Please join us virtually for the inaugural St. Jude Pediatric Translational Neuroscience Symposium.
Recent years have seen the research and care of rare neurological diseases evolve from diagnosis and comfort care into treatment and hope, driven by advancements in biological understanding of disease and therapeutic technologies. The St. Jude Pediatric Translational Neuroscience Initiative (PTNI) is uniquely positioned to translate this knowledge into impactful clinical outcomes for children with these conditions.
The 2025 St. Jude Pediatric Translational Neuroscience Symposium, hosted by PTNI April 8-9, 2025, will bring together leading experts in pediatric neuroscience to discuss topics critical to accelerating advancement in the field. This inaugural Symposium will tackle critical issues including gene discovery, movement from identification of genetic variants to development of therapeutic candidates, and considerations for gene therapy and translational research. Speakers and attendees, including researchers, clinicians, patient advocates, and industry partners, will come together to learn about, share, and guide advances in addressing rare pediatric neurological diseases.
The 2025 St. Jude Pediatric Translational Neuroscience Symposium is presented by the St. Jude Biomedical Research Symposium, an annual series typically exclusive to the St. Jude research community. This year we are happy to give the public an opportunity to participate virtually. We look forward to robust engagement with attendees from across the rare pediatric neurological disease community.
2025 Keynote Speaker
Huda Zoghbi, MD, is a Distinguished Service Professor at Baylor College of Medicine, a Howard Hughes Medical Institute (HHMI) Investigator, and founding Director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital.
Dr. Zoghbi graduated from the American University of Beirut and received her medical degree from Meharry Medical College. She completed pediatrics and neurology residencies and postdoctoral research training in molecular genetics at Baylor College of Medicine.
Dr. Zoghbi’s expertise ranges from neurodevelopment to neurodegeneration. She and Dr. Harry Orr discovered that spinocerebellar ataxia type 1 (SCA1) is caused by expansion of a polyglutamine tract. Their mechanistic studies on SCA1 inspired her work on common neurodegenerative diseases such as Alzheimer’s disease and Parkinson’s disease. She discovered that mutations in MECP2 cause Rett syndrome, characterized the molecular and circuit pathogenic mechanisms in Rett, and highlighted the importance of MeCP2 levels for normal brain function. Her group also discovered Math1/Atoh1 and showed that it governs the development of several components of the proprioceptive, balance, hearing, vestibular, and breathing pathways.
Dr. Zoghbi has trained more than 115 scientists and physician-scientists and has served on the National Academy of Medicine (NAM) Council, the National Institutes of Health (NIH) National Advisory Neurological Disorders and Stroke (NINDS) Council, and numerous scientific advisory boards. She has been elected to the National Academy of Medicine, the National Academy of Sciences, and the American Academy of Arts and Sciences.
Dr. Zoghbi’s honors include the Breakthrough Prize in Life Sciences, the National Academy of Sciences Jessie Stevenson Kovalenko Medal, the Lundbeck Foundation’s Brain Prize, and the Kavli Foundation’s Kavli Prize.
Agenda
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Time Session 9:00 – 9:15 am Opening remarks
