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Orrin Devinsky MD

Neurosurgeon, NYU Langone, Chief of Service, NYU Epilepsy
Dr. Orrin Devinsky is a leading neurologist and neurosurgeon at NYU Langone and professor of neurology, neurosurgery, and psychiatry at the New York University School of Medicine. His epilepsy research includes sudden unexpected death in epilepsy (SUDEP), cannabinoids, phenome-genome correlations, autism, neural markers and imaging, therapeutic electrical stimulation, quality-of-life, cognitive and behavioral issues, and surgical therapy.

Devinsky received his medical degree from Harvard Medical School, founded Finding A Cure for Epilepsy and Seizures (FACES) and co-founded epilepsy.com. Devinsky was a lead neurologist in CBD clinical trials for the treatment of epilepsy.

 
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Robin Kleiman, Ph.D

Senior Director, Biogen Research Associate, Boston Children’s Hospital
Dr. Kleiman has contributed to the discovery and development of multiple novel clinical candidates and has worked at companies that span the big pharma, start-up and biotech industries. She was one of several founding members of the Pfizer Neuroscience Phosphodiesterase platform team that produced multiple clinical candidate molecules, 3 first-in-human clinical trials for distinct and completely novel mechanisms followed by 2 proof-of-concept clinical trials.

Kleiman joined the Translational Neuroscience Center leadership team and faculty at Boston’s Children’s Hospital in August of 2013 as the Head of Preclinical Research to provide drug discovery expertise to the members of the TNC community, develop a robust portfolio of translational research projects and foster more in-depth collaboration with industry and disease-based foundations to develop novel therapies to improve the lives of pediatric and adolescent patients.

 
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Kiran Reddy, MD

CEO, Praxis Precision Medicines, Former Partner, Clarus Ventures
Dr. Reddy is President & CEO of Praxis Precision Medicines, a Cambridge, Massachusetts based biotechnology company operating in stealth mode. Formerly, he was a Venture Partner at Clarus where he focused on new company formation and at Biogen as part of the Corporate Strategy leadership team.

Reddy was part of the founding team and interim Chief Business Officer for SAGE Therapeutics through its IPO, and co-inventor of SAGE-547 the Phase 3 program for the rare epilepsy disorder refractory status epilepticus. He was part of the team that launched Foundation Medicine, and he has served as a Board Observer for Alnara pharmaceuticals, Rhythm pharmaceuticals, and PanOptica pharmaceuticals. Reddy holds MD and MBA degrees from Georgetown University, and is a board certified neurologist; he has authored several peer-reviewed scientific papers in the field of neuroimmunology and neurodegenerative diseases.

 
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Aris Economides, PhD

Vice President of Research, Regeneron Pharmaceuticals
Dr Economides leads Genetic Research at Regeneron which includes Genome Engineering Technologies and Skeletal Diseases TFA. He is a co-inventor of the Cytokine Trap technology that led to the development of the IL-1 trap, a currently approved biologic drug (ARCALYST™). He is also a co-inventor of the VelociGene® technology, that has led to the development of VelocImmune®, a method for the generation of all-human antibodies in mice.

More recently, Economides has been spearheading the development of new methods for the generation of transgenic mice using BAC as transgene vectors, and has also pioneered a new method for generating conditional alleles.

 
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Steven Gray, PhD

Scientist, Gene Therapy Center at UNC School of Medicine
Dr. Gray is a leading expert in gene therapy for brain related diseases. His research focuses on developing virus systems that can be implanted with the corrected genes of various genetic disorders and introduced to the nervous system to repair mutated genes.

Gray has also begun preclinical animal model studies using these virus systems to treat neurological diseases, and is currently involved in the first human trial with AveXis Biotech to cure the MECP2 mutation (RETT’s Syndrome).

 
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Ingrid Scheffer, PhD

Pediatric Neurologist, Physician Scientist, University of Melbourne & Florey Institute
Dr. Scheffer is Chair of Paediatric Neurology Research at The University of Melbourne and Senior Principal Research Fellow at the Florey Institute of Neuroscience and Mental Health. Professor Scheffer is a founding fellow of the Australian Academy of Health and Medical Sciences and currently its Vice-President. Scheffer is credited with finding the first gene implicated in epilepsy. She has also described and classified novel epileptic syndromes such as Epilepsy limited to Females with Mental Retardation.

Scheffer has worked to characterise new epilepsy syndromes, from infancy to adulthood, which have permitted appropriate treatment and diagnosis, such as Dravet Syndrome. She has considerably expanded our understanding of the spectrum of epilepsies associated with glucose transporter deficiency; this body of work carries major treatment implications as this disorder responds to the ketogenic diet.

