Dear colleagues and friends,
We are delighted to announce we will be co-hosting the upcoming event “Fragile X Syndrome: Innovative Approaches to Finding a Cure” alongside the <a href="https://www.fraxa.org/">FRAXA Research Foundation</a>, which will be held virtually on July 22, 2022 on Fragile X Syndrome Awareness Day as we continue to shape new thinking in advancing breakthroughs for rare disease patients and to raise public awareness.
This event will feature 30 leading researchers, drug developers, physicians, patients and advocates, and the innovative approaches towards better solutions for patients suffering from Fragile X Syndrome.
This 10th episode of our “Collaborations That Transform” series is part of our common conviction that we must work collaboratively to solve industry-wide problems, so we can bring transformational medicines to patients affected by rare diseases.
Please stay tuned for more program details. Click <a href="https://www.eventbrite.com/e/fragile-x-syndrome-innovative-approaches-to-finding-a-cure-tickets-373626806837?aff=Content">here</a> to reserve your complimentary event pass. For the convenience of our global audience, this program will premiere on July 22nd, 2022 from 8:00am PDT – 10:00am PDT (11:00am EDT – 1:00pm EDT) and from 7:00pm PDT – 9:00pm PDT (10:00pm EDT – 12:00am EDT). We will send an on-demand link to registrants after the event for those who are not able to attend the live premiere, or for those who cannot stay through the entire webinar.
Our featured speakers include:
Katie Clapp, Co-Founder & President, FRAXA Research Foundation
Michael Tranfaglia, Medical Director & CSO, FRAXA Research Foundation
Elizabeth Berry-Kravis, Professor of Pediatrics, Neurological Sciences, Anatomy and Cell Biology, Rush University Medical Center
Peter Kind, Director, Simons Initiative for the Developing Brain, Patrick Wild Centre; Professor of Developmental Neuroscience, University of Edinburgh
Wendy Bruce, Executive Director, Fragile X Association of Australia
Jeannie Lee, Vice Chair of Molecular Biology, MGH; Professor of Genetics, Harvard Medical School
David Hampson, Professor, Department of Pharmaceutical Sciences, University of Toronto
Ralph Laufer, CSO, Lysogene
Ernie Pedapati, Associate Professor of Clinical-Affiliate, Psychiatry and Behavioral Neuroscience, College of Medicine, University of Cincinnati
Raymond Turner, Professor, Department of Biological Sciences, University of Calgary
Kathryn Whitehead, Professor, Departments of Chemical Engineering and Biomedical Engineering, Carnegie Mellon University
Mark Bear, Professor of Neuroscience, Picower Institute for Learning and Memory, MIT
Craig Erickson, Professor of Clinical Psychiatry, Cincinnati Children’s Hospital and the University of Cincinnati College of Medicine
Daniel Alkon, President, Scientific Founder & CSO, Synaptogenix
John Atack, Director, Medicines Discovery Institute, Cardiff University
Mara Dierssen, Senior Scientist, Center for Genomic Regulation
Lili Mao, Co-Founder, Director of Clinical Development Unit, Head of Project Management Office, GEXVal
Christopher Missling, President & CEO, Anavex Life Sciences
Adrian Newman-Tancredi, CEO, Neurolixis
Stephen O’Quinn, VP, Medical Affairs, Zynerba Pharmaceuticals
Robert Ring, CEO, Kaerus Bioscience
Fabio Tucci, Co-Founder & COO, Epigen Biosciences
David Walker, Director of Chemistry, Sentinel Oncology
Bruce Bloom, Chief Collaboration Officer, Healx
Andres Centellas, Board Member, FRAXA Research Foundation
Isaac Bentwich, Founder & CEO, Quris
Randall Carpenter, Co-Founder, Allos Pharma
Lynn Durham, Founder & CEO, STALICLA
Michael Hayden, Founder & CEO, Prilenia Therapeutics; Killam Professor, University of British Columbia