J. Paul Taylor, MD, PhD9:15 – 10:45 am Session I: Gene Discovery – What’s Next?
Session Chair: Peter J. McKinnon, PhD- Wendy Chung, MD, PhD
- Heather C. Mefford, MD, PhD
- Francesco Muntoni, MD
10:45 – 11:00 am Break 11:00 am – 12:30 pm Session II: From Genetic Variant to Therapeutic Candidates
Session Chair: Richard Finkel, MD- Kevin M. Flanigan, MD
- Charlotte Sumner, MD
- C. Frank Bennett, PhD
12:30 – 1:15 pm Break 1:15 – 2:15 pm Keynote speaker – Huda Zoghbi, MD
Introduction: J. Paul Taylor, MD, PhD2:15 – 3:45 pm Session III: From Lead Candidate to Dosing the Patient
Session Chair: Elizabeth (Beth) Fox, MD, MS- Mustafa Sahin, MD, PhD
- Paul Tesar, PhD
- Andrea Gropman, MD
3:45 – 4:00 pm Break 4:00 – 5:30 pm Session IV: Pre and Post Gene Therapy Considerations
Session Chair: Mitchell J. Weiss, MD, PhD- Carsten G. Bönnemann, MD
- Florian Eichler, MD
- Katherine (Kathy) High, MD
5:30 pm Closing remarks
J. Paul Taylor, MD, PhD -
Time Session 9:00 – 9:10 am Opening remarks
J. Paul Taylor, MD, PhD9:10 – 10:40 am Session V: Unique Considerations in Translational Research for Ultrarare Diseases
Session chair: Richard Finkel, MD- James Dowling, MD, PhD
- James Wheless, MD
- Shengdar Tsai, PhD
10:40 – 10:50 am Break 10:50 am – 12:20 pm Session VI: Beyond the Science: Ethical, Economic, and Equitable Access Considerations
Session chair: Kristin Stephenson, MHA, JD- Andrew Lo, PhD
- Elizabeth Phimister, PhD
- Philip J. (P.J.) Brooks, PhD
12:20–12:30 pm Closing remarks
J. Paul Taylor, MD, PhD
Speakers
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C. Frank Bennett, PhD
Frank Bennett, PhD, is Executive Vice President, Chief Scientific Officer, and a founding member of Ionis Pharmaceuticals. He oversees the discovery and development of antisense drugs and Ionis’ leadership in genetic medicines, including gene-editing technologies. For 35 years, Dr. Bennett has been involved in advancement of antisense technology as therapeutic agents across multiple disciplines and has led projects across all phases of drug discovery and development. His work in antisense technology for neurological diseases has been foundational, leading to the development of tofersen (QALSODY) for ALS and nusinersen (SPINRAZA) for spinal muscular atrophy. Over 20 different antisense drugs, many based on his research and discovered and developed at Ionis under his leadership, have entered the clinic for the treatment of neurodegenerative, neurological diseases and neurodevelopmental disorders. Dr. Bennett is a co-recipient of the 2019 Breakthrough Prize in Life Sciences and the 2021 Gabbay Award for his contributions to discovery and development of SPINRAZA, the 2018 Hereditary Disease Foundation's Leslie Gehry Brenner Prize for Innovation in Science for his leadership in developing antisense therapies for Huntington's disease, and the 2023 Rainwater Prize for Outstanding Innovation in Neurodegenerative Research. He holds a pharmacy degree from the University of New Mexico and a PhD in pharmacology from Baylor College of Medicine.
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Carsten G. Bönnemann, MD
Carsten Bönnemann, MD, graduated from University of Freiburg in Germany and trained in pediatrics in Hamburg and Göttingen and in neurology at Massachusetts General Hospital/Harvard Medical School (HMS) in Boston. He completed postdoctoral research in genetics and neuromuscular specialty training at Boston Children’s Hospital/HMS. 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 as Adjunct Full Professor of Neurology. In 2010, he joined the NIH as Senior Investigator and Chief of the Neuromuscular and Neurogenetic Disorders of Childhood Section in the Neurogenetics Branch of NINDS. His work focuses on early-onset neuromuscular disorders, such as congenital myopathies and muscular dystrophies, focusing on development of molecular and gene-directed treatments, including first-in-human intrathecal and intravenous AAV-mediated gene transfer trials. Dr. Bönnemann has received numerous awards, including the Pew Biomedical Sciences Fellowship, the 2010 Derek Denny-Brown Neurological Scholar Award, the 2023 Jacoby Award from the American Neurological Association, the 2022 Legacy Award of the Muscular Dystrophy Association USA, and the 2023 International Duchenne-Erb Price of the German DGM. He has been elected to the American Association of Physicians and is Co-Editor-in-Chief of The Journal of Neuromuscular Diseases.
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Philip J. Brooks, PhD
Philip J. (P.J.) Brooks, PhD, is the acting director of the National Institutes of Health (NIH)/National Center for Advancing Translational Sciences (NCATS) Division of Rare Diseases Research Innovation. Dr. Brooks represents NCATS in the NIH-wide Gene Therapy Working Group, the Regenerative Medicine Innovation Project, and the International Rare Diseases Research Consortium. He also is the working group co-coordinator for the NIH Common Fund’s Somatic Cell Genome Editing program, one of the leaders of the Platform Vector Gene Therapy pilot project, and the co-chair of the Bespoke Gene Therapy Consortium. Dr. Brooks was the recipient of the 2022 Sonia Skarlatos Public Service Award from the American Society of Gene & Cell Therapy for consistently fostering and enhancing the field of gene and cell therapy.
