Fragile X Syndrome: Innovative Approaches to Finding a Cure

EVENT RECAP
July 22, 2022
Events

In commemorating Fragile X Syndrome Awareness Day on July 22nd, we are proud to have co-hosted the special virtual event, “Fragile X Syndrome: Innovative Approaches to Finding a Cure,” again alongside the FRAXA Research Foundation. Together we were able to enhance public awareness of Fragile X, and provide a global stage for key stakeholders to collaborate in order to overcome any barriers standing in the way of finding a cure for this devastating rare disease.

This is the 10th episode of our “Collaborations That Transform” series dedicated to rare disease patients. At WuXi AppTec, we are committed to providing a platform serving to catalyze transformational change for patients.

Special thanks to our 30+ distinguished speakers, and our supportive partners: Fragile X Association of Australia, Fragile X Association of China, Fragile X Society India, Fragile X Syndrome Parent’s Group Argentina, Fragile X South Africa, and Fragile X Center Georgia.

You are welcome to watch the on-demand video by clicking on the banner image above, or by visiting our WuXi AppTec YouTube Channel. Please follow us on Twitter and LinkedIn to stay up-to-date on upcoming WuXi AppTec news and events.

Below are our past events in the “Collaborations that Transform” series.

2022:
Moving the Needle on ALS & Neuromuscular Disorders

2021:
DMD: Charting the Path for New Therapeutics and Better Care
Fragile X Syndrome: In Pursuit of a Cure
Winning the War on Huntington’s Disease
Progress in Halting Pompe Disease
WuXi Rare Disease Awareness Concert on International Rare Disease Day 2021

2020:
Emerging Opportunities in Multiple Sclerosis and Neuroscience
Spinal Muscular Atrophy (SMA)
Advances in Rare Diseases: Hemophilia
Neurofibromatosis

AGENDA

8:00 AM - 8:05 AM

Welcome

Hui Cai, Program Chair, VP and Head of Content, WuXi AppTec
Katie Clapp, President & Co-Founder, FRAXA Research Foundation

8:05 AM - 8:30 AM

Opening Plenary

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
Session Leader: Michael Tranfaglia, Medical Director & CSO, FRAXA Research Foundation

8:30 AM - 8:50 AM

Innovation Showcase I: Gene Therapy/Gene Reactivation/Protein Replacement

Hosts: Wendy Bruce, Executive Director, Fragile X Association of Australia, and 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
Ernest Pedapati, Associate Professor, Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine
Kathryn Whitehead, Professor, Departments of Chemical Engineering and Biomedical Engineering, Carnegie Mellon University

8:50 AM - 9:30 AM

Innovation Showcase II: Targeted Therapy

Hosts: Mark Bear, Professor of Neuroscience, Picower Institute for Learning and Memory, MIT, and Craig Erickson, Professor of Clinical Psychiatry, Cincinnati Children’s Hospital and the University of Cincinnati College of Medicine.
Daniel Alkon, President, CSO & Scientific Founder, Synaptogenix
John Atack, Director, Medicines Discovery Institute, Cardiff University
Mara Dierssen, Senior Scientist, Center for Genomic Regulation
Lynn Durham, Founder & CEO, STALICLA
Lili Mao, Head of Project Management Office and Director, Clinical Development Unit, 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, COO & Co-Founder, Epigen Biosciences
David Walker, Director of Chemistry, Sentinel Oncology

9:30 AM - 10:00 AM

Innovation Showcase III: Drug Repurposing

Hosts: Bruce Bloom, Chief Collaboration Officer, Healx, and Andres Centellas, Board Member, FRAXA Research Foundation.
Isaac Bentwich, Founder & CEO, Quris
Randall Carpenter, Co-Founder, Allos Pharma
George Drakakis, CEO, Purposeful
Michael Hayden, Founder & CEO, Prilenia Therapeutics; Killam Professor, University of British Columbia
Mahmoud Pouladi Associate Professor, Department of Medical Genetics, Faculty of Medicine, University of British Columbia
Nik Subramanian, CTO & Co-Founder, Kantify
Gordon Wang, Clinical Associate Professor, Department of Psychiatry & Behavioral Sciences; Director, Neuroscience Microscopy Service, Wu Tsai Neurosciences Institute, Stanford University
Xinyu Zhao, Jenni and Kyle Professor, Waisman Center and Department of Neuroscience, University of Wisconsin-Madison

10:00 AM - 10:05 AM

Closing Remarks

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

SPEAKERS

Hui Cai

VP and Head of Content, WuXi AppTec

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

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

Katie Clapp

Co-Founder & President, FRAXA Research Foundation

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.