Nik Subramanian, Co-Founder & CTO, Kantify
Gordon Wang, Clinical Assistant Professor, Department of Psychiatry and Behavioral Sciences, Stanford University; Director, Neuroscience Microscopy Service, Wu Tsai Neurosciences Institute
Xinyu Zhao, Jenni and Kyle Professor, Waisman Center and Department of Neuroscience, University of Wisconsin-Madison
Richard Soll, Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec
Hui Cai, Program Chair, VP and Head of Content, WuXi AppTec
At WuXi AppTec, we are dedicated to providing a platform for real change for patients through our “Collaborations That Transform” series. For more information and to view our previous events, please visit the links below.
2022:
<a href="https://wxpress.wuxiapptec.com/events/moving-the-needle-on-als-neuromuscular-disorders/">Moving the Needle on ALS & Neuromuscular Disorders</a>
2021:
<a href="https://wxpress.wuxiapptec.com/events/dmd-charting-the-path-for-new-therapeutics-and-better-care/">DMD: Charting the Path for New Therapeutics and Better Care</a> 
<a href="https://wxpress.wuxiapptec.com/events/fragile-x-syndrome-in-pursuit-of-a-cure/">Fragile X Syndrome: In Pursuit of a Cure</a> 
<a href="https://wxpress.wuxiapptec.com/events/winning-the-war-on-huntingtons-disease/">Winning the War on Huntington’s Disease</a>
<a href="https://wxpress.wuxiapptec.com/events/progress-in-halting-pompe-disease">Progress in Halting Pompe Disease</a>
<a href="https://r20.rs6.net/tn.jsp?f=001a9rn9vm3FxTl-jTCRqk3o946hS_5LvzsHD2Yapd__u830rRjrp61XvBYJtwfsZJqc0LGWvb2Y9dIKDtAJ6lgAXmADAA8upf0lFZ970i51XzbFGbLSBM4J7cWA2bupjYK0Xwxdx3Q4MMtupGdrgqMbVtfOfPuIpppTKVpwDlrYa1wBN4FuIHDxLaRFdge4e623-UTpuTaxVNh3LCgt8TsIflyV8gnq72Prs2kcDg16Wx5djnCu0sfQA==&c=kY4zRw5EJBydmiDDbDkvnST1yo04xiIb9KUMcrZGOzB_qHwXMbtz3Q==&ch=f7Qh9rmVFqY4rGEhxvTjNIXWMw2StkwlQu8ez0yOt_VRIJSga2QiyQ==">WuXi Rare Disease Awareness Concert on International Rare Disease Day 2021</a>
2020:
<a href="https://r20.rs6.net/tn.jsp?f=001a9rn9vm3FxTl-jTCRqk3o946hS_5LvzsHD2Yapd__u830rRjrp61XvBYJtwfsZJqCnCrDT6X6qCg0B_Qq0Sc2IyzytrRd69YHK_NU5-3M1NSNaGH0XDBtxmdTRAhKiM7hay4Nh2aH97n96YhLqm7hlgjLIQBSgYL8MsWZBodvtxLMaddPFzUHkQMwmcnTkBI&c=kY4zRw5EJBydmiDDbDkvnST1yo04xiIb9KUMcrZGOzB_qHwXMbtz3Q==&ch=f7Qh9rmVFqY4rGEhxvTjNIXWMw2StkwlQu8ez0yOt_VRIJSga2QiyQ==">Emerging Opportunities in Multiple Sclerosis and Neuroscience</a> 
<a href="https://r20.rs6.net/tn.jsp?f=001a9rn9vm3FxTl-jTCRqk3o946hS_5LvzsHD2Yapd__u830rRjrp61XtgSjRyc5G5E3Jk2ou8Bwhs4vjfc1Rm3TavCjbFwU0NYpbCwkzrGQxoR1xgCNSA9hd3q7fP_F6WtR_pBN9H_26PMjXARnC1nWYETU2lCmzbir5bv_t7308CV6kyFJE3-BsMCWUMH_pUN&c=kY4zRw5EJBydmiDDbDkvnST1yo04xiIb9KUMcrZGOzB_qHwXMbtz3Q==&ch=f7Qh9rmVFqY4rGEhxvTjNIXWMw2StkwlQu8ez0yOt_VRIJSga2QiyQ==">Spinal Muscular Atrophy (SMA)</a> 
<a href="https://r20.rs6.net/tn.jsp?f=001a9rn9vm3FxTl-jTCRqk3o946hS_5LvzsHD2Yapd__u830rRjrp61XtgSjRyc5G5EJbXfO_E_4bG7uFHJE9fvKfUvF-CMS-b3S3xCRFWOimo9g5XwQnJwSNNJk5W21dUTJb_XpCB0lsKy-mVPlXQFeJ8pn_NON3EUMlYNmZ2mC6rdVPL7YdTmtoPsAt7uFr89W3PL0lKt4dw=&c=kY4zRw5EJBydmiDDbDkvnST1yo04xiIb9KUMcrZGOzB_qHwXMbtz3Q==&ch=f7Qh9rmVFqY4rGEhxvTjNIXWMw2StkwlQu8ez0yOt_VRIJSga2QiyQ==">Advances in Rare Diseases: Hemophilia</a> 
<a href="https://r20.rs6.net/tn.jsp?f=001a9rn9vm3FxTl-jTCRqk3o946hS_5LvzsHD2Yapd__u830rRjrp61XtgSjRyc5G5EjGAdNyAAr_rzNQwsuy46t8C_q8qrdmHwp3fx2G0hofWSLYNujg3Xu06jxUdt7agfBrv3qqSNjHa679PHYMEtSDKxc9oYgFkg_bmuq2kjy43vmyejgrHw7HZTJCEmPB4R&c=kY4zRw5EJBydmiDDbDkvnST1yo04xiIb9KUMcrZGOzB_qHwXMbtz3Q==&ch=f7Qh9rmVFqY4rGEhxvTjNIXWMw2StkwlQu8ez0yOt_VRIJSga2QiyQ==">Neurofibromatosis</a> 
<a href="https://www.eventbrite.com/e/fragile-x-syndrome-innovative-approaches-to-finding-a-cure-tickets-373626806837">Register Now</a>

