Dear Colleagues & Friends,
We are honored to have been able to unite 3,000+ colleagues this year from around the world for our virtual event, “Moving the Needle on ALS & Neuromuscular Disorders,” on June 21st, 2022. Together we were able to enhance awareness and provide a global stage for key stakeholders to connect and collaborate to overcome barriers to finding cures for these devastating rare diseases.
This is our 9th episode of our “Collaborations That Transform” series. At WuXi AppTec, we are dedicated to providing a platform to catalyze transformational change for patients.
Special thanks to our more than 20 distinguished speakers, and to our supportive partners: ALS Association, Muscular Dystrophy Association, Motor Neurone Disease Association Singapore, and Beijing Oriental Rain ALS/MND Care Center.
You are welcome to watch this event on-demand here: https://youtu.be/ITcuG6oD_KM
If you have any questions, please contact us at firstname.lastname@example.org.
Carsten Bönnemann, Chief, Neuromuscular & Neurogenetic Disorders of Childhood Section; Senior Investigator & Acting Chief, Neurogenetics Branch, NINDS, NIH
Matthew Harms, Associate Professor of Neurology, Institute for Genomic Medicine, Columbia University
Moderator: Sharon Hesterlee, Chief Research Officer, Muscular Dystrophy Association
Kuldip Dave, SVP of Research, The ALS Association
Sabrina Paganoni, Co-Director, Neurological Research Institute at Massachusetts General Hospital; Physician Scientist, Healey & AMG Center for ALS
Jeffrey Rothstein, Director of Brain Science Institute, Director, Robert Packard Center for ALS Research, Johns Hopkins University
Neil Cashman, CSO, ProMIS Neurosciences; Professor Emeritus, University of British Columbia
Michael McGrath, UCSF Emeritus Professor of Medicine; CSO & Founder, Neuvivo
Robert Scannevin, CSO, Verge Genomics
Fernando Vieira, CEO & CSO, ALS Therapy Development Institute
Moderator: Murali Gopalakrishnan, Global Head, Neuroscience Search & Evaluation, Corporate Strategy Office, AbbVie
Michael Snyder, Professor & Chair, Department of Genetics; Director, Center for Genomics and Personalized Medicine, Stanford University
Richard Soll, Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec
Calaneet Balas became CEO and President of The ALS Association in December 2017. She joined the organization in June 2016 as Chief of Strategy, leading all three mission areas including global research, public policy and care services, which work in an integrated fashion to find a cure, advance treatments and enhance the quality of life for people living with ALS. Prior to joining the fight against ALS, Calaneet served as Chief Executive Officer of the Ovarian Cancer National Alliance (OCNA). In December 2018, she was elected as Chairwoman of the International Alliance of ALS/MND Associations. She also sits on the board of the Truman State University Foundation. Ms. Balas holds a Master of Business Administration from Herriot-Watt University, a Master of Science in Human Movement Science Education from the University of Memphis, and a Bachelor of Science in Exercise Science from Truman State University.
Lin Kah was the Founding President of the Motor Neurone Disease Association Singapore (MNDa@Singapore).
He is a late stage ALS patient. After his diagnosis in 2013, he became wheelchair-bound (since 2017), and has been physically sustained by Non-invasive Ventilation (2018), PRG feeding tube (2020) and Tracheostomy (2021). He has been using the eye gazing technology to communicate since 2019, after losing his voice.
Lin Kah, aged 64, is being taken care of at his house by his wife, helper and 3 adult children. He realises the importance and appreciation of the support from family, friends and medical professionals, as well as spiritual guidance. He found calmness and the meaning of his illness in Theravada Buddhism (2017). He has pledged to donate his brain and other related organs and tissues to Brain Bank Singapore for research to find a cure, upon his passing.
In 2021, Lin Kah led a group of MND patients, caregivers and volunteers to form the MNDa@Singapore on 27 April 2021. The Vision is: "Every MND Patient and Caregiver to live their lives with Dignity, Care and Hope". Since then, MNDa@Singapore has rolled out some outreach and awareness programmes, including actively making the Association known to the MND community via social media and hospitals, hospices, etc. The aim is to let the patients and caregivers know that they are not alone and there is a self-help platform for sharing of experiences and information, and more importantly, the mutual support and encouragement are available.
Before his diagnosis, Lin Kah was a registered Professional Engineer (Civil) working in Singapore's public service. During his professional career, he was involved in environmental management and engineering projects, as well as public health management. He was also the Assistant CEO (2008-2011) of the on-going joint-government Sino-Singapore Tianjin Eco-City project.