Elizabeth Berry-Kravis

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

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.

Peter Kind

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

Professor Kind is Director of the Simons Initiative for the Developing Brain at the Patrick Wild Centre for Research into Autism, Fragile X Syndrome (FXS) and Intellectual Disability and Professor of Developmental Neuroscience at the University of Edinburgh. He is also Associate Director at the Centre for Brain Development and Repair (CBDR) at the Institute for Stem Cell Biology and Regenerative Medicine (Instem), Bangalore, India. Professor Kind completed his postdoctoral training with Professor Colin Blakemore at Oxford University and Professor Susan Hockfield at Yale University. Professor Kind received his PhD from Oxford University in 1993. He joined the University of Edinburgh in 2020.

The Kind laboratory examines the cellular dysfunction associated with monogenic forms of moderate to severe neurodevelopmental disorders, with particular focus on Fragile X Syndrome. The laboratory focuses on synaptic function, from the physiological and morphological alterations to the behavioural phenotypes associated with mouse and rat models of these disorders. We also test two further hypotheses: 1) that distinct genetic causes of ID/ASD convergence on common biochemical and cellular pathologies associated that may be amenable to similar therapeutic approaches. 2) that different monogenic forms of ID/ASD can be stratifield have different critical periods for effective therapeutic intervention.

Michael Tranfaglia

Medical Director & CSO, 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.

Wendy Bruce

Executive Director, Fragile X Association of Australia

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.

Jeannie Lee

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

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.

David Hampson

Professor, Department of Pharmaceutical Sciences, University of Toronto

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.

Ralph Laufer

CSO, Lysogene

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.

Ernest Pedapati

Associate Professor, Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine

Dr. Ernie Pedapati, MD, is a psychiatrist and pediatrician who provides clinical care for individuals with Fragile X of all ages. He received his MD from the University of Massachusetts and completed his residency at Cincinnati Children’s (CCHMC) in 2014. Dr. Pedapati remained at CCHMC and is an Associate Professor in the Division of Child Psychiatry and Neurology. His clinical research focus is on understanding brain activity in Fragile X and developing new treatments to enhance cognition. He co-directs the Fragile X gene therapy program at Cincinnati Children’s and is the principal investigator of several investigational drug trials in Fragile X. His laboratory has been supported by the National Institutes of Health since 2017 to study the neurophysiology of Fragile X.

Kathryn Whitehead

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

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.

Mark Bear

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

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.

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

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.

John Atack

Director, Medicines Discovery Institute, Cardiff University

Dr. Atack is a Professor of Translational Drug Discovery at Cardiff University where he is the Director of the Medicines Discovery Institute. Prior to commencing his academic career (originally at the University of Sussex and more recently Cardiff University) he spent 25 years in industry (Merck and then Janssen) as an in vitro and in vivo biologist focusing on neuroscience drug discovery. His team currently consists of around 40 biologists and medicinal chemists work on a number of different early stage preclinical neuroscience and cancer drug discovery projects that over the last 10 years have attracted funding of around £40M for funders such as Cardiff University, the UK Medical Research Council (MRC) and the Wellcome Trust. His interest in fragile X syndrome arose from an improved understanding of the disease mechanisms that highlighted a specific kinase in regulating the structure and function of synapses in preclinical models of fragile X syndrome. In turn, this led to the hypothesis that small molecule inhibitors of a specific kinase might restore the disruption of synaptic function that results as a loss of FMRP; an hypothesis that resulted in the MRC funding a team of biologists, chemists and external collaborators to identify such compounds.

Mara Dierssen

Senior Scientist, Center for Genomic Regulation

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.

Lynn Durham

Founder & CEO, STALICLA

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.

Lili Mao

Head of Project Management Office and Director, Clinical Development Unit, GEXVal

Dr Lili Mao has extensive knowledge and experience in drug discovery for rare diseases. After receiving PhD from University of Medicine and Dentistry of New Jersey, she continued basic research in Rutgers University and University of Minnesota as a postdoctoral fellow. Dr Mao completed her doctoral degree in biochemistry and postdoctoral training in anti-viral drug development, from where she achieved publications of research articles and book chapters including a chapter in Encyclopedia of Biophysics (2013), as well as two patents issued in the U.S. In 2015, she joined Takeda and started her career in extra value generation research unit for early drug discovery. In 2018, she started a new career as an entrepreneur and spun out from Takeda to focus on the rare disease drug discovery. At GEXVal, Dr Mao is intensively engaged in business planning, fundraising, and served as CSO for two terms during startup phase leading the research unit to successfully complete the IND-submission. She is now Director of Clinical Development and Head of Project Management of GEXVal based in Fujisawa, Kanagawa, Japan.