Fragile X Banner-final_40%

In 1994, after her son Andy was diagnosed with Fragile X, Katie Clapp, her husband Michael Tranfaglia, and a third parent founded FRAXA Research Foundation to find a cure for Fragile X syndrome. Katie has a BA in History from Harvard University and a Masters degree in Computer Science from UNC Chapel Hill. She worked as a software engineer and college instructor in computer science before founding FRAXA. She has won a number of prizes for her work at FRAXA, including a Community Hero award from the American Red Cross and a Women of Justice award from Massachusetts Lawyers Weekly.

Katie Clapp

Co-Founder & President, FRAXA Research Foundation

Dr. Tranfaglia serves as Medical Director and Chief Scientific Officer of FRAXA, coordinating the Foundation's research strategy and working with university and industry scientists to develop new therapeutic agents for Fragile X, autism, and related developmental disorders. He graduated from Harvard University with a BA in Biology and studied medicine at the University of North Carolina at Chapel Hill. After obtaining his MD, he stayed at UNC for Psychiatry residency training, and then entered private practice in 1991 in Newburyport, MA. Mike had specialized in the treatment of Anxiety Disorders prior to his son's Fragile X diagnosis in 1992 and before he and his wife founded FRAXA in 1994.

Michael Tranfaglia

Medical Director & CSO, FRAXA Research Foundation

Elizabeth Berry-Kravis MD, PhD is a Professor of Pediatrics, Neurological Sciences, and Biochemistry at Rush University Medical Center in Chicago. She attended the University of Notre Dame for undergraduate studies and the University of Chicago for her MD/PhD and training in Pediatric Neurology. She moved to Rush in 1992 and established the Fragile X Clinic and Research Program, through which she provides care to over 700 patients with fragile X syndrome (FXS). She has studied medical issues, epilepsy and psychopharmacology in FXS, and has been a leader in translational research in FXS including development of outcome measures/biomarkers, natural history studies, newborn screening, and particularly clinical trials of new targeted treatments. Her laboratory studies cellular roles of fragile X mental retardation protein (FMRP), relationship to phenotypes, and optimization of genetic testing methods. 

In the past decade she has expanded clinical translational work to other neurodevelopmental disorders and genetic neurodegenerative diseases including autism spectrum disorders, Phelan McDermid syndrome, Rett syndrome, Angelman syndrome, Smith-Magenis syndrome, DDX3X syndrome, Niemann-Pick type C, Batten disease, pantothenate kinase-associated neuro-degeneration, and creatine transporter deficiency. She has over 250 publications on genetic neurological diseases and is on Advisory/Review Boards for the FRAXA Research Foundation, National Fragile X Foundation, Phelan McDermid Syndrome Foundation, Rettsyndrome.org, Angelman Syndrome Foundation, Foundation for Angelman Syndrome Therapeutics and GATHER Foundation. She has received the NFXF Jarrett Cole Clinical Award, FRAXA Champion Award, NFXF William and Enid Rosen Research Award, March of Dimes Jonas Salk Research Award, American Academy of Neurology Sidney Carter Award in Child Neurology, John Merck Fund Sparkplug Award, FRAXA Ingenuity Award, and Child Neurology Society Denckla Award for her work in treatment translation for FXS and genetic cognitive disorders.

Elizabeth Berry-Kravis

Professor of Pediatrics, Neurological Sciences, Anatomy and Cell Biology, Rush University Medical Center

Peter Kind

Director, Simons Initiative for the Developing Brain, Patrick Wild Centre; Professor of Developmental Neuroscience, University of Edinburgh

Wendy joined Fragile X Association of Australia in 2013. Wendy is a passionate supporter and advocate for the Fragile X community. She is keen to promote education and awareness of Fragile X and to influence public policy change in the areas of testing, diagnosis and treatment related to Fragile X-associated conditions. Before joining the organisation, Wendy worked in the area of knowledge management with several large professional services firms. She also has wide experience with small member-based community organisations, working in management and volunteer Board roles. Wendy previously served as a volunteer director on the board of Genetic Alliance Australia, a not-for-profit organisation which provides information and peer support to individuals affected by rare genetic conditions. Wendy has a Bachelor of Social Science from RMIT University in Australia. Members of Wendy's extended family are affected by Fragile X.

Wendy Bruce

Executive Director, Fragile X Association of Australia

Jeannie T Lee is The Philip A. Sharp, PhD, Endowed Chair in Molecular Biology, Professor of Genetics (and Pathology) at Harvard Medical School, and is Vice Chair of the Department of Molecular Biology at the Massachusetts General Hospital. Dr. Lee specializes in the study of epigenetic regulation by long noncoding RNAs and uses X-chromosome inactivation as a model system. She is a Member of the National Academy of Sciences, a Harrington Rare Disease Scholar of the Harrington Discovery Institute, a recipient of the Lurie Prize from the Foundation for the National Institutes of Health, an awardee of the Centennial Prize from the Genetics Society of America, the 2010 Molecular Biology Prize and the 2020 Cozzarelli Prize from the National Academy of Sciences, U.S.A, and a Fellow of the American Association for the Advancement of Science. Dr. Lee was also named a Distinguished Graduate of the University of Pennsylvania School of Medicine and an Investigator of the Howard Hughes Medical Institute. From 2013-2018, she co-launched the Epigenetics Initiative at Harvard Medical School and served as its Co-Director. Serving on the Board of Directors of the Genetics Society of America (GSA), Dr. Lee spearheaded the TAGC (The All-Genetics) Conference in 2016. As GSA's President, Dr. Lee established a Strategic Plan and a Development strategy for the society in 2018. She received her A.B. in Biochemistry and Molecular Biology from Harvard University and obtained M.D.-Ph.D degrees from the University of Pennsylvania School of Medicine. Dr. Lee then carried out postdoctoral work at the Whitehead Institute & MIT and became Chief Resident of Clinical Pathology at the Massachusetts General Hospital prior to joining the Faculty at Harvard Medical School. As a new investigator, she received the Basil O'Connor Scholar Award from the March of Dimes and the Pew Scholars Award. To translate basic knowledge to the clinic, Dr. Lee co-founded Translate Bio and Fulcrum Therapeutics, two biotech companies specializing in RNA and epigenetic therapies.