Carsten Bönnemann graduated from Medical School at the University of Freiburg in Germany. He trained in pediatrics in Hamburg and Göttingen (where he was awarded the Habilitation in pediatrics), and in neurology/child neurology at Massachusetts General Hospital/Harvard Medical School in Boston. He did postdoctoral research in genetics and neuromuscular specialty training at Boston Children’s Hospital/Harvard Medical School. From 2002 he was Co-Director of the Neuromuscular Program and Director of the Pediatric Neurogenetics Clinic at Children's Hospital of Philadelphia/University of Pennsylvania School of Medicine, where he continues to be on faculty as Adjunct Full Professor of Neurology. In 2010 he joined the NIH as a tenured Senior Investigator and Chief of the Neuromuscular and Neurogenetic Disorders of Childhood Section in the Neurogenetics Branch of the National Institute of Neurological Disorders and Stroke. Clinical, genomic and translational work in the Section centers in particular around early onset neuromuscular disorders such as the congenital myopathies and congenital muscular dystrophies and on the development of molecular and gene directed treatment approaches to these conditions, including first-in-human intrathecal and intravenous AAV mediated gene transfer trials. Dr. Bönnemann was a Pew Fellow in the Biomedical Sciences,in 2010 received the Derek Denny-Brown Neurological Scholar Award from the American Neurological Association and in 2022 the Legacy Award of the Muscular Dystrophy Association USA for excellence in clinical research. He is Co-Editor-in-Chief of the Journal of Neuromuscular Diseases (JND). He has authored more than 330 papers, reviews and chapters in the field of pediatric neuromuscular and neurogenetic disorders.
Matthew Harms, MD is an Associate Professor of Neurology at Columbia University in New York City. During neuromuscular medicine and clinical neurophysiology fellowships he became interested in Neurogenetics and uncovered the genes for dominant spinal muscular atrophy and limb-girdle muscular dystrophy type 1D. At Columbia, his laboratory straddles the Motor Neuron Center and the Institute for Genomic Medicine, with a focus on generating, integrating, and analyzing clinical, genomic and transcriptomic datasets in amyotrophic lateral sclerosis and other neurological disorders. Dr. Harms sees patients in the Eleanor and Lou Gehrig ALS Center and both the adult and pediatric MDA Clinics.
Dr. Hesterlee is Chief Research Officer of the Muscular Dystrophy Association. She has over 20 years of experience in neuromuscular research in both the nonprofit and industry space. She has served as head of research for Parent Project Muscular Dystrophy, the Myotonic Dystrophy Foundation, the Association for Frontotemporal Degeneration and the Muscular Dystrophy Association’s MDA Venture Philanthropy. Dr. Hesterlee has also served as project lead for rare disease gene therapy programs at Pfizer, Inc; as Chief Executive Officer of Lion Therapeutics, a special purpose entity of Askbio, Inc.; and as Executive Vice President Portfolio development at Askbio Inc. She has been involved in numerous efforts to remove barriers to therapy development for rare disease and foster interactions between patient advocacy groups and industry. Dr. Hesterlee has served on the governing board of the Health Research Alliance, the NINDS Council, and the Department of Health and Human Service's Muscular Dystrophy Coordinating Committee. She currently serves on the Congressionally Directed Medical Research Program’s Duchenne Muscular Dystrophy Programmatic Review Panel. She received her Ph.D. in neuroscience from the University of Arizona in 1999.
Dr. Kuldip Dave is Senior Vice President of Research and joined The ALS Association in May 2019. In this role, he leads the world’s largest philanthropic research funding program for ALS. He develops and implements the broader research vision by setting annual priorities and department goals including research funding strategy, research programs, and strategic initiatives and is also responsible for the operational, staffing, and budgetary oversight of the research program. Dr. Dave received his bachelor’s degree in Biology from Rutgers University. He received his Ph.D. degree in Pharmacology and Physiology from Drexel University College of Medicine in Philadelphia. He went on to work for pharmaceutical industry for 2 years before joining The Michael J. Fox Foundation for Parkinson’s research. After nine years at MJFF, he joined the ALS Association as the head of the Research program.
Sabrina Paganoni, MD, PhD, is the Co-Director of the Neurological Research Institute at the Massachusetts General Hospital, physician scientist at the Healey & AMG Center for ALS, and Associate Professor at Harvard Medical School. Her research focuses on clinical trials and therapy development for ALS. She has served as PI of several ALS clinical trials and has been using novel trial designs, novel endpoints and digital technology tools to innovate the way investigational products are tested in ALS. She is the co-PI of the HEALEY ALS Platform Trial, the first platform trial for ALS in the world. She recently reported the positive results of the CENTAUR trial and is the co-Chair of the global PHOENIX trial. Her research has been funded by the NIH, non-profits, and industry; she published more than 100 peer-reviewed manuscripts and received several awards for her work including the 2021 Top 10 Clinical Research Achievement Award for the CENTAUR trial.
Dr. Rothstein is Professor of Neurology and Neuroscience at Johns Hopkins University. He is the Founder and Director of the Robert Packard Center for ALS Research and the Director of the Brain Science Institute and the Founder and Co-Director of the ALS Clinic. His career has focused on ALS pathophysiology and the development of model systems to validate therapies. These efforts began in 1990 when his lab first discovered that excitotoxicity might be a common pathophysiological process in sporadic ALS. With the discovery of the C9orf72 gene mutation, his lab in collaboration with Ionis pharma, discovered on the development of antisense oligonucleotides therapy for C9 ALS/FTD patients. His lab has made discoveries on fundamental pathways that underlay familial and sporadic ALS including: excitotoxicity, astroglial dysfunction, oligodendroglial dysfunction and the role of nuclear pore complex and nucleocytoplasmic transport in familial and sporadic ALS. He developed the Robert Packard Center for ALS research as a model organization of mandatory academic collaboration and data sharing and the development of preclinical models. Recently he founded the Answer ALS program, a comprehensive biological program to understand sporadic ALS and to be the starting point for “personalized medicine” approach to ALS. The Answer ALS program combines longitudinal clinical data, at home smartphone data collection and the generation of iPS neurons from each patient, and their comprehensive biological analytics, leading to a data set of 6 billion biological and clinical data points per patient. This program freely shares this enormous data collection to academic and companies worldwide.
In prior roles, Murali held various positions of increasing responsibility at Abbott / AbbVie leading research programs across multiple therapeutic areas including neuroscience, pain, nephrology and urology, and delivering multiple clinical candidates to the R&D pipeline. At Abbott, Murali has also served as the Head of the Global External Research group, with responsibility for creating and leading a Discovery-focused external innovation team focused on the establishment of partnerships on emerging science, targets, technologies and preclinical assets. Subsequently, he assumed responsibility for the growth and leadership of AbbVie China R&D Center in Shanghai, and subsequently led the Renal Therapeutic Area as Discovery Head, across sites in Chicago & Shanghai, and via a network of external partnerships. More recently, he has taken on a leadership position within AbbVie’s Search & Evaluation team as Global Head, Search & Evaluation in Neuroscience with responsibilities for sourcing and enabling external partnerships.
Murali obtained his undergraduate degree in Pharmacy (B. Pharm) from Banaras Hindu University, India, PhD in Pharmacology from School of Pharmacy, University at Buffalo, New York, and subsequently completed his post-doctoral training and MBA degree. He has published over 150 papers including review articles and contributed to 15 US patents.
Two central ideas inform my scientific work: first, neurodegenerative diseases develop via prion-like propagated protein misfolding, and second, antibodies are well suited to specifically bind to misfolded proteins while sparing normal proteins from autoimmune attack. I originally applied these ideas to prion disease itself, identifying targets for vaccination against animal prion infections (e.g., Paramithiotis et al, Nat Med 2003). More recently, I have applied this knowledge to develop novel therapeutics for amyotrophic lateral sclerosis (ALS) and Alzheimer's disease (AD).
For ALS, I discovered that propagated protein misfolding of Cu/Zn superoxide dismutase (SOD1) could be blocked by antibodies that may arrest clinical progression of this disease (e.g., Grad et al, PNAS 2014). For AD, I identified Aβ oligomer-specific epitopes; antibodies directed against these epitopes can neutralize the toxicity of oligomers, while sparing the non-toxic forms of Aβ, which will enable a safe and effective AD treatment (e.g., Silverman et al, ACS Chem Neurosci 2018, Gibbs et al Scientific Reports 2019). To translate lab discoveries to clinic, I have been a prolific inventor (>100 patent applications) and founded ProMIS Neurosciences. As a bibliometric indicator of my output, I have published as corresponding author in high impact journals including the New England Journal of Medicine (Impact Factor=53.3), Nature (IF=36.3); Cell (IF=32.4), Nat Rev Drug Discov (IF=29), Nature Medicine (IF=22.5), and PNAS (IF=9.7). By Google Scholar metrics, I have over 19,000 citations (>5,500 since 2016) with a cumulative H-index of 68.
