March 2, 2023 from 1 – 4 p.m. The Blackwell Inn & Pfahl Conference Center 2110 Tuttle Park Pl, Columbus, OH 43210 Join us for presentations about new and impactful technologies emerging through research and innovation at Nationwide Children’s Hospital, an update by our licensee Sarepta Therapeutics, and a keynote presentation on the role of the U.S. Food and Drug Administration in accelerating gene and cell therapy innovation to benefit patients. A networking reception will follow the in-person event. In-person seating is limited. Register Now This event will also be livestreamed, virtually. Keynote Speaker – Peter Marks, MD, PhD
Director, Center for Biologics Evaluation and Research U.S. Food and Drug Administration (FDA)
Moving Gene Therapy to the Next Level Gene therapy offers tremendous promise for the treatment of both rare and common diseases. Despite initial successes, particularly in the area of genetically-modified cellular therapies such as CAR-T cells, growth of the field has been slower than predicted.
By leveraging characteristics that products have in common when possible, by focusing on advancing manufacturing, by maximally employing expedited development pathways, and by fostering international collaboration, FDA hopes to accelerate the pace of bringing advances to the benefit of patients.
Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching and caring for patients and in the industry on drug development and is an author or co-author of over 100 publications. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in 2016. Over the past several years he has been integrally involved in the response to various public health emergencies, and in 2022 he was elected a member of the National Academy of Medicine.
Agenda
Introduction
Matthew McFarland, RPh, PhD Vice President, Commercialization and Industry Relations Nationwide Children’s Hospital
The Nationwide Innovation Center Endeavor; Innovation in Nursing Practice Cory Criss and Timothy Landers will highlight Nationwide Children’s innovative models and discuss programming established to foster innovative ideas. Presenters:
Cory Criss, MD General Pediatric Surgery Nationwide Children’s Hospital Timothy Landers, PhD APRN-CNP FAAN Nurse Scientist, Center for Nursing Excellence Nationwide Children’s Hospital
Child-Resistant and Senior-Friendly Packaging Testing for Patented, Two-Stage Sprayer Lara McKenzie will describe the development and proposed child-resistant and senior-friendly packaging testing of a two-stage trigger system to prevent accidental operation of a spray bottle containing household chemicals and dangerous solutions. Presenter:
Lara McKenzie, PhD, MA, FAAHB Principal Investigator and Director of Training and Education, Center for Injury Research and Policy Nationwide Children’s Hospital
Development and Commercialization of Organs on Chips Gail Besner and Belgacem Mihi will discuss the development of a human intestinal organ-on-a-chip platform as a reliable alternative to pre-clinical animal testing, in order to improve the efficiency of bench-to-bedside investigational drug discovery. Presenters:
Gail E. Besner, MD Chief, H. William Clatworthy Jr. Professor of Surgery, Department of General Pediatric Surgery Principal Investigator, Center for Perinatal Research Nationwide Children’s Hospital Belgacem Mihi, DVM, PhD Research Scientist, Center for Perinatal Research Nationwide Children’s Hospital
Manipulating Mammalian Gene Expression with Bacterial Extracellular Vesicles Christian Lauber will discuss how exposure to P. gingivalis outer membrane vesicles alters neurodevelopment of offspring in utero. Presenter:
Christian Lauber, PhD Microbial Ecologist Nationwide Children’s Hospital
Delivering on the Promise for Transformative Gene Medicine for Muscular Dystrophies As a precision genetic medicine company focused on bringing a better life to patients with rare neuromuscular diseases, Sarepta Therapeutics is a valuable contributor to the Ohio biotechnology ecosystem. Groundbreaking work in Duchenne and limb-girdle muscular dystrophies is driven by multi-platform technologies in gene therapy, RNA and gene editing. Sarepta’s work in gene therapy, rooted in Ohio at its Genetic Therapies Center of Excellence (GTCOE), represents the most promising and clinically advanced in the world. Presenter:
Louise Rodino-Klapac, PhD Executive Vice President, Head of Research and Development and Chief Scientific Officer Sarepta Therapeutics, Inc., Genetic Therapies Center of Excellence (GTCOE)
March 2, 2023 from 1 – 4 p.m.
Join us for presentations about new and impactful technologies emerging through research and innovation at Nationwide Children’s Hospital, an update by our licensee Sarepta Therapeutics, and a keynote presentation on the role of the U.S. Food and Drug Administration in accelerating gene and cell therapy innovation to benefit patients.
A networking reception will follow the in-person event.
In-person seating is limited.
Register Now
This event will also be livestreamed, virtually.