Jeannie Lee

Vice Chair of Molecular Biology, MGH; Professor of Genetics, Harvard Medical School

Kathryn A. Whitehead is a Professor in the Departments of Chemical Engineering and Biomedical Engineering (courtesy) at Carnegie Mellon University. Her lab develops drug delivery systems for RNA, proteins, and applications in maternal and infant health. She obtained bachelor and doctoral degrees in chemical engineering (Univ. of Delaware; Univ. of California, Santa Barbara) before an NIH Postdoctoral Fellowship at MIT. Prof. Whitehead is the recipient of numerous awards, including the NIH Director's New Innovator Award, the DARPA Director's Fellowship, and the ASEE Curtis W. McGraw Research Award. She has also received the Controlled Release Society's Young Investigator Award and served on its Board of Directors. Prof. Whitehead is an elected Fellow of the American Institute for Medical and Biological Engineering and the Controlled Release Society. In 2021, she gave a TED talk on the lipid nanoparticles (i.e., "fat balls") used in the in the COVID-19 mRNA vaccines. Her publications have been cited over 9,000 times, and her patents have been licensed and sublicensed for reagent and therapeutic use. 

Kathryn Whitehead

Professor, Departments of Chemical Engineering and Biomedical Engineering, Carnegie Mellon University

Dr. Ralph Laufer is the Chief Scientific Officer at Lysogene, a gene therapy company based in Paris, focusing on the development of innovative treatments for neurological diseases.  Before joining Lysogene in 2018, Dr. Laufer was Senior Vice President and Head of Discovery and Product Development at Teva Pharmaceutical Industries. Prior to that, he held the positions of Scientific Director of IRBM Science Park, a drug discovery partnering organization in Rome, Italy, and Head of Pharmacology at IRBM-Merck Research Laboratories Rome. He is the recipient of the American Chemical Society 2013 Heroes of Chemistry Award for his role in the discovery and development of Isentress (raltegravir), the first integrase inhibitor approved for use in HIV infected patients. Dr. Laufer's scientific achievements include the discovery of the tachykinin NK-3 receptor and the anti-obesity activity of ciliary neurotrophic factor. He is the author of over 90 peer-reviewed articles and inventor of more than 20 patents. Dr. Laufer obtained his PhD in Biochemistry (summa cum laude) and M.Sc. in Chemistry from the Hebrew University of Jerusalem. He conducted postdoctoral training at the Institut Pasteur in Paris.

Ralph Laufer

CSO, Lysogene

Ernie Pedapati

Associate Professor of Clinical-Affiliate, Psychiatry and Behavioral Neuroscience, College of Medicine, University of Cincinnati

Raymond Turner

Professor, Department of Biological Sciences, University of Calgary

Currently Professor in the Dept. of Pharmaceutical Sciences at the University of Toronto.

Previously was the Director of the Canadian Institutes of Health Research, Strategic Training Program in Biological Therapeutics.

Have worked on the study and development of viral vector gene therapy for treating fragile X syndrome (FXS) using rodent models of FXS for the past 8 years. We are currently partnered with a gene therapy biotech company to continue and complete our preclinical work on this project.

David Hampson

Professor, Department of Pharmaceutical Sciences, University of Toronto

Craig Erickson

Professor of Clinical Psychiatry, Cincinnati Children’s Hospital and the University of Cincinnati College of Medicine

Dr. Mark Bear is Picower Professor of Neuroscience in The Picower Institute for Learning and Memory and the Department of Brain and Cognitive Sciences, Massachusetts Institute of Technology. He was an Investigator of the Howard Hughes Medical Institute for 22 years from 1994 to 2015, and served as Director of the Picower Institute from 2007 to 2009. Prior to moving to MIT in 2003, Dr. Bear was the Sidney A. and Dorothy Doctors Fox Professor at Brown University School of Medicine for 17 years. After receiving his B.S. degree from Duke University, he earned his Ph.D. degree in neurobiology at Brown. He took postdoctoral training from Wolf Singer at the Max Planck Institute for Brain Research in Frankfurt, Germany, and from Leon Cooper at Brown. His long-standing scientific interest in how the brain is modified by experience, and his lab is currently focused on applying knowledge of the elementary mechanisms of synaptic plasticity to overcome genetic or environmental adversity. Bear's research on Fragile X led to the influential mGluR theory and development of new, potentially disease-modifying therapies.

Mark Bear

Professor of Neuroscience, Picower Institute for Learning and Memory, MIT

John Atack

Director, Medicines Discovery Institute, Cardiff University

Dr. Tucci has over 20 years of experience in drug discovery, medicinal chemistry and organic synthesis. In this role, Dr. Tucci is responsible for managing certain aspects of the company, including project management, medicinal chemistry design, investor relations, operations and administration functions. As the Director of Chemistry at Tanabe Research Laboratories, he led the Medicinal Chemistry Department and advanced several programs on inflammation and metabolic diseases to the pipeline of Mitsubishi Tanabe Pharma Corporation in Japan. While at Neurocrine Biosciences, Dr. Tucci served as Principal Scientist in the Medicinal Chemistry Department where he was part of the team that designed and optimized elagolix (Orilissa), a potent small-molecule GnRH antagonist that has recently gained FDA approval. Dr. Tucci received a Ph.D. degree in organic chemistry from the Universidade de São Paulo, Brazil, and conducted postdoctoral studies at the University of Wisconsin, Madison and The Scripps Research Institute, La Jolla. He authored/co-authored 65 peer-reviewed publications and is an inventor in more than 20 patents and patent applications.

Fabio Tucci

Co-Founder & COO, Epigen Biosciences

David is a medicinal chemist with over 20 years' experience in Biotech & Pharma. David has held positions at Pfizer, Cambridge Discovery Chemistry, Millennium Pharmaceuticals & Astex Therapeutics. Whilst at Sentinel Oncology, David has spent over 14 years managing small molecule drug discovery and development projects via an outsourcing model. David is technical lead on Sentinel's program to develop a therapeutic for Fragile X Syndrome. David is a named inventor on multiple patents and co-author on several scientific publications.

David Walker

Director of Chemistry, Sentinel Oncology

Robert Ring

CEO, Kaerus Bioscience

Christopher U. Missling, PhD is the President and CEO of Anavex Life Sciences, has over 20 years of healthcare industry experience within large pharmaceutical and biotech industry. Prior to joining Anavex, he served as Officer at Curis and ImmunoGen and previously at Aventis (now Sanofi). Christopher's work is dedicated to finding potential cures for rare neurodevelopmental diseases, like Rett syndrome, autism spectrum disorder as well as degenerative diseases like Alzheimer's and Parkinson's disease by utilizing precision medicine. Dr. Missling is working with his team to advance new potential treatments through clinical trials involving the respective patient's advocacy groups. Dr. Missling has an MS and PhD from the University of Munich in Chemistry and an MBA from Northwestern University Kellogg School of Management and WHU.

Christopher Missling

President & CEO, Anavex Life Sciences

Lili Mao

Co-Founder, Director of Clinical Development Unit, Head of Project Management Office, GEXVal

Dr. O'Quinn is a senior pharmaceutical executive with over 30 years of experience in clinical development, medical affairs and commercialization of medicines in multiple therapy areas, including neurology and psychiatry. Prior to joining Zynerba, Dr. O'Quinn spent over 20 years with GlaxoSmithKline in senior leadership roles. Following his time at GlaxoSmithKline, he spent 5 years as a consultant to pharmaceutical clients supporting clinical development and medical affairs activities. Dr. O'Quinn earned a Doctor of Pharmacy from the University of North Carolina at Chapel Hill. He completed a post-doctoral fellowship in Cardiovascular Pharmacotherapy with the UNC School of Pharmacy and Division of Cardiology.

Stephen O'Quinn

Stephen O’Quinn

VP, Medical Affairs, Zynerba Pharmaceuticals

Dr. Newman-Tancredi has over 30 years' experience of neuroscience research. He is co-founder and Chief Executive Officer of Neurolixis, a biopharmaceutical company that develops innovative treatments for neurological disorders including autism spectrum disorders (ASD) and Parkinson's disease (PD). Previously, he was Director of Neurobiology at Pierre Fabre Laboratories, a French pharmaceutical company, and responsible for identifying novel antipsychotic, antidepressant and analgesic drug candidates. Prior to his position at Pierre Fabre, Dr. Newman-Tancredi investigated signal transduction mechanisms of monoamine receptors at the Servier Laboratories (another French pharmaceutical company). He has published over 190 articles in peer-reviewed international scientific journals, is co-inventor on a dozen patents and serves as European Councilor for the International Society for Serotonin Research. His principal current focus of interest is the development of first-in-class direct-acting serotonergic "biased agonists" (also known as "functional selectivity"), notably the Phase 1-ready drug candidate, NLX-101, for the treatment of Fragile X syndrome and other ASDs. Other compounds in the Neurolixis pipeline are befiradol (NLX-112) which is undergoing a Phase 2 proof-of-concept study for treatment of dyskinesia in PD patients, and NLX-204, a preclinical stage antidepressant candidate. Dr. Newman-Tancredi previously characterized several approved drugs, including milnacipran (Savella), piribedil (Trivastal), agomelatine (Valdoxan) and levomilnacipran (Fetzima) for various CNS indications.

Adrian Newman-Tancredi

Adrian Newman

CEO, Neurolixis

Daniel Alkon, M.D., serves as President, Chief Scientific Officer and Scientific Founder of the Synaptogenix. Dr. Alkon was the founding Scientific Director of the Rockefeller Neurosciences Institute (BRNI) from 1999 to 2016. He received his undergraduate degree in chemistry in 1965 at the University of Pennsylvania. After earning his M.D. at Cornell University and finishing an internship in medicine at the Mount Sinai Hospital in New York, he joined the staff of the National Institutes of Health where during his 30 year career he became a Medical Director in the U.S. Public Health Service at the National Institute of Neurological Disorders and Strokes and Chief of the Laboratory of Adaptive Systems. Dr. Alkon occupied the Toyota Chair in Neurodegenerative diseases at BRNI from June 2006 until September 23, 2016 and served as Research Professor in Biophysics at the Johns Hopkins University. 

