KEYNOTE SPEAKER
Fyodor Urnov
Professor of Molecular Therapeutics;
Director of Therapeutic R&D, Innovative Genomics Institute,
University of California, Berkeley
Prof. Fyodor Urnov co-developed the toolbox of human genome and epigenome editing, co-named genome editing, and was on the team that advanced all of its first-in-human applications to the clinic. He also led the effort that identified the genome editing target for an approved medicine to treat sickle cell disease and beta-thalassemia.
A key focus of his work is expanding access to CRISPR therapies for genetic disease. As part of that effort Prof. Urnov directs the Danaher-IGI Beacon for CRISPR Cures – a first-in-class academia-industry partnership developing and advancing to the clinic CRISPR-based platform approaches to treat severe Mendelian diseases of the immune system.
In May 2025 a paper in the New England Journal of Medicine described the world’s first on-demand engineered CRISPR therapy for a newborn with a severe metabolic disorder; Urnov was the IGI lead of this multisite collaboration that reduced key components of “CRISPR as a platform” approach to clinical practice.
SESSION SPEAKERS
EDWARD A. FON
An attending neurologist, professor and clinician-scientist listed among the top 1% most cited researchers in the world by Clarivate, Edward Fon is the Director of The Neuro (Montreal Neurological Institute-Hospital) at McGill University.
His research has focused on the molecular mechanisms underlying the neurodegeneration in Parkinson’s disease, with particular emphasis on α-synuclein, GBA1, LRRK2, Parkin, and PINK1—genes implicated in familial forms of the disease. Through the development of patient-derived induced pluripotent stem cell (iPSC) models, his work is paving the way for innovative therapeutic strategies by shedding light on the mechanisms of neurodegeneration.
He is also Director of the McGill Parkinson Program (a Parkinson’s Foundation Centre of Excellence) and Co-Director of the Canadian Open Parkinson Network.
NAVPREET + STALINJEET GILL
Navpreet Singh Gill, DMD, and Stainljeet Gill, DDS, are the parents of Gurmoh Singh Gill, a three-year-old from Surrey BC who was diagnosed with a de novo pathogenic mutation in the SPAST gene: c.1496G>A (p.Arg499His). They founded Hope for SPG4 Hope Foundation with the goal to accelerate research and develop gene therapy that could change the future for their child and others like him.
They are working with Ziv Gan-Or, executive director of the Clinical Research Unit at The Neuro (Montreal Neurological Institute-Hospital) and its Platform for Advanced Clinical Therapies to work with international researchers to bring an advanced therapeutic to trial. The Gills also undertook a Journey of Hope with stops from coast to coast across Canada to collect messages of hope from rare disease organiztions to the federal Minister of Health in Ottawa, raising awareness for the difficulty of accessing Canada’s Rare Disease funding. spg4hope.org.
YONG-HUI JIANG
Yong-Hui Jiang is Dorys McConnell Duberg Professor of Neuroscience and Chief of Medical Genetics at Yale School of Medicine. His clinical expertise focusses on the clinical and biochemical genetics of rare and undiagnosed diseases in children and adult.
Dr. Jiang’s research interests include uncovering the genetic and epigenetic bases of neurodevelopmental disorders or rare diseases with neurodevelopmental defects; modelling genetic diseases using human patients derived cellular models and genetic mutant mice; understanding the circuit and molecular mechanisms underlying autism spectrum disorder; and developing novel molecular and epigenetic targeted therapies for genetic and epigenetic diseases.
He is the Director of Yale NORD Center of Excellence and Principal Investigator of the Yale Diagnostic Center of Excellence for Undiagnosed Diseases-NIH Undiagnosed Disease Network Phase III.
ANDRES LOZANO
Andres Lozano, OC, MD, PhD, FRCSC, FRSC, FCAHS, FCNS is a 1983 graduate of the University of Ottawa Faculty of Medicine. He trained in Neurosurgery at McGill University and became a Fellow of the Royal College of Physicians and Surgeons in Canada in 1990, also earning a PhD in Experimental Medicine in during his residency. He studied stereotactic and functional neurosurgery with Dr. Ronald Tasker, at Toronto Western Hospital, and joined the neurosurgical staff at the University of Toronto in 1991. He holds the distinguished title of University Professor in the Department of Surgery, and holds the Alan & Susan Hudson Cornerstone Chair in Neurosurgery at University Health Network. He is the past Chairman of Neurosurgery at the University of Toronto (2010-2020) and Past President of the American Society for Stereotactic and Functional Neurosurgery (ASSFN) and the World Society for Stereotactic and Functional Neurosurgery (WSSFN). Since 2020 he has served as the fourth Editor-in-Chief of the journal “Stereotactic and Functional Neurosurgery”. Dr. Lozano has served as a founding member of several research organizations, including the Michael J. Fox Foundation. He has been designated a highly cited scientist yearly for over 10 years (Clarivate). His awards include the Olivecrona Medal, Salk Award, Dandy Medal and Segerfalk Award, and he has been elected to the Royal Society of Canada, Order of Spain and an Officer of the Order of Canada.