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Wendy K. Chung, MD, PhD
Wendy Chung, MD, PhD, is a clinical and molecular geneticist and Chief of the Department of Pediatrics at Boston Children’s Hospital and the Mary Ellen Avery Professor of Pediatrics at Harvard Medical School. She leads NIH-funded research in human genetics of pulmonary hypertension, breast cancer, obesity, diabetes, autism, birth defects including congenital diaphragmatic hernia, and congenital heart disease. She has advanced newborn screening for spinal muscular atrophy, Duchenne muscular dystrophy, and GUARDIAN (Genomic Uniform screening Against Rare Diseases In All Newborns). Dr. Chung is a national leader in the ethical, legal, and social implications of genomics. She was the recipient of the Rare Impact Award from the National Organization of Rare Disorders and is a member of the National Academy of Medicine and the American Academy of Physicians. Dr. Chung received her BA in biochemistry from Cornell University, her MD from Cornell University Medical College, and her PhD from The Rockefeller University in genetics.
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James Dowling, MD, PhD
Dr. Jim Dowling, MD, PhD, is a staff clinician and senior scientist at the Hospital for Sick Children, a Professor at the University of Toronto, and the Mogford Campbell Family Chair of Paediatric Neuroscience and will soon begin a new role as Director of the Penn Neurogenetic Therapy Center and the Van Meter Professor of Neurology at the University of Pennsylvania (UPenn). Dr. Dowling holds a BSc and MSc from Yale University and an MD/PhD from the University of Chicago. He completed his residency in child neurology at Children’s Hospital of Philadelphia and postdoctoral research at UPenn and the University of Michigan. Dr. Dowling’s clinical expertise is in childhood neuromuscular disorders; his research spans pre-clinical and clinical translational medicine and examines disease pathomechanisms and therapy development for early onset neurogenetic conditions. He has authored/coauthored more than 200 papers and received funding from multiple sources, including the NIH. Dr. Dowling serves on several scientific and medical advisory boards, is the neurogenetics lead of the Ontario Provincial Genetics Program, and was recently honored with the Researcher of the Year Award from Muscular Dystrophy Canada.
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Florian Eichler, MD
Florian Eichler, MD, is a Professor of Neurology at Massachusetts General Hospital (MGH) and Harvard Medical School, holding the Katherine B. Sims Chair in Neurogenetics. At MGH he is Co-Director of the Precision Therapeutic Unit in the Center for Genomic Medicine and Director of the Leukodystrophy Service and the Center for Rare Neurological Diseases (CRND), where he focuses on advancing care and treatment for devastating neurogenetic conditions. After completing a neurogenetics fellowship at Johns Hopkins and residency training at MGH, Dr. Eichler established a laboratory exploring the mutant genes, biochemical defects, and neurodegeneration. His research identified neurotoxic desoxysphingoid bases in hereditary sensory neuropathy type 1 (HSAN1), leading to the first clinical trial of substrate supplementation therapy. He is co-PI of the gene therapy trial for adrenoleukodystrophy (ALD), which received FDA approval in 2022. He also serves as co-PI of the Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN) and is president of ALD Connect, a patient-powered research network. He leads several gene therapy trials at MGH for diseases including Canavan, Tay Sachs and Sandhoff, and Alexander.
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Richard S. Finkel, MD
Richard S. Finkel, MD, pediatric neurologist, directs the Center for Experimental Neurotherapeutics within the Pediatric Translational Neuroscience Initiative (PTNI) and holds the George J. Pedersen Endowed Chair in Neurotherapeutics at St. Jude Children’s Research Hospital. Clinical practice and research interests have focused upon neuromuscular disorders, especially spinal muscular atrophy, Duchenne muscular dystrophy, and Charcot-Marie-Tooth. Dr. Finkel has participated in numerous clinical trials and natural history studies and has contributed to the development of standard-of-care guidelines, outcome measures, and clinical trial design. He has published more than 200 manuscripts. Dr. Finkel received the Sidney Carter Award in Child Neurology from the American Academy of Neurology (2018) and shared the International Prize for Translational Neuroscience form the Max Plank Institute (2019).
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Kevin M. Flanigan, MD
Kevin Flanigan, MD, is the Director of the Center for Gene Therapy at the Abigail Wexner Research Institute of Nationwide Children’s Hospital (NCH), where he holds the Robert F. & Edgar T. Wolfe Foundation Endowed Chair in Neuromuscular Research and is a Professor of Pediatrics and Neurology at the Ohio State University. He is also the director of the NCH P50-funded Wellstone Muscular Dystrophy Specialized Research Center. Dr. Flanigan’s laboratory work is directed toward the molecular characterization and therapy of neuromuscular diseases using both gene replacement and RNA-modifying therapies and toward the identification of genetic modifiers of disease severity in the dystrophinopathies. He is an experienced clinical trialist and has conducted multiple clinical trials of gene-modifying gene transfer therapies in Duchenne muscular dystrophy (DMD) as well as the childhood neurodegenerative disorders mucopolysaccharidosis types 3A and 3B.
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Elizabeth (Beth) Fox, MD, MS
Beth Fox, MD, MS, is Senior Vice President, Clinical Trials Admininistration Comprehensive Cancer Center, Associate Director of Clinical Research at St. Jude Children’s Research Hospital; Dr. Fox leads regulatory and clinical trial operations as well as clinical research prioritization and strategy at St. Jude Children’s Research Hospital. Nationally, she is the vice-chair of Developmental Therapeutics for the Children’s Oncology Group and the Pediatric Early Phase Clinical Trials Network. Dr. Fox has expertise in quantitative clinical and pre-clinical pharmacology, pediatric clinical oncology, and clinical research and trial design including response and toxicity biomarker endpoint development. She applies an integrative approach to clinical drug development, utilizing animal models and pre-clinical and clinical pharmacology to evaluate new agents and novel trial designs and improve outcome for children with catastrophic diseases.
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Dr. Andrea Gropman, MD
Dr. Andrea Gropman, MD, is Director of Neurometabolic Translational Research in the Pediatric Translational Neuroscience Initiative (PTNI) and the Mark F. Tamer Endowed Chair in Pediatric Neurology at St. Jude Children’s Research Hospital. A neurogeneticist with over 20 years of experience in rare neurogenetic and neurometabolic conditions, Dr. Gropman specializes in pediatrics, child neurology, genetics, and neurodevelopmental disabilities. Her work integrates neurology, metabolism, and developmental pediatrics to identify biomarkers and therapeutics for rare disorders, utilizing advanced imaging technologies. Dr. Gropman holds a BA in biology and biochemistry from Brandeis University and earned her MD from the University of Massachusetts Chan Medical School. She completed pediatric residency at Johns Hopkins Hospital, a neurology fellowship at Children’s National Hospital, fellowships in clinical and biochemical genetics, and postdoctoral research at the National Human Genome Research Institute. In September 2024, she joined St. Jude, where she is establishing a neurometabolic, urea cycle, and mitochondrial research program and clinics for Mendelian disorders linked to neurodevelopmental disabilities, including Smith-Magenis syndrome and X, Y chromosome aneuploidies. As principal investigator of the Urea Cycle Disorders Consortium, Dr. Gropman led a successful NIH-funded multicenter project, bringing two new treatments to market. Her research focuses on neurometabolic conditions and neuroimaging to advance diagnostics and biomarker development for clinical trials.
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Katherine High, MD
Katherine (Kathy) High, MD, has more than 35 years of experience in internal medicine, hematology, molecular genetics, and gene therapy. Her pioneering gene therapy investigations for hemophilia led to studies on the human immune response to AAV gene delivery vectors. A long-time faculty member at the Perelman School of Medicine of the University of Pennsylvania, Dr. High was also an HHMI Investigator at the Children's Hospital of Philadelphia. She served on the U.S. FDA Advisory Committee on Cell, Tissue and Gene Therapies and is a past president of the American Society of Gene & Cell Therapy. In 2013, she co-founded Spark Therapeutics and led the team that achieved the first FDA approval of a gene therapy for genetic disease, Luxturna, for a rare form of inherited blindness. She also led the successful phase 1/2 studies for Beqvez, a gene therapy for hemophilia B. Spark was acquired by Roche in 2019. In 2021 she joined AskBio, as President of Therapeutics and member of the Board of Directors, before becoming a Visiting Professor at Rockefeller University in 2022. She received a bachelor’s degree in chemistry from Harvard University, MD from the University of North Carolina (UNC) School of Medicine, business certification from the UNC Business School’s Management Institute for Hospital Administrators, and master’s degree from the University of Pennsylvania. She is an elected member of several prestigious academies and serves on multiple boards.