 
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Elli Brimble, MSc, MS, CGC

Genetic Counselor, Lucile Packard Children’s Hospital at Stanford
Brimble serves as FOXG1 Research’s Scientific Advisory Board Liaison to facilitate communication with research communities. She is a genetic counselor in Child Neurology at Stanford Children’s Health, where she is working to develop a FOXG1 multidisciplinary clinic. Brimble completed her undergraduate training in genetics at Western University in Ontario, and Masters degrees in molecular genetics and genetic counseling at the University of Toronto and Boston University, respectively.

Brimble’s primary clinical and research interests are to improve understanding of genetic mechanisms that contribute to epilepsy, and to develop protocols that ensure comprehensive and consistent evaluations for genetic epileptic encephalopathies.

 

Neil Kumar, PhD

CEO, BridgeBio Pharma, Former Principal, Third Rock Ventures
Dr Kumar is CEO of biotech company BridgeBio, focused on the development of new therapies for rare genetic disorders. Previously, he was a principal at Third Rock Ventures, where he supported and managed various biotech portfolio companies, in addition to focusing on new company formation and due diligence. He also held the role of Vice President, Business Development and Operations for MyoKardia.

Kumar is the author of several peer-reviewed papers in the fields of oncology and systems biology. He holds BS and MS degrees in chemical engineering from Stanford University, and his PhD in chemical engineering from the Massachusetts Institute of Technology.

 
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Xilma Ortiz-Gonzalez, MD, PhD

Pediatric Neurologist, CHOP, Neurology Instructor, University of Pennsylvania
Dr. Ortiz-Gonzalez is a pediatric neurologist at Children’s Hospital of Philadelphia (CHOP) where she sees patients with FOXG1 Syndrome as well as those with numerous other genetic mutations.

Ortiz-Gonzalez’s research focus is on mitochondrial disorders often present in childhood with systemic disease, including cardiac, skeletal muscle, visual and neurologic symptoms. She is interested in using iPSC technology to generate in vitro disease models to understand cardiac and neural phenotypes from patients affected with mitochondrial diseases and has published numerous articles on the topic.

 
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Mary Jones, MD

Pediatrician, Head, Katie's RETT Clinic at UCSF Oakland
Dr. Mary Jones spent 31 years as a pediatrician as part of the East Bay Pediatrics family in California. When one of her patient’s - Katie - was diagnosed with RETT Syndrome, she became dedicated to understanding the disease in order to provide the specialized care needed. She decided to leave general pediatric practice and establish Katie's Clinic for Rett Syndrome at Children's Hospital and Research Center Oakland. Katie's Clinic offers both clinical and emotional support for individuals with Rett Syndrome and their families.

Dr Jones’ Katie’s Clinic is part of the NIH funded Natural History Study for RETT, CDLK5 and FOXG1. She is zealously working to extend clinics such as hers and create greater focus on the study of rare diseases.

 
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Gordon Fishell, PhD

Scientist, Broad Institute; Professor, Harvard Medical School
Dr. Fishell received his PhD in neurobiology from the University of Toronto-Neurobiology Research Group, and works on the study of inhibitory interneurons in the brain and other deep, foundational brain sciences.

Fishell recently joined the Broad Institute, where the CRISPR cut-and-paste gene editing method was discovered, and is Professor at Harvard Medical School. He was formerly a Julius Raynes Professor of Neuroscience and Associate Director of the NYU Neuroscience Institute. Fishell’s research interests are focused on the Molecular Genetics of Regional Patterning in the Mammalian Telencephalon, and Molecular Control of Neural Stem Cell Proliferation and Specification. He has published over 100 original papers, and five books.

 
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Heather Olson, MD, MS

Neurologist, Boston Children's; Neurology Instructor, Harvard Medical
Dr. Olson completed all of her post-graduate medical training in Boston including training in Pediatric Neurology, Epilepsy and Clinical Neurophysiology and Epilepsy Genetics. Since joining the faculty at Boston Children’s Hospital in 2013, she works as a clinician and clinical researcher with a focus on Neurogenetic disorders and Epilepsy.

Olsen has recently opened a FOXG1 clinic at Children’s Hospital Boston, and is assisting with the creation of a sister clinic at Children’s Hospital Stanford. She is also director of the CDKL5 Center of Excellence, a combined clinical and research program.