Michael S. McGrath, MD, PhD is Emeritus Professor of Medicine at The University of California San Francisco (UCSF). Dr. McGrath earned his BS in Biology at the University of Minnesota. He obtained his MD at Stanford University School of Medicine and his PhD in Cancer Biology also at Stanford.
He has authored over 200 scientific articles in the fields of AIDS, cancer and neurological disease since joining UCSF in 1985 and has recurring NIH funding to support his research programs in excess of $1.5M per year.
Dr. McGrath has been a pioneer in the recognition of the role that inflammation, and especially macrophages, play in multiple diseases. His research focus has been on the study of macrophage activation in the pathogenesis of chronic diseases and over the past 20 years, amyotrophic lateral sclerosis (ALS).
Dr. McGrath co-founded and is the Chief Scientific Officer of Neuvivo Inc. (Palo Alto, CA) with Dr. Ari Azhir and Vern Norv iel in 2020 to continue the development of a macrophage targeted approach to the treatment of ALS using a novel first in class immunoregulatory molecule, NP001. Earlier studies of NP001 in ALS patients with moderate levels of inflammation showed significant activity in slowing both disease progression and loss of respiratory function. Neuvivo is currently in discussion with the FDA regarding next steps towards obtaining approval of NP001 for use in ALS.
Dr. Scannevin has more than 21 years of leadership experience in drug discovery and development at both large pharma and early-stage biotech, and has seen multiple drugs from discovery through FDA approval. Before joining Verge, Dr. Scannevin was the Head of Research at Yumanity Therapeutics where he oversaw biology and chemistry research focused on new target discovery and therapeutic development for neurodegenerative diseases. While at Yumanity, he built a robust and efficient drug discovery function that delivered a deep discovery and clinical development pipeline. Dr. Scannevin provided scientific leadership from early stages through multiple successful financing rounds, a collaboration with a major large pharmaceutical company, and to a successful public debut.
Prior to Yumanity, Dr. Scannevin held leadership roles at Biogen, Johnson and Johnson and Wyeth. While at Biogen, Dr. Scannevin was involved in delivering multiple candidates into clinical development, and was heavily involved in the development and worldwide marketing approval of Tecfidera® (dimethyl fumarate), an oral therapeutic for multiple sclerosis, in which he served as the Global Research Lead for this first-in-class therapy.
Dr. Scannevin earned his Ph.D. at SUNY Stony Brook in the laboratory of Dr. James S. Trimmer, and received his postdoctoral training at the Johns Hopkins University School of Medicine in the laboratory of Dr. Richard Huganir. He is a co-author on more than 55 peer-reviewed research articles and an inventor on over a dozen patent applications and issued patents.
Fernando Vieira is the CEO and Chief Scientific Officer at the ALS Therapy Development Institute where he leads a multidisciplinary team of scientists and researchers in their efforts to discover and develop effective treatments and biomarkers for amyotrophic lateral sclerosis (ALS). He has been focused on ALS research since 2001. His research findings – spanning basic discovery, preclinical drug discovery and optimization, clinical development, and translational ALS research have been widely cited. Specifically, he has focused on optimizing animal models for ALS preclinical pharmacology and drug screening. He has led or contributed to the identification and preclinical validation of four drugs that have been advanced into human clinical assessment and has been awarded multiple patents for those drugs. His teams have contributed important basic research findings to the study of genetic ALS, focusing on SOD1 mutation biology and C9orf72 mutation biology. Dr. Vieira also serves on the Scientific Advisory Board of the ALS Investment Fund. Dr. Vieira received his medical degree from Harvard Medical School and a Bachelor’s degree in Biological Engineering from the University of Florida.
Bibhash is a venture capital fund manager, investor, company builder and board member with a dozen years of experience in biotech/pharma. Prior to founding Sound Bioventures, Bibhash worked as an investor with a therapeutic area focused fund (Pivotal Life Sciences) and with the biotech practice of a large, diversified venture fund (NEA). Prior to that he has operating experience in BD and corporate strategy from both Astra Zeneca and Johnson & Johnson. He has been involved in numerous private investments, M&A deals and IPOs from venture experience and multiple major in-licensing, divestiture and acquisitions from pharma experience, in multiple cross-border (Europe / US) deals and on boards of multiple private and public companies as well as advisory roles in incubators.