Daniel Alkon

President, Scientific Founder & CSO, Synaptogenix

With a PhD in Neurobiology from the University of Cantabria, she is a research director at the Centre for Genomic Regulation in Barcelona. Her main area of research is inquiring into how the brain brings about the specific behaviour of each individual. In particular, she has specialised in Down syndrome, a field in which she has produced trailblazing internationally recognised research into the genetic bases of intellectual impairment. Moreover, her work in the therapeutic area has led to improvements in cognitive performance for people with Down syndrome. She has received numerous awards during her career as a researcher, notable among them the 2008 National Prize for Thought and Scientific Culture [Generalitat (Government) of Catalonia], and two “Jaime Blanco” prizes for her research into Down syndrome. She has also received other honours for her work of promoting scientific knowledge through lectures and publications. She belongs to, and has been president of the Spanish Society of Neuroscience (2013-2015), the International Behavioral and Neural Genetics Society and the Trisomy 21 Research Society. She is president of the Spanish Brain Council and is elected member of the Academia Europaea, which brings together leading scientists and university professors from around Europe and the Royal Academy of Medicine.

Mara Dierssen

Senior Scientist, Center for Genomic Regulation

Dr. Bruce Bloom is Chief Collaboration Officer of Healx, a Cambridge UK biotech using AI and drug redevelopment to create novel therapies for rare disease patients. He founded and led the global charity Cures Within Reach that brought over a dozen redeveloped drug therapies to patients through proof-of-concept clinical trials, and developed CureAccelerator, the online drug redevelopment collaboration marketplace. Dr. Bloom is an Ashoka Social Entrepreneur Fellow, the Patient Advisory Board Chair for the Institute for Translational Medicine, Board member of the Rare Disease Company Coalition, member of the Vanderbilt Institute for Clinical and Translational Research External Advisory Board, Executive Board member of Mission: Cure, and is on the Science Advisory Boards of the Dr. Ralph and Marian Falk Medical Research Trust Awards Programs, the Findacure Fundamental Disease Charity, the Rare Disease Research Hub of the Westchester Biotech Project, ReBoot Rx, and the editorial board of ASSAY and Drug Development Technologies.

Bruce Bloom

Bruce Bloom_

Chief Collaboration Officer, Healx

Andres Centellas

Board Member, FRAXA Research Foundation

Nik Subramanian is the Chief Technology Officer and co-founder of Kantify, a startup specialized in Artificial Intelligence for life sciences and based in Brussels, Belgium. Before cofounding Kantify, Nik was the Director of International Operations of a European mobility leader, focusing on the implementations of new technologies, and overseeing more than 1200 staff members. Nik is a guest lecturer on Machine Learning at the University of Maastricht and holds a Master in Management, a Master in Finance and a Master in Sociology.

Nik Subramanian

Nik Subramanian_

Co-Founder & CTO, Kantify

Dr. Carpenter is currently a Co-Founder of Allos Pharma, CMO of the Rett Syndrome Research Trust, a Research Affiliate in the Department of Neuroscience at MIT, and a member of the Scientific Advisory Boards of EU-AIMS and of the Translational Neuroscience Center at Boston Children's Hospital. While in industry, he led translational medicine teams responsible for 8 successful IND submissions and over 50 GCP-compliant clinical trials. Prior to joining industry, his academic focus was in translational medicine. He is personally motivated by his experiences as the sibling of an individual with intellectual disability.

Randall Carpenter

Randall Carpenter_

Co-Founder, Allos Pharma

Lynn is the Founder and CEO of STALICLA, a clinical stage biotech company deploying and applying a first-in-class discovery platform to bring precision medicine to patients with Neurodevelopmental Disorders (NDDs), with a first application in Autism Spectrum Disorder (ASD). STALICLA currently has one precision medicine candidate in the clinic and plans to bring two assets into Phase 2 in 2022. Driven by her lifelong involvement with the ASD community, Lynn has instituted a paradigm shift in the NDD drug discovery space by pioneering the creation of the first NDD specific precision medicine discovery platform. In parallel, Lynn has raised $28 M to support STALICLA's development.
 
Lynn has extensive experience in business development and healthcare that she gained through successive positions working for the World Economic Forum, venture initiatives in the Lemanic area and at the Faculty of Medicine of the University of Geneva. Lynn has authored and co-authored 11 patents and is the recipient of a Brain Foundation Award for outstanding contribution to the advancement of treatments in Neurodevelopmental disorders. Lynn holds a Master's degree in political science from ICP Paris, a Master's degree in corporate communication from NEOMA Business School and a post graduate Master's degree in clinical trials and drug development from the Faculty of Medicine of the University of Geneva.

Lynn Durham

Founder & CEO, STALICLA

I came to the way of Neuroscience by literature and engineering. After brief stints in graphic design and economic policy prediction, I completed my Ph.D. with Dr. Mu-Ming Poo at UC Berkeley. I further honed my academic and technological acumen with Dr. Stephen Smith and Philippe Mourrain at Stanford. I am currently a Clinical Assistant Professor in the Department of Psychiatry and Behavioral Sciences at Stanford. My lab focuses on developing and using spatial proteomic techniques from sub-cellular to brain level to address the role of metabolic stress from circuit to synapses in development, aging and sleep.