Christopher Missling

President & CEO, Anavex Life Sciences

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.

Adrian Newman-Tancredi

CEO, Neurolixis

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.

Stephen O'Quinn

VP, Medical Affairs, Zynerba Pharmaceuticals

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.

Robert Ring

CEO, Kaerus Bioscience

Dr. Robert Ring is chief executive officer of Kaerus Bioscience, a rare disease biotech developing targeted therapeutics for genetic syndromes of neurodevelopment. A neuroscientist by training, Dr. Ring’s diverse research career in CNS medicines discovery and development has featured leadership positions in industry, governmental agencies, and non-profit research foundations. Prior to his current role at Kaerus, he served Chief Science Officer of Autism Speaks, the global science and advocacy foundation. While at Autism Speaks, he also led the launch and served as founding President of that foundation’s venture arm DELSIA. Prior to Autism Speaks, he served as Head of Autism Research at Pfizer, where he oversaw the first dedicated research unit in large pharma focused exclusively on autism and related neurodevelopmental disorders such as Fragile X syndrome. Prior to Pfizer, Dr Ring served as head of Mood/Anxiety Disorders research at Wyeth in that company’s CNS drug discovery division (Princeton, NJ). He was appointed to the US Department of Health and Human Services’ Interagency Autism Coordinating Committee (IACC) in 2015, where he served for 5 years as a strategic advisor shaping research priorities in autism across the federal agencies. Dr Ring also serves on the scientific advisory board of numerous non-profit organizations in the rare CNS space, including the Pitt Hopkins Research Foundation, the Phelan-McDermid Syndrome Research Foundation, and Global Genes. Dr Ring holds adjunct faculty positions in the department of Psychiatry at the Icahn School of Medicine at Mount Sinai, and Pharmacology & Physiology at Drexel University College of Medicine.

Fabio Tucci

Co-Founder & COO, Epigen Biosciences

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.

David Walker

Director of Chemistry, Sentinel Oncology

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.

Bruce Bloom

Chief Collaboration Officer, Healx

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.

Andres Centellas

Board Member, FRAXA Research Foundation

Isaac Bentwich

Founder & CEO, Quris

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.

Randall Carpenter

Co-Founder, Allos Pharma

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.

George Drakakis

CEO, Purposeful

Dr. Georgios Drakakis is the CEO of the in silico drug repurposing company Purposeful, focused on finding treatments for rare diseases. His research interests include machine learning, data mining, machine vision, as well as chemo- and bioinformatics. After obtaining the EPSRC Doctoral Prize, Dr. Drakakis worked as a research associate for a combined 3.5 years at the National Technical University of Athens and the University of Cambridge. He has recently co-authored 15 research publications in these fields and contributed to 2 book chapters.

Michael Hayden

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

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

Dr. Hayden was named one of the 50 Canadians born in the 20th century who have changed the world. Dr. Hayden is the co-founder of five biotechnology companies including: Prilenia 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.

Mahmoud Pouladi

Associate Professor of Medical Genetics, University of British Columbia

Mahmoud Pouladi is an Associate Professor of Medical Genetics at the University of British Columbia (UBC), Vancouver, Canada. Dr Pouladi obtained a Masters degree in Immunology from McMaster University (Hamilton, Ontario, Canada), and completed his PhD studies in Medical Genetics at UBC. He has been the recipient of a number of awards and distinctions including the Canadian Institutes of Health Research Brain Star Award, and the British Columbia Innovation Council’s Ripples of Hope Award in Biotechnology & Entrepreneurship, and the Michael Smith British Columbia Health Research Scholar Award. Dr. Pouladi’s research aims to understand how discrete genetic factors contribute to the development of neurological disorders. His lab develops isogenic human pluripotent stem cells and transgenic animals to model disease, with the goal of delineating novel approaches to influence outcomes for neurological disorders and in particular Fragile X Syndrome (FXS) and Huntington disease (HD), the most common genetic causes of intellectual disability and dementia, respectively.

Nik Subramanian

Co-Founder & CTO, Kantify

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.

Gordon Wang

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

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 Associate 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.

Xinyu Zhao

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

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.

Richard Soll

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

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

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

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