James Burns joined Locanabio in December 2019 as the CEO and Director of the Board. Dr. Burns joins us from Casebia, where he served as the chief executive officer and led the team in discovering and developing new CRISPR/Cas9-based breakthrough therapeutics to treat blood disorders, blindness and heart disease. Prior to Casebia, he spent the bulk of his career at Sanofi-Genzyme, where he held several leadership roles with increasing responsibility, including North America Site head for R&D where he coordinated R&D operations across key therapeutic areas. Dr. Burns is a former board member of MassBio, a member of the National Academy of Engineering and is a member of the External Advisory Committee for the Biomedical Engineering Department at Boston University. He also served as the industry representative for the Food and Drug Administration General Plastic Surgery Panel. Dr. Burns earned a Bachelor of Science degree in biology from Purdue University and Master of Science and Doctorate degrees in bioengineering from the University of Illinois-Chicago, where his thesis work focused on drug delivery. Following his graduate studies, he was a post-doctoral researcher at the University of Florida.
Dr. Clive Svendsen did his pre-doctoral training at Harvard University. He received his PhD from the University of Cambridge in England, where he subsequently became a Welcome Fellow and established a laboratory focusing on stem cell research. He then moved to the University of Wisconsin in 2000 as Professor of Neurology and Anatomy and founded their Stem Cell and Regenerative Medicine Center. In 2010, he moved to Los Angeles and founded the Cedars-Sinai Board of Governors Regenerative Medicine Institute (RMI), which currently has 23 faculty members and over 120 staff. A main focus of the Institute is to both model and treat various human diseases with the use of induced pluripotent stem cells (or iPSCs), which are adult human cells taken back in time to a young stem cell state capable of making any tissue of the human body. At the heart of the RMI is the Cedars-Sinai Biomanufacturing Center which manufactures iPSCs and other cell types for research purposes and clinical trials. Additionally, Dr. Svendsen maintains a large lab that focuses on modeling neurodegenerative diseases including Spinal Muscular Atrophy and Amyotrophic Lateral Sclerosis (ALS), as well as Huntington’s, Parkinson’s and Alzheimer’s Disease. Using “organ on chip” technology that combines stem cells and engineering, Dr. Svendsen is creating multicellular human systems for research and drug development. He has received many recognitions for this work, including a recent feature on the front cover of National Geographic as “The Future of Medicine”. The other focus of his lab involves cutting-edge clinical trials using combinations of stem cells and growth factors. He spearheaded one of the first clinical trials to deliver a growth factor (GDNF) to patients with Parkinson’s Disease, and is currently working closely with neurosurgeons, neurologists and other scientists to develop novel ways of using stem cells modified to release GDNF for Parkinson’s patients. He is also finding ways to use stem cells for other neurological diseases such as Huntington’s and Alzheimer’s Disease, and is leading a new clinical trial using stem cells to treat an incurable eye disease Retinitis Pigmentosa. Finally, Dr. Svendsen has a long-standing interest in ALS and he was the Sponsor of the first-ever clinical trial delivering stem cells and GDNF to patients that was recently completed at Cedars-Sinai.
Michael Snyder is the Stanford Ascherman Professor and Chair of Genetics and the Director of the Center of Genomics and Personalized Medicine. He received his Ph.D. training at the California Institute of Technology and carried out postdoctoral training at Stanford University. Dr. Snyder has pioneered the use of “big data” and multiomics to advance scientific discovery and transform healthcare. His laboratory has invented many technologies that are widely used in medicine and research, including methods for characterizing genomes and their products (e.g. RNA-Seq, NGS paired end sequencing, ChIP-Chip and later ChIP-Seq, protein arrays, machine learning for disease gene discovery, and wearables for monitoring personal health and exposures). His application of omics and wearable technologies to perform longitudinal profiling of people when they are healthy and ill is transforming medicine and healthcare. Indeed, his laboratory’s recent work to use smartwatches and wearables to detect illness, including infectious disease such as COVID-19, prior to symptom onset is being used by many thousands of people. He has helped colead many large scale projects including ENCODE, HMP, HuBMAP and HTAN. He has cofounded many biotechnology companies, including Personalis, Q Bio, January AI, Filtricine and RTHM.
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.