Gordon Wang

Gordon Wang_

Clinical Assistant Professor, Department of Psychiatry and Behavioral Sciences, Stanford University; Director, Neuroscience Microscopy Service, Wu Tsai Neurosciences Institute

Dr. Xinyu Zhao graduated from Peking (Beijing) University with a B.S. degree in Biology and then earned her Ph.D. degree in Pharmacology from the University of Washington (Seattle). She obtained postdoctoral training on neural stem cells from Dr. Fred Gage at the Salk Institute for Biological Studies (La Jolla, California). Dr. Zhao started her own independent research at the University of New Mexico School of Medicine in 2003 and then moved to the University of Wisconsin-Madison in 2011. She is currently a Jenni and Kyle Professor in the Department of Neuroscience and an investigator of the Waisman Center. Dr. Zhao's research focuses on understanding the molecular mechanisms that regulate brain development with the goal of developing novel treatment strategies for neurodevelopmental disorders including fragile X syndrome. She was recognized with a Research Award from the National Fragile X Foundation in 2018. 

Xinyu Zhao

Jenni and Kyle Professor, Waisman Center and Department of Neuroscience, University of Wisconsin-Madison

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 Therapeutics, 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 Disorders 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.

Michael Hayden

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

I am a medical doctor by training and am the founder and CEO of Quris, an AI-Pharma company that is a leader in AI drug safety prediction. Quris' first AI-based drug, for Fragile X, is preparing for clinical testing. Prior to Quris I founded and led three other AI life-science companies that led AI revolutions in medicine, genomics and agriculture. One of these developed a groundbreaking medical decision-support software and was acquired by McKesson (formerly HBOC). A second company I founded, Rosetta Genomics (Forbes: Treasures in the Trash, 2005) used AI to analyze the human genome, finding hundreds of all-important microRNA genes, more so than all of the world's universities combined, and applied these genes to cancer diagnostics; Rosetta's subsidiary, Rosetta Green was acquired by Monsanto for $35M. A third company I founded, CropX (FORBES: Top 50 AgTech Companies, 2020) uses AI and soil sensors to analyze agricultural fields, allowing farmers to grow more crops with less water and other resources. 

Isaac Bentwich

Founder & CEO, Quris

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.

Richard Soll

Rich Soll

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

Dr. Hui Cai joined WuXi AppTec in 2009 as Vice President of Business Development, and is currently Vice President and Head of Content Division.

Prior to WuXi, Dr. Cai spent 10 years at Johnson & Johnson Pharmaceutical Research and Development leading multiple drug discovery programs in the therapeutic areas of inflammation and autoimmune diseases. She is a co-author and co-inventor to over 50 scientific publications and issued or pending patents. Dr. Cai is a Councilor of the American Chemical Society (ACS), a member of BayHelix, and a member of the UCSD Alumni Board. In her past capacity, she served as a Commissioner at the City of San Diego Science and Technology Commission, Chair of SABPA, and President of SDCA. Dr. Cai received her BS and MS in Chemistry from Peking University, PhD from The Scripps Research Institute, and MBA from UCSD Rady School of Management as a DLA Piper – Athena Scholar.

Hui Cai

hui-cai

VP and Head of Content, WuXi AppTec

biotech

Events

Featured

Fragile X

Fragile X Syndrome

Rare Disease

rare disease

Fragile X Syndrome: Innovative Approaches to Finding a Cure

<a href="#hui-cai">Hui Cai</a>, Program Chair, VP &amp; Head of Content, WuXi AppTec 
<a href="#calaneet-balas">Calaneet Balas</a>, President &amp; CEO, The ALS Association 
<a href="#lin-kah-ooi">Lin Kah Ooi</a>, Founding President, Motor Neurone Disease Association Singapore

Welcome

<a href="#carsten-bonnemann">Carsten Bönnemann</a>, Chief, Neuromuscular &amp; Neurogenetic Disorders of Childhood Section; Senior Investigator &amp; Acting Chief, Neurogenetics Branch, NINDS, NIH 
<a href="#matthew-harms">Matthew Harms</a>, Associate Professor of Neurology, Institute for Genomic Medicine, Columbia University 
Moderator: <a href="#sharon-hesterlee">Sharon Hesterlee</a>, Chief Research Officer, Muscular Dystrophy Association

The Unmet Needs in Neuromuscular Disorders 

<a href="#kuldip-dave">Kuldip Dave</a>, SVP of Research, The ALS Association 
<a href="#sabrina-paganoni">Sabrina Paganoni</a>, Co-Director, Neurological Research Institute at Massachusetts General Hospital; Physician Scientist, Healey &amp; AMG Center for ALS 
<a href="#jeffrey-rothstein">Jeffrey Rothstein</a>, Director of Brain Science Institute, Director, Robert Packard Center for ALS Research, Johns Hopkins University

Spotlight on ALS 

<a href="#neil-cashman">Neil Cashman</a>, CSO, ProMIS Neurosciences; Professor Emeritus, University of British Columbia 
<a href="#michael-mcgrath">Michael McGrath</a>, UCSF Emeritus Professor of Medicine; CSO &amp; Founder, Neuvivo 
<a href="#robert-scannevin">Robert Scannevin</a>, CSO, Verge Genomics 
<a href="#fernando-vieira">Fernando Vieira</a>, CEO &amp; CSO, ALS Therapy Development Institute 
Moderator: <a href="#murali-gopalakrishnan">Murali Gopalakrishnan</a>, Global Head, Neuroscience Search &amp; Evaluation, Corporate Strategy Office, AbbVie

