2021 Technology Showcase
Thursday, March 4, 2021
Presented virtually via Vimeo
Thank you to our presenters and all those who attended. This year's event recording is now available. Join us in learning about and celebrating the innovative discoveries and technologies improving pediatric health.
Gene Therapy for FSHD
Dr. Harper will discuss translation of a gene silencing therapy for one of the most common forms of muscular dystrophy.
Presenter: Scott Harper, PhD
Principal Investigator, Center for Gene Therapy, Nationwide Children’s Hospital; Professor of Pediatrics, The Ohio State University College of Medicine
Scott Harper, PhD, is a professor of Pediatrics in the College of Medicine at The Ohio State University and a principal investigator in the Center for Gene Therapy at the Abigail Wexner Research Institute. Dr. Harper is a molecular biologist who has spent his career working to develop gene therapy approaches for muscular dystrophies and neurodegenerative diseases, including Duchenne muscular dystrophy (DMD), Huntington’s Disease, Charcot-Marie-Tooth disorders, dominant forms of limb-girdle muscular dystrophies (LGMD), epilepsy and facioscapulohumeral muscular dystrophy (FSHD). Dr. Harper was selected as the Outstanding New Investigator by the American Society of Gene and Cell Therapy in 2014.
Genome editing to express full-length dystrophin in Duchenne muscular dystrophy
Genome editing approaches allow the possibility of expression of full-length dystrophin (rather than engineered miniaturized dystrophin) in the muscle of boys with Duchenne muscular dystrophy (DMD).
Presenter: Kevin Flanigan, MD
Director, Center for Gene Therapy, Nationwide Children’s Hospital; Professor of Pediatrics and Neurology, Ohio State University
Kevin Flanigan, MD, is the Director of the Center for Gene Therapy at Nationwide Children’s Hospital in Columbus, Ohio, where he holds the Robert F. & Edgar T. Wolfe Foundation Endowed Chair in Neuromuscular Research. He also directs the Nationwide Children’s Hospital Neuromuscular Program as well as Nationwide Children’s NIAMS P50-funded Center for Research Translation (CORT) in Muscular Dystrophy Therapeutic Development. Dr. Flanigan trained in Neurology & Neuromuscular Medicine at Johns Hopkins University, pursued a post-doctoral fellowship in Human Molecular Biology and Genetics at the University of Utah, and currently holds appointments as professor of Pediatrics and Neurology at The Ohio State University. Dr. Flanigan’s research focuses on genotype/phenotype correlations in the muscular dystrophies, with a goal of understanding molecular mechanisms that lead to amelioration of symptoms in order to identify new therapeutic pathways. His laboratory works primarily on adeno associated vector (AAV) based approaches to gene therapy, including viral-vector based exon skipping and gene replacement.
Advancing Development and Recovery Through Movement
Explore groundbreaking solutions in early intervention designed to establish improved care pathways for patients with loss of mobility and review initial findings from ongoing clinical studies of non-invasive neuromotor screening, led by Nathalie Maitre, MD, PhD, and autonomous gait training for lower-limb amputees immediately following surgery.
Presenter: Marissa Koscielski, MS
Founder, Enlighten Mobility
Marissa Koscielski founded Enlighten Mobility in 2017 while completing her master’s degree in the ESTEEM Graduate Program at the University of Notre Dame. Having suffered temporary unilateral immobility as a teen and experiencing firsthand the lack of effective rehabilitation solutions for her own recovery, Marissa set out to challenge the status quo of rehabilitation, which remains at the core of the Enlighten Mobility mission. Marissa’s passion for patient-centered innovation to improve quality of life fuels her entrepreneurial pursuits. In addition to Enlighten Mobility, Marissa has founded three healthcare ventures and has held a leadership role in another successful medical device startup. Marissa is a mentor to Mayo Clinic Pediatric Rehabilitation, where she was treated, and a former intern for A Rosie Place respite care hospital. In addition to her ESTEEM master’s degree, Marissa holds a bachelor’s degree in mathematics and pre-health from the University of Notre Dame.
Development of a Humanized Monoclonal Antibody As a Unique Therapy for Infections Caused by Bacterial Biofilms
Clarametyx Biosciences is developing licensed technology from Nationwide Children’s Hospital that rapidly collapses bacterial biofilms in a pathogen-agnostic fashion; the first clinical evaluation of this technology will occur in patients hospitalized with bacterial pneumonia.
Presenter: Charles McOsker, PhD
Co-founder & Chief Scientific Officer, Clarametyx Biosciences
Charles McOsker, PhD, is a veteran scientific executive with over 30 years of experience in drug discovery and development. Before joining Clarametyx, Dr. McOsker was Chief Technical Operations Officer at BioMotiv, which identifies and accelerates the development of breakthrough new therapeutics. Previously, Dr. McOsker founded Airway Therapeutics, which was created to develop pulmonary therapeutics based on the pulmonary research program at Cincinnati Children’s Hospital. Dr. McOsker has also served as Director of Business Development for the Drug Discovery Center at the University of Cincinnati. Dr. McOsker began his career at Procter & Gamble Pharmaceuticals, where he held both research and management positions in drug discovery and development over his 23-year tenure. Dr. McOsker received his bachelor’s degree in chemistry from Hope College in Michigan and his doctoral degree in biochemistry and molecular biology from Cornell University.
AAV Gene Therapy for Lysosomal Acid Lipase Deficiency
This presentation will describe the engineering and testing of a new AAV gene therapy treatment for Lysosomal Acid Lipase Deficiency.
Presenter: Paul T. Martin, PhD
Principal Investigator, Center for Gene Therapy, Nationwide Children’s Hospital
Professor of Pediatrics and Professor of Physiology and Cell Biology at The Ohio State University College of Medicine
Paul T. Martin, PhD, is a Principal Investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital. He is Professor of Pediatrics and Professor of Physiology and Cell Biology at The Ohio State University College of Medicine. He is also Associate Director of the NIH Center of Research Translation in Muscular Dystrophy Therapeutic Develpment at Nationwide Children’s Hospital. Dr. Martin’s NIH-funded research program focuses on defining functional roles for cell surface carbohydrates in neuromuscular development and disease. Dr. Martin’s lab has identified particular carbohydrates that, when overexpressed in muscle cells, can prevent muscular dystrophy from occurring in a number of forms of the disease. Current work is focused on translating these findings into therapies that can be used by patients with these disorders. Additional work focuses on on the development of novel protein and gene therapies for congenital forms of muscular dystrophy.