 
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Hourinaz Behesti, PhD

Developmental Neurobiologist, Rockefeller University
Dr. Behesti obtained her PhD in developmental biology from University College London (UCL), studying the genetic control of patterning and growth during eye development. Later, she carried out postdoctoral research at the Rockefeller University (New York), studying the cellular and molecular functions of an autism-risk gene in the cerebellum. As a Developmental Neurobiologist, she is interested in the molecular control of early brain development and function and her research is currently focused on derivation of human cerebellar neuronal subtypes for the purpose of modeling human brain development and disease.

 
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Toni Cathomen, PhD

Director, Institute for Cell and Gene Therapy, University of Freiburg
Dr. Cathomen is a molecular biologist. Since 2013 he has been Director of the Institute for Transfusion Medicine and Gene Therapy at the University Hospital Freiburg. He is researching the possibilities of gene therapy and genome editing with so-called gene scissors, such as TALEN and CRISPR-Cas.

Cathomen received his doctorate in Zurich from Roberto Cattaneo on the topic of Functional Analysis of Measles Virus Envelope Assembly. Subsequently, he was a postdoc with Matthew D. Weitzman at the Salk Institute . From 2003 to 2009, Cathomen was a junior professor at the Berlin Charité and from 2009 to 2012 Professor of Gene Modification of somatic cells at the Hannover Medical School . Since 2012 he has been researching and teaching as a professor at the Albert-Ludwigs-Universität Freiburg.

 
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Ritu Lal, PhD

Clinical Pharmacology Consultant
Dr. Lal has over 20 years of experience as a senior clinical pharmacologist within the biotechnology and pharmaceutical space. She has led Preclinical and Clinical Development teams at companies like Hoffman la Roche, Abbott Labs and XenoPort, taken compounds from Discovery to Regulatory Approval and filed more than 15 Investigational New Drug Applications (IND) in disease areas of neurology, inflammation, virology, pain and oncology. Two compounds have received New Drug Application (NDA) approvals from the US FDA and Ministry of Health and Welfare, Japan (Kaletra for the treatment of HIV and Horizant (Regnite in Japan) for Restless Legs Syndrome (RLS) and Post Herpetic Neuralgia (PHN)).

Lal has a doctorate in Pharmaceutical Sciences and Pharmacology from the University of Maryland and a Masters degree in Pharmacology from University of Arizona. She has over 60 publications, book chapters and presentations and recently received a Master’s in Management from Stanford University’s MSx (Sloan) Program.

 
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MARK PYKETT, PhD

Chief Innovation Officer, PTC Therapeutics, former CEO, Agilis Biotherapeutics
Dr Pykett brings more than 20 years of scientific, clinical, operational, and commercial experience, and proven leadership in the biotechnology, pharmaceutical and healthcare industries. He recently joined PTC Therapeutics through their acquisition of Agilis Biotherapeutics where he served as CEO, advancing an innovative gene therapy platform for rare monogenic diseases that affect the central nervous system.

Prior to joining Agilis, Dr. Pykett served as President and CEO of Navidea Biopharmaceuticals where he catalyzed the Company’s transformation into an innovative precision medicine company, drove the development pipeline to in-license two Phase 3 assets in Alzheimer’s and Parkinson’s disease, led the company to a successful FDA approval, commercialized its first oncology product. Pykett received his B.A. degree from Amherst College, a Ph.D. and V.M.D. from the University of Pennsylvania, and an M.B.A. from Northeastern University, and completed post-doctorate fellowships at the University of Pennsylvania and Harvard University.


 
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Dr Nadia Bahi-Buisson, MD, PhD

Pediatric Neurology, Necker Enfants Malades University Hospital, Paris
Dr Bahi-Buisson is a pediatric neurologist and professor at Necker Enfants Malades University Hospital in Paris, where she conducts the rare disease program on Rett syndrome and brain malformation. She is also part of the group "Genetics and Cerebral cortex development" (A Pierani) of the Imagine Institute at Paris Descartes University.

Bahi-Buisson is focused on understanding the cellular and molecular mechanisms of brain development, especially neurogenesis, and neuronal migration. Her main goal is to understand the molecular basis of Malformations of Cortical Development which are important causes of intellectual disability and account for 20-40% of drug-resistant childhood epilepsy. Her group conducts integrated multidisciplinary studies involving cellular and molecular biologists, foetal pathologists and radiologists. They use a wide range of approaches including genetics, biochemistry and cellular biology to better understand the signalling pathways involved in these processes and how their dysregulations can lead to pathologies.

Bahi-Buisson holds a doctorate from Paris V University.


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