Keynote Speaker – Peter Marks, MD, PhD
Director, Center for Biologics Evaluation and Research U.S. Food and Drug Administration (FDA)
Moving Gene Therapy to the Next Level Gene therapy offers tremendous promise for the treatment of both rare and common diseases. Despite initial successes, particularly in the area of genetically-modified cellular therapies such as CAR-T cells, growth of the field has been slower than predicted.
By leveraging characteristics that products have in common when possible, by focusing on advancing manufacturing, by maximally employing expedited development pathways, and by fostering international collaboration, FDA hopes to accelerate the pace of bringing advances to the benefit of patients.
Director, Center for Biologics Evaluation and Research U.S. Food and Drug Administration (FDA)
Moving Gene Therapy to the Next Level Gene therapy offers tremendous promise for the treatment of both rare and common diseases. Despite initial successes, particularly in the area of genetically-modified cellular therapies such as CAR-T cells, growth of the field has been slower than predicted.
Director, Center for Biologics Evaluation and Research U.S. Food and Drug Administration (FDA)
Moving Gene Therapy to the Next Level
Gene therapy offers tremendous promise for the treatment of both rare and common diseases. Despite initial successes, particularly in the area of genetically-modified cellular therapies such as CAR-T cells, growth of the field has been slower than predicted.
Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching and caring for patients and in the industry on drug development and is an author or co-author of over 100 publications. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in 2016. Over the past several years he has been integrally involved in the response to various public health emergencies, and in 2022 he was elected a member of the National Academy of Medicine.
Agenda
Introduction
Matthew McFarland, RPh, PhD Vice President, Commercialization and Industry Relations Nationwide Children’s Hospital
The Nationwide Innovation Center Endeavor; Innovation in Nursing Practice Cory Criss and Timothy Landers will highlight Nationwide Children’s innovative models and discuss programming established to foster innovative ideas. Presenters:
Cory Criss, MD General Pediatric Surgery Nationwide Children’s Hospital Timothy Landers, PhD APRN-CNP FAAN Nurse Scientist, Center for Nursing Excellence Nationwide Children’s Hospital
Child-Resistant and Senior-Friendly Packaging Testing for Patented, Two-Stage Sprayer Lara McKenzie will describe the development and proposed child-resistant and senior-friendly packaging testing of a two-stage trigger system to prevent accidental operation of a spray bottle containing household chemicals and dangerous solutions. Presenter:
Lara McKenzie, PhD, MA, FAAHB Principal Investigator and Director of Training and Education, Center for Injury Research and Policy Nationwide Children’s Hospital
Development and Commercialization of Organs on Chips Gail Besner and Belgacem Mihi will discuss the development of a human intestinal organ-on-a-chip platform as a reliable alternative to pre-clinical animal testing, in order to improve the efficiency of bench-to-bedside investigational drug discovery. Presenters:
Gail E. Besner, MD Chief, H. William Clatworthy Jr. Professor of Surgery, Department of General Pediatric Surgery Principal Investigator, Center for Perinatal Research Nationwide Children’s Hospital Belgacem Mihi, DVM, PhD Research Scientist, Center for Perinatal Research Nationwide Children’s Hospital
Manipulating Mammalian Gene Expression with Bacterial Extracellular Vesicles Christian Lauber will discuss how exposure to P. gingivalis outer membrane vesicles alters neurodevelopment of offspring in utero. Presenter:
Christian Lauber, PhD Microbial Ecologist Nationwide Children’s Hospital
Delivering on the Promise for Transformative Gene Medicine for Muscular Dystrophies As a precision genetic medicine company focused on bringing a better life to patients with rare neuromuscular diseases, Sarepta Therapeutics is a valuable contributor to the Ohio biotechnology ecosystem. Groundbreaking work in Duchenne and limb-girdle muscular dystrophies is driven by multi-platform technologies in gene therapy, RNA and gene editing. Sarepta’s work in gene therapy, rooted in Ohio at its Genetic Therapies Center of Excellence (GTCOE), represents the most promising and clinically advanced in the world. Presenter:
Louise Rodino-Klapac, PhD Executive Vice President, Head of Research and Development and Chief Scientific Officer Sarepta Therapeutics, Inc., Genetic Therapies Center of Excellence (GTCOE)
Agenda
Introduction
Matthew McFarland, RPh, PhD Vice President, Commercialization and Industry Relations Nationwide Children’s Hospital
The Nationwide Innovation Center Endeavor; Innovation in Nursing Practice Cory Criss and Timothy Landers will highlight Nationwide Children’s innovative models and discuss programming established to foster innovative ideas. Presenters:
Cory Criss, MD General Pediatric Surgery Nationwide Children’s Hospital Timothy Landers, PhD APRN-CNP FAAN Nurse Scientist, Center for Nursing Excellence Nationwide Children’s Hospital
Child-Resistant and Senior-Friendly Packaging Testing for Patented, Two-Stage Sprayer Lara McKenzie will describe the development and proposed child-resistant and senior-friendly packaging testing of a two-stage trigger system to prevent accidental operation of a spray bottle containing household chemicals and dangerous solutions. Presenter:
Lara McKenzie, PhD, MA, FAAHB Principal Investigator and Director of Training and Education, Center for Injury Research and Policy Nationwide Children’s Hospital
Development and Commercialization of Organs on Chips Gail Besner and Belgacem Mihi will discuss the development of a human intestinal organ-on-a-chip platform as a reliable alternative to pre-clinical animal testing, in order to improve the efficiency of bench-to-bedside investigational drug discovery. Presenters:
Gail E. Besner, MD Chief, H. William Clatworthy Jr. Professor of Surgery, Department of General Pediatric Surgery Principal Investigator, Center for Perinatal Research Nationwide Children’s Hospital Belgacem Mihi, DVM, PhD Research Scientist, Center for Perinatal Research Nationwide Children’s Hospital
Manipulating Mammalian Gene Expression with Bacterial Extracellular Vesicles Christian Lauber will discuss how exposure to P. gingivalis outer membrane vesicles alters neurodevelopment of offspring in utero. Presenter:
Christian Lauber, PhD Microbial Ecologist Nationwide Children’s Hospital
Delivering on the Promise for Transformative Gene Medicine for Muscular Dystrophies As a precision genetic medicine company focused on bringing a better life to patients with rare neuromuscular diseases, Sarepta Therapeutics is a valuable contributor to the Ohio biotechnology ecosystem. Groundbreaking work in Duchenne and limb-girdle muscular dystrophies is driven by multi-platform technologies in gene therapy, RNA and gene editing. Sarepta’s work in gene therapy, rooted in Ohio at its Genetic Therapies Center of Excellence (GTCOE), represents the most promising and clinically advanced in the world. Presenter:
Louise Rodino-Klapac, PhD Executive Vice President, Head of Research and Development and Chief Scientific Officer Sarepta Therapeutics, Inc., Genetic Therapies Center of Excellence (GTCOE)
Introduction
Matthew McFarland, RPh, PhD Vice President, Commercialization and Industry Relations Nationwide Children’s Hospital
The Nationwide Innovation Center Endeavor; Innovation in Nursing Practice Cory Criss and Timothy Landers will highlight Nationwide Children’s innovative models and discuss programming established to foster innovative ideas. Presenters:
Cory Criss, MD General Pediatric Surgery Nationwide Children’s Hospital Timothy Landers, PhD APRN-CNP FAAN Nurse Scientist, Center for Nursing Excellence Nationwide Children’s Hospital
Child-Resistant and Senior-Friendly Packaging Testing for Patented, Two-Stage Sprayer Lara McKenzie will describe the development and proposed child-resistant and senior-friendly packaging testing of a two-stage trigger system to prevent accidental operation of a spray bottle containing household chemicals and dangerous solutions. Presenter:
Lara McKenzie, PhD, MA, FAAHB Principal Investigator and Director of Training and Education, Center for Injury Research and Policy Nationwide Children’s Hospital
Development and Commercialization of Organs on Chips Gail Besner and Belgacem Mihi will discuss the development of a human intestinal organ-on-a-chip platform as a reliable alternative to pre-clinical animal testing, in order to improve the efficiency of bench-to-bedside investigational drug discovery. Presenters:
Gail E. Besner, MD Chief, H. William Clatworthy Jr. Professor of Surgery, Department of General Pediatric Surgery Principal Investigator, Center for Perinatal Research Nationwide Children’s Hospital Belgacem Mihi, DVM, PhD Research Scientist, Center for Perinatal Research Nationwide Children’s Hospital
Manipulating Mammalian Gene Expression with Bacterial Extracellular Vesicles Christian Lauber will discuss how exposure to P. gingivalis outer membrane vesicles alters neurodevelopment of offspring in utero. Presenter:
Christian Lauber, PhD Microbial Ecologist Nationwide Children’s Hospital
Delivering on the Promise for Transformative Gene Medicine for Muscular Dystrophies As a precision genetic medicine company focused on bringing a better life to patients with rare neuromuscular diseases, Sarepta Therapeutics is a valuable contributor to the Ohio biotechnology ecosystem. Groundbreaking work in Duchenne and limb-girdle muscular dystrophies is driven by multi-platform technologies in gene therapy, RNA and gene editing. Sarepta’s work in gene therapy, rooted in Ohio at its Genetic Therapies Center of Excellence (GTCOE), represents the most promising and clinically advanced in the world. Presenter:
Louise Rodino-Klapac, PhD Executive Vice President, Head of Research and Development and Chief Scientific Officer Sarