="http://www.newenglandhemophilia.org">New England Hemophilia Association</a> and <a href="https://www.bioindustry.org/">UK Bioindustry Association</a> for their great support of the “Collaborations That Transform” webinar series. </div>
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Karen Chen is the Chief Executive Officer of the Spinal Muscular Atrophy Foundation, responsible for overseeing the full range of scientific and drug discovery programs, as well as managing the operations activities. Dr. Chen is a senior research scientist and manager with over 30 years of experience, including 15 years in the pharmaceutical industry, planning, directing, and conducting preclinical research. She has managed departments and groups working on a variety of projects for the discovery and development of novel therapeutics for neurological disorders. Dr. Chen joined the SMA Foundation from Roche Palo Alto where she was Director of Neurosciences and Head of the Alzheimer’s Disease and Neurodegeneration Group. Prior to Roche she was Director of Pharmacology and Head of the In Vivo Neurodegeneration and Behavior Groups at Elan Pharmaceuticals working primarily on therapies for Alzheimer’s Disease and Parkinson’s Disease. Her post-doctoral training was obtained at Genentech investigating neurotrophic factors therapies for neurodegenerative conditions. Dr. Chen holds an A.B. from Harvard University, and obtained her Ph.D. in Neurosciences from the University of California, San Diego working with Dr. Fred (Rusty) Gage on the behavioral and morphological effects of fetal grafts and neurotrophin administration in models of age-related degeneration. She has over 50 scientific publications and several patents. Dr. Chen currently serves on the Board of Directors of the Bluefield Project and the N-Lorem Foundation, and is an advisor to several other non-profit organizations. Dr. Chen has served on the National Institute of Neurological Disorders and Stroke (NINDS) National Advisory Council. She also is the Chief Executive Officer and Cofounder of Imago Pharmaceuticals and on the Board of Directors of Nitrome Biosciences.


Karen Chen

Karen S. Chen

CEO, Spinal Muscular Atrophy Foundation

Dr. Tom Crawford has been a member of the Department of Neurology since 1987. He is co-director of the MDA clinic for Neuromuscular Disorders and Neurologist for the Ataxia Telangiectasia Clinical Center at Johns Hopkins. His practice involves general child neurology with a principal interest in caring for children with neuromuscular, neuromotor, and ataxic disorders. Primary research interests involve the basic science and clinical characterization of two important neurologic disorders that affect children: Spinal Muscular Atrophy and Ataxia Telangiectasia. He is also actively involved in the Biology of neurofilaments by characterization of transgenic animal models. He is on the Medical and Scientific Advisory Boards of Families of Spinal Muscular Atrophy, and the Medical Advisory Committee for the Muscular Dystrophy Association. He is the Neurologist for the Ataxia Telangiectasia Clinical Center at Johns Hopkins, which has evaluated almost half of the known patients with this disorder in the United States. Additional specific clinical interests include evaluation and treatment of children with brachial plexus palsies.
Dr. Crawford received his medical degree from the University of Southern California. He completed a pediatric internship and residency at the Pediatric Pavilion of the Los Angeles County / University of Southern California Medical Center, followed by a pediatric chief residency. He completed his training in Neurology with Special Qualification in Child Neurology at the Los Angeles Childrens Hospital. He then traveled east to the laboratory of Dr John Griffin at Johns Hopkins for a fellowship in Neuromuscular Disorders. Prior to medical training and Neurology residency, he majored in Psychology and Religion at Yale College.
Dr. Crawford has published extensively and presented nationally and internationally. He has an active role in teaching medical students and residents in neurology. In addition, Dr. Crawford has special interest and experience in EMG studies of children and adults.

Thomas Crawford

Professor of Neurology and Pediatrics, Johns Hopkins Hospital, Johns Hopkins Children’s Center, Kennedy Krieger Institute             

Dr. Crooke is the founder and former chief executive officer of Ionis Pharmaceuticals. In 2020, Dr. Crooke assumed the executive chairman role at Ionis Pharmaceuticals, continuing to lead research into the molecular mechanisms of antisense oligonucleotides and lead n-Lorem, a charitable foundation he has founded to create and supply antisense oligonucleotide medicines for patients with ultra-rare diseases. During his tenure at Ionis, he has led the scientific development of a new platform for drug discovery, antisense technology and engineered the creation of one of the largest and more advanced development pipelines in the biotechnology industry. Early in Dr. Crooke’s career, he led the creation of the first broad anticancer program in the industry at Bristol-Myers, bringing numerous anticancer drugs to the market in the first five years of his career. He then assumed responsibility for worldwide R&D at SmithKline Beckman. Dr. Crooke has received a number of awards, most recently, the American Chemical Society’s E.B. Hershberg Award for Important Discoveries in Medicinally Active Substances, the Lifetime Achievement Award presented by the Oligonucleotide Therapeutics Society, the Scrip Lifetime Achievement Award and the 2019 Massry Prize. Dr. Crooke received his M.D. and Ph.D. degrees and house staff training at Baylor College of Medicine and has been an active scientist throughout his career. He has published more than 500 scientific publications, edited more than 20 books, has numerous patents, and led the development of more than 23 drugs that have been commercialized.


Stanley Crooke

Chairman of the Board and CEO, Ionis Pharmaceuticals; Founder, CEO and Chairman of the Board, n-Lorem Foundation

Paulo Fontoura is the Senior Vice President and Global Head of Neuroscience and Rare Diseases Clinical Development at Roche, in charge of late stage development programs. In this role, he manages one of the industry-leading pipelines in this area, including the development and launch of ocrelizumab (Ocrevus) in Relapsing and Primary Progressive Multiple Sclerosis, plus several projects in Alzheimer disease, Neuromyelitis Optica, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Huntington disease and Autism Spectrum Disorders.
He joined Roche in 2008 and became VP and Global Head of Neuroscience Translational Medicine in 2010, responsible for the early development of new medicines for neurological and psychiatric diseases, including Multiple Sclerosis, Alzheimer disease, schizophrenia, depression and autism. Later his group pioneered projects in genetically caused Rare Diseases such as fragile X syndrome, Down syndrome, Spinal Muscular Atrophy and Huntington disease. In 2015 he moved to his current position in late stage development.
Paulo received his MD and PhD in Neuroimmunology from the New University of Lisbon, and performed his neurology residency at the Egas Moniz Hospital in Lisbon. He did a postdoctoral fellowship at Stanford University where he made significant contributions to research on antigen-specific therapies such as DNA vaccines for Multiple Sclerosis and spinal cord injury. After graduating, he worked as a neurologist and Assistant Professor of Immunology at the New University of Lisbon, focusing on MS research, in collaboration with the Gulbenkian Foundation, was a Visiting Professor Neurology at Stanford University, and consulted with pharmaceutical and biotechnology companies. More recently, he was a Visiting Professor to the University of Basel and University of Aveiro.
Paulo is a Fulbright Fellow, and has received several awards throughout his career, including a European Charcot Foundation Young Investigator Award, Gulbenkian Investigator and Christopher Reeve Foundation Awards. Since being at Roche he has received the pRED STAR leadership award and the RMM Award of Excellence for his work on Autism.

Global Head and SVP, Neuroscience and Rare Diseases Clinical Development, Roche

Mencía de Lemus is President of the European SMA patient organisation, SMA Europe. Her background on International Relations and International Politics has provided her with the adequate experience to lead the effort to bring a united patient voice to the decision making environment related to the drug development process for SMA. She has been involved in patient organisations since the SMA diagnosis of her two children.
Mencía is convinced that building solid partnerships with all stakeholders that are participating on shaping a better future for people living with SMA is the best way to contribute to the ultimate goal of SMA Europe, which is to bring effective therapies to people living with SMA in a timely and sustainable way, and to ensure optimal care. For this reason, Mencía is leading the efforts to create and consolidate a succesful cooperation with researchers, HCPs, regulators, patient organisations across the globe, and pharmaceutical industry.

Mencía de Lemus

President of SMA Europe; President of FundAME, National SMA patient organisation, Spain

Dr. Oscar Henry Mayer is a Professor of Clinical Pediatrics at The Perelman School of Medicine at the University of Pennsylvania, and an Attending Pulmonologist within the Division of Pulmonology at the Children’s Hospital of Philadelphia (CHOP), where he is the medical director of the CHOP Pulmonary Function Testing (PFT) laboratory. For the last 17 years he has been the pulmonologist in the Neuromuscular Center and in the Center for Thoracic Insufficiency Syndrome since its inception 10 years ago and directs much of the outpatient and inpatient management of these patients. He has broad clinical interests including managing patients with chronic respiratory failure, the pulmonary manifestations of neuromuscular disease, and assessing and managing patients with complex chest wall and spine disease. He is on a number of national committees and study groups involved in pediatric pulmonary function testing, the assessment and management of children with complex thoracospinal disorders and neuromuscular disorders, on which he has lectured nationally and internationally and has published a variety of journal articles and book chapters.

Oscar Henry Mayer

Oscar Mayer

Professor of Clinical Pediatrics, The Perelman School of Medicine at the University of Pennsylvania, Division of Pulmonology, Children’s Hospital of Philadelphia

Kristin Schneeman joined FasterCures in April 2005 as director of programs, with
primary responsibility for its innovation portfolio of projects and activities, focused on best practices in the funding and conduct of medical research and innovative collaborations among players in the research enterprise. Among other initiatives, she runs the TRAIN (The Research Acceleration and Innovation Network) program, which provides a platform for knowledge sharing and relationship building to support the growth of venture philanthropy in medical research.
Kristin brings to FasterCures more than 25 years’ experience in public policy, politics,
academia, and the media. Schneeman served for three years as a senior adviser and policy director to a gubernatorial candidate in Massachusetts, as a policy aide to a U.S. Congressman, and for four years as the front-line manager and chief-of-staff for a senior adviser to Vice President Al Gore. At Harvard University she directed research projects on future challenges facing governments, and on complex negotiations in business, politics, and international relations. Schneeman began her career as a producer of documentary films, for which she was the recipient of an Emmy Award in 1990.

Kristin Schneeman

Kristin_Schneeman

Director, FasterCures, a center of the Milken Institute

Laurent Servais

Professor of Pediatric Neuromuscular Disease, MDUK Oxford Neuromuscular Centre; Head of Specialized Translational Research Oxford Neuromuscular Group: Invited Professor of Child Neurology, Liège University

Lauren has written on emerging technologies and therapeutics since joining BioCentury in 2007. As Senior Editor, she guides BioCentury’s coverage of translation and clinical development and has written extensively on technologies including CRISPR, cell and gene therapies, the microbiome and gene control, as well as IP issues affecting the industry. Lauren holds a B.S.E. in Bioengineering from the University of Pennsylvania and an MBA from Temple University.


Lauren Martz

Lauren

Senior Editor, Head of Translation & Clinical Development, BioCentury

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.

David Chang

CEO, WuXi Advanced Therapies, 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.

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.

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