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Andrew W. Lo, PhD
Andrew W. Lo, PhD, is the Charles E. and Susan T. Harris Professor at the Massachusetts Institute of Technology (MIT) Sloan School of Management, director of MIT’s Laboratory for Financial Engineering, and principal investigator at MIT's Computer Science and Artificial Intelligence Laboratory. His healthcare-related research interests include new financial engineering tools and business models for drug and device development and healthcare delivery, especially for rare and ultra-rare diseases; statistical methods for incorporating patient preferences into the drug approval process; predicting clinical trial outcomes via machine learning techniques; and novel reimbursement models for creating a robust gene and cell therapy ecosystem. He is a co-founder and director of BridgeBio Pharma, a director of AbCellera, Atomwise, n-Lorem, and Vesalius; a co-founder and chairman of QLS Advisors; and a member of the advisory board to the American Cancer Society’s BrightEdge Impact Fund. Dr. Lo received his BA in economics from Yale University and his AM and PhD in economics from Harvard University.
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Peter McKinnon, PhD
Peter McKinnon, PhD, is the Director of the Center for Pediatric Neurological Disease Research within the St. Jude Pediatric Translational Neuroscience Initiative (PTNI). He received his PhD from the Flinders University of South Australia, Adelaide, where he worked on chromatin structure and ataxia-telangiectasia (A-T), subsequently developing an interest in the neurobiology of A-T. His postdoctoral work focused on molecular neurobiology and disease modeling using mouse genetics. Upon moving to St. Jude Children’s Hospital, he established a research program to study the pathogenesis of neurodegeneration in A-T. This work broadened to include multiple other pediatric neurodegenerative diseases that result from defective genome maintenance.
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Heather C. Mefford, MD, PhD
Heather Mefford, MD, PhD, is a physician scientist and Member in the Center for Pediatric Neurological Disease Research at St. Jude Children’s Research Hospital. She completed her MD and PhD in genetics at University of Washington (UW), followed by a pediatrics residency and a medical genetics fellowship at UW and Seattle Children’s Hospital. Dr. Mefford established her research program in the genetics of rare pediatric disease at UW before moving to St. Jude in 2021. Her research focuses on gene discovery in pediatric neurological diseases, particularly epilepsies, with the goal to leverage information about genetic etiology to develop precision therapies. Her work helped define the genetic landscape of rare developmental and epileptic encephalopathies using cutting-edge genomic technologies. Using engineered and patient-derived cells, her group develops cellular models of genetic epilepsy for characterization and testing potential targeted therapies. She is a scientific advisor for several patient advocacy groups, co-chairs the ClinGen Epilepsy Gene & Variant Curation Working Groups, was co-PI for the Epi4K Consortium, and is Secretary of the Board for the American Society of Human Genetics.
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Francesco Muntoni, MD
Francesco Muntoni, MD, is a Professor of Paediatric Neurology and Director of the Dubowitz Neuromuscular Centre at the University College London (UCL) Great Ormond Street Institute of Child Health and Great Ormond Street Hospital, London. He led the Novel Therapies Programme of the Biomedical Research Centre from 2008 and 2022 and the Developmental Neuroscience Programme from 2008 to 2018. Since 2022, he has directed the Genetic Therapy Accelerator Centre, based at Queen Square Institute of Neurology, in close collaboration with the UCL Great Ormond Street Institute of Child Health. Dr. Muntoni's research in rare neuromuscular disorders, including Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), and congenital myopathies, has led to the development of FDA-approved treatments. He is the UK chief investigator for multiple clinical trials for DMD and SMA and has received multiple international awards, including the 2022 Ottorino Rossi Award, the World Duchenne Organisation 2022 Leadership Awards, the 2023 European Paediatric Neurology Society Jean Aicardi Award, and the 2024 Muscular Dystrophy UK President Award.
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Elizabeth Phimister, PhD
Elizabeth Phimister, PhD, is a Deputy Editor of the New England Journal of Medicine. She evaluates manuscripts describing advances in genetic and genomic research. She earned a PhD in oncology at the University of London and, as a post-doctoral fellow at the University of Cambridge, researched adhesion molecules in pancreatic islets. She served as Editor-in-Chief of Nature Genetics before joining the Journal in 2002.