MARYAM OSKOUI
Dr. Maryam Oskoui is a pediatric neurologist and Full Professor in the Departments of Pediatrics and Neurology and Neurosurgery. She is a recipient of a Distinguished Research Scholar award from the FRQ-S. Dr Oskoui is a member of the Scientific Advisory Committee for Muscular Dystrophy Canada, and is an investigator in several clinical trials in pediatric neuromuscular disorders. She is a long-standing methodologist for the American Academy of Neurology Guideline Development Committee, advancing evidence-based approaches to improve the diagnosis, prognosis, and treatment of neurological disorders worldwide.
Dr.Oskoui’s research integrates population-based epidemiology, clinical registries, patient partnership and clinical trials to better understand disease trajectories, evaluate innovative therapies, and improve outcomes for children with neuromotor disorders. Her work spans the continuum from evidence generation to implementation, with a focus on ensuring that therapeutic advances translate into meaningful improvements in clinical care.
MASSIMO PANDOLFO
Massimo Pandolfo is the Medical Director of the Clinical Research Unit at The Neuro (Montreal Neurological Institute-Hospital) at McGill University. Prior to coming to McGill, Dr. Pandolfo was Chief of Neurology at Erasme Hospital and Professor of Neurology at the Université Libre de Bruxelles (ULB), where he was also Director of the Laboratory of Experimental Neurology.
His research interests focus on neurogenetics. In 1996, an international collaboration led by Dr. Pandolfo was the first to identify the Friedreich’s ataxia (FRDA) gene.
He has since contributed to the study of the molecular pathogenesis of the disease, to the development of therapeutics, and to its clinical characterization. He has been the coordinator of EFACTS (European Friedreich’s Ataxia Consortium for Translational Studies) and is currently on the Board of Directors of the Ataxia Charlevoix-Saguenay Foundation.
In addition, he has contributed to the study of several monogenic epilepsies and participated in genetic studies on common epilepsies and on epilepsy pharmacogenetics.
JAGDEEP WALIA
Dr. Jagdeep Walia is a medical geneticist and full-time professor in the Division of Medical Genetics, Department of Pediatrics. A graduate of the Guru Nanak Dev University School of Medicine, he joined Queen’s University in 2012. In his clinical work, Dr. Walia consults across a broad range of genetic issues that affect children and adults, including cancer, prenatal diagnosis, metabolics and general genetics. He teaches genetics at the undergraduate and postgraduate levels.
Dr. Walia launched a clinical and basic genetics research program soon after joining Queen’s. His lab focuses on developing novel gene therapy approaches for inherited and acquired neurodegenerative disorders. His research has yielded very encouraging results in a mouse model of GM2 gangliosidoses—a group of three related genetic disorders (Tay-Sachs disease, Sandhoff disease and AB variant) that cause progressive deterioration of nerve cells and death—demonstrating an almost three-fold increase in survival. The translational nature of this work is exemplified by the initiation of a phase 1/2 clinical trial for infantile GM2 gangliosidoses at Queen’s University/Kingston Health Sciences Centre using adeno-associated virus vector (AAV) as a tool for gene transfer to the central and peripheral nervous system. Dr. Walia is also working on the development of gene therapies for other genetic conditions. His other research interest encompasses genetic, epigenetic and metabolic changes in autism spectrum disorder.
MODERATORS
ZIV GAN-OR
At The Neuro (Montreal Neurological Institute-Hospital), Ziv Gan-Or is the executive director of the Clinical Research Unit and director of the Neurogenomics and Precision Medicine lab. His team works to identify targets for drug development in neurodegenerative disorders using genetic methods; to understand how genetics affect disease risk, progression and response to medications; and to learn how to apply genomics to improve clinical trials by targeting specific genetic subpopulations of patients.
His work on the GBA1 gene in Parkinson’s disease (PD) helped advance the first ever phase 2 trial on a genetic subtype of PD. Other genes, that Dr. Gan-Or’s research identified as important in PD, including SMPD1, TMEM175, GALC, are currently being developed as therapeutic targets. He is also the Director of the GBA1-Canada Program, the lead of the International REM-sleep Behavior Disorder Genomics Consortium, the lead of the Clinical Genetics and Gene Discovery team of the Canadian Consortium for Neurodegeneration in Aging (CCNA), among other initiatives.
SIMON THEBAULT
Key research areas include validating the clinical utility of emerging serum biomarkers such as neurofilament light chain and glial fibrillary acidic protein, characterizing cerebrospinal fluid protein and immune cell profiles as indicators of CNS-compartmentalized inflammation, and investigating the role of Epstein-Barr virus in priming disease activity in MS, both as a potential biomarker and a therapeutic target.
YANG ZHOU
Yang Zhou is a researcher at The Neuro (Montreal Neurological Institute-Hospital). His lab aims to deepen the understanding of autism and severe forms of Neurodevelopmental Disorders (NDDs), including intellectual disabilities and epilepsy. Learning from clinical and genetic discoveries, his group applies experimental models to characterize gene mutations associated with NDDs.
Through extensive collaboration, his group carries out research utilizing molecular, genetic, genomic, and neurobiological approaches. They work on (1) uncovering the function of risk genes in developmental and adult brains, (2) identifying novel mechanisms/pathophysiology underlying NDDs, and (3) correcting mutated genes and examining the reversibility of impaired neural and behavioral functions. The ultimate goal of his group is to provide findings and knowledge that may accelerate the development of therapies for affected individuals.