In the Pipeline 

<a href="#jim-burns">Jim Burns</a>, CEO, Locanabio 
<a href="#clive-svendsen">Clive Svendsen</a>, Professor &amp; Executive Director, Board of Governors Regenerative Medicine Institute 
Moderator: <a href="#bibhash-mukhopadhyay">Bibhash Mukhopadhyay</a>, Managing Partner, Sound Bioventures

Challenges and Opportunities in New Modalities 

<a href="#michael-snyder">Michael Snyder</a>, Professor &amp; Chair, Department of Genetics; Director, Center for Genomics and Personalized Medicine, Stanford University

The Future of Genetic and Personalized Medicines 

<a href="#richard-soll">Richard Soll</a>, Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec

Closing Remarks 

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: <a href="https://www.als.org/">ALS Association</a>, <a href="https://www.mda.org/">Muscular Dystrophy Association</a>, <a href="https://www.mnda.org.sg/">Motor Neurone Disease Association Singapore</a>, and <a href="http://www.jiandongren.org/">Beijing Oriental Rain ALS/MND Care Center</a>.
You are welcome to watch this event on-demand here: <a href="https://youtu.be/ITcuG6oD_KM">https://youtu.be/ITcuG6oD_KM</a>
Thank you again, and make sure to follow us on <a href="https://r20.rs6.net/tn.jsp?f=001tdaY26Eu5DakCjhvoEbxHbj7qbiNoBJ47ciVYpkrMHCsp46_igPlsAhxo_UZF_DNcjDk_qcrWGnhvxq67FqG4-99_OnG-Tizm5Hvo2fZa4Wugd7VDt4HgHA5-G_bSsVg2N9TqZxpAp6W2OBaofYbapyi5pwWbnVH&c=bnff8h6Kfj-Phei4iHfn5eCX_lD-5hoF9ooEY2hjpNMJii4cbewC2A==&ch=0gqjl4DDURjwghMJVQXHPXnMg2IWWgG6tH96VUzTHeDg0lPQyqxlLQ==">Twitter</a> and <a href="https://r20.rs6.net/tn.jsp?f=001tdaY26Eu5DakCjhvoEbxHbj7qbiNoBJ47ciVYpkrMHCsp46_igPlsFzFDxTRfhgpW8qsKVXpt2YnQHGTiX_SeL04n-x_173AWktJqdo73-e875lMUWIcADygwgs4ogcLlfwJ2mHHKzX7ouOF0sFAj6s82QB7hJGYXeBWDZWRag8CGe87SNVznQ==&c=bnff8h6Kfj-Phei4iHfn5eCX_lD-5hoF9ooEY2hjpNMJii4cbewC2A==&ch=0gqjl4DDURjwghMJVQXHPXnMg2IWWgG6tH96VUzTHeDg0lPQyqxlLQ==">LinkedIn</a>, and visit our WXPress website to stay up-to-date on upcoming WuXi AppTec news and events.
If you have any questions, please contact us at <a href="mailto:wuxiforum@wuxiapptec.com">wuxiforum@wuxiapptec.com</a>.

ALS-banner

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.

Calaneet Balas

Calaneet balas high res

President & CEO, The ALS Association

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.

Lin Kah Ooi

Founding President, Motor Neurone Disease Association Singapore

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.

Carsten Bönnemann

Carsten Bonnemann

Chief, Neuromuscular & Neurogenetic Disorders of Childhood Section; Senior Investigator & Acting Chief, Neurogenetics Branch, NINDS, NIH

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.

Matthew Harms

Matthew Harms Headshot

Associate Professor of Neurology, Institute for Genomic Medicine, Columbia University

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.

Sharon Hesterlee

Chief Research Officer, Muscular Dystrophy Association 

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.

Kuldip Dave

SVP of Research, The ALS Association 

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. 

Sabrina Paganoni

Co-Director, Neurological Research Institute at Massachusetts General Hospital; Physician Scientist, Healey & AMG Center for ALS 

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.

Jeffrey Rothstein

Director of Brain Science Institute, Director, Robert Packard Center for ALS Research, Johns Hopkins University 

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.  

Murali Gopalakrishnan

Global Head, Search & Evaluation-Neuroscience in the Corporate Strategy Office, AbbVie

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.

Neil Cashman

CSO, ProMIS Neurosciences; Professor, Emeritus University of British Columbia 

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.

Michael McGrath

UCSF Emeritus Professor of Medicine; CSO & Founder, Neuvivo

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.

Robert Scannevin

CSO, Verge Genomics 

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.

Fernando Vieira

Fernando Vieira_

CEO & CSO, ALS Therapy Development Institute 

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.

Bibhash Mukhopadhyay

Managing Partner, Sound Bioventures

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.

Jim Burns

CEO, Locanabio 

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.

Clive Svendsen

Clive Svendsen_

Professor & Executive Director, Board of Governors Regenerative Medicine Institute 

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.

Michael Snyder

Professor & Chair, Department of Genetics; Director, Center for Genomics and Personalized Medicine, Stanford University 

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.

Events

UPCOMING EVENT

Fragile X Syndrome: Innovative Approaches to Finding a Cure

Events, Rare Disease

EVENT RECAP

Moving the Needle on ALS & Neuromuscular Disorders

Events