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Mustafa Sahin, MD, PhD
Mustafa Sahin, MD, PhD, is Neurologist-in-Chief, a pediatric neurologist, and a developmental neurobiologist at Boston Children's Hospital as well as a Professor of Neurology at Harvard Medical School and the Rosamund Stone Zander Chair at Boston Children’s Hospital. He received his ScB degree from Brown University and his MD and PhD from Yale School of Medicine. He completed a pediatrics residency at Children's Hospital of Philadelphia and a child neurology residency at Boston Children's Hospital. At Boston Children’s, Dr. Sahin is the Director of the Translational Research Program, managing director of the Rosamund Stone Zander Translational Neuroscience Center, and the chair of Clinical and Translational Research Executive Committee. He is the co-PI of the Intellectual and Developmental Disabilities Research Center. He directs a national consortium to study biomarkers and comparative pathobiology in three genetic disorders (TSC, PHTS, and Phelan McDermid Syndrome), all associated with autism and intellectual disability. He was elected to the National Academy of Medicine in 2023.
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Kristin Stephenson, MHA, JD
Kristin Stephenson, MHA, JD, is Vice President and Director of the Office of Strategy & Alliances in the Pediatric Translational Neuroscience Initiative (PTNI), which has primary responsibility for developing strategic alliances with external stakeholders and for driving internal alignment to advance the PTNI Mission. Prior to coming to St. Jude in 2021, Kristin served as EVP of a national patient advocacy organization. Before joining the non-profit space, Kristin worked as an attorney in the health care litigation practice groups of Fulbright & Jaworski, LLP, and Akin, Gump, Strauss, Hauer & Feld, LLP, and provided business consulting services in the healthcare practice of Arthur Andersen, LLC.
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Charlotte Sumner, MD
Charlotte Sumner, MD, is a Professor of Neurology, Neuroscience, and Genetic Medicine at Johns Hopkins University School of Medicine. She is a Daniel Nathans Scientific Innovator and Vice Chair for Clinical Research in the Department of Neurology. Dr. Sumner cares for patients with genetically mediated neuromuscular diseases and co-directs the Johns Hopkins Muscular Dystrophy Association Care Center and the Spinal Muscular Atrophy (SMA) and Charcot-Marie-Tooth (CMT) clinics, which deliver multidisciplinary clinical care, engage in international natural history studies, and provide cutting-edge therapeutics. Dr. Sumner’s laboratory research focuses on the genetic and cellular pathogenesis of motor neuron and peripheral nerve disorders, particularly in identifying disease genes and developing therapeutics. Her work contributed to the approval of three gene-directed SMA therapeutics. Dr. Sumner has received a NINDS R35 Research Program Award and has been elected to the American Society of Clinical Investigators, the Interurban Clinical Club, and the American Association of Physicians. She serves as an advisor to several nonprofit foundations as well as to government and private companies and has been recognized with teaching and mentorship awards the NINDS Landis Award for Outstanding Mentorship.
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J. Paul Taylor, MD, PhD
J. Paul Taylor, MD, PhD, is the Scientific Director and Executive Vice President of St. Jude, the Director of the St. Jude Pediatric Neuroscience Initiative (PTNI), and the Edward Barry Endowed Chair in Cell and Molecular Biology. Dr. Taylor’s research program focuses on the molecular basis of inherited neurological disorders. Among Dr. Taylor’s contributions are the discovery that intrinsically disordered protein segments mediate phase separation that underlies the assembly of biomolecular condensates such as RNP granules. Dr. Taylor also discovered several disease-causing mutations in RNA-binding proteins that impair phase transitions. Dr. Taylor won the 2021 Potamkin Prize from the American Academy of Neurology for his work revealing that disturbance of biological phase transitions contribute to neurodegenerative disease.
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Paul Tesar, PhD
Paul Tesar, PhD, is the Dr. Donald and Ruth Weber Goodman Professor of Innovative Therapeutics and Director of the Institute for Glial Sciences at Case Western Reserve University School of Medicine. He earned his undergraduate degree in biology from Case Western Reserve University and a DPhil (PhD) from the University of Oxford as a recipient of an NIH scholarship. Dr. Tesar’s laboratory has pioneered glial cell-focused approaches to understand and treat multiple sclerosis, pediatric leukodystrophies, and other neurodegenerative diseases. He was named a Robertson Investigator by the New York Stem Cell Foundation in 2011 and received the Outstanding Young Investigator Award from the International Society for Stem Cell Research in 2015 and the New York Stem Foundation – Robertson Stem Cell Prize in 2017. In 2019, he was recognized in Crain’s Cleveland Business’ “Forty Under 40” and as a “HomeGrown Hero” in Academic Research by Cleveland.com/The Plain Dealer. He is an elected senior member of the National Academy of Inventors, received the NIH Landis Award for Outstanding Mentoring, and co-founded Convelo Therapeutics, a Cleveland-based biotechnology company focused on remyelination medicines. He is dedicated to elevating science education and giving back to the greater Cleveland community and co-founded the non-profit Rise Up Northeast Ohio.
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Shengdar Tsai, PhD
Shengdar Tsai, PhD, is an Associate Member in the Department of Hematology at St. Jude Children’s Research Hospital. His lab’s research focuses on developing genome editing technologies for therapeutics, with a special interest in editing human HSCs for treatment of hemoglobinopathies such as sickle cell disease and T cells for cancer immunotherapy. His group has advanced state-of-the-art methods for defining the genome-wide activity of genome editors, including GUIDE-seq and CHANGE-seq, and cellular and biochemical assays. In 2020, Dr. Tsai was chosen as one of the American Society for Gene and Cell Therapy Outstanding New Investigators. He co-leads a St. Jude Blue Sky Project Partnership to Advance Development of Individualized Genomic Medicines (PARADIGM) and is a co-chair of the NIH Somatic Cell Genome Editing Consortium Steering Committee. Dr. Tsai completed a postdoctoral fellowship at Massachusetts General Hospital & Harvard Medical School, a PhD in Functional Genomics, a MS in Bioinformatics from North Carolina State University, and a BS from the University of Michigan.
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James W. Wheless, BScPharm, MD
Dr. James W. Wheless, BScPharm, MD, is a leading neurologist and researcher specializing in pediatric antiepileptic drug development, the ketogenic diet, epilepsy surgery, and noninvasive brain mapping (TMS, MEG). He is Professor and Chief of Pediatric Neurology and Le Bonheur Chair in Pediatric Neurology at the University of Tennessee Health Science Center and Director of the Neuroscience Institute and Le Bonheur Comprehensive Epilepsy Program at Le Bonheur Children’s Hospital in Memphis, Tenn. Dr. Wheless holds the Tom Horton & Donna Wiener Endowed Chair in Neurosciences at Le Bonheur Children’s Hospital and is an adjunct clinical faculty member at St. Jude Children’s Research Hospital. He is board certified in child neurology, clinical neurophysiology, and epilepsy and a fellow of the American Academy of Pediatrics, the American Academy of Neurology, and the American Epilepsy Society. Dr. Wheless has authored over 950 chapters, articles, and abstracts and edited four textbooks on epilepsy. He is also a member of the editorial board for the Journal of Child Neurology and lectures widely on pediatric epilepsy, leading an interdisciplinary team in epilepsy treatment.
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Mitchell J. Weiss, MD, PhD
Mitchell (Mitch) J. Weiss, MD, PhD, became Chair of the Hematology Department at St. Jude Children’s Research Hospital in 2014. Before that, Dr. Weiss was the Associate Director for the Human Embryonic Stem Cell Core Facility at The Children’s Hospital of Philadelphia. Dr. Weiss sees pediatric patients with non-malignant blood disorders in the hospital and studies these diseases in the laboratory. His research interests are to better understand the mechanisms of normal red blood cell development and use this information to treat common blood disorders, including sickle cell disease and beta thalassemia.
About the St. Jude Pediatric Translational Neuroscience Initiative (PTNI)
St. Jude Children’s Research Hospital is committed to transforming the treatment landscape for children living with catastrophic pediatric neurological disorders through its Pediatric Translational Neuroscience Initiative (PTNI). Launched in 2020, PTNI serves as a formalized mechanism for translational neuroscience that extends from fundamental bench science to experimental neurotherapeutics with an emphasis on working closely with stakeholders across multiple sectors, including other researchers and institutions, patient and community groups, industry, and policymakers.
Through collaborations and thought partnership, PTNI engages thought leaders to identify promising paths forward in the pediatric neuroscience space, convening scientists, clinicians, patient advocates, and others to address the unmet needs of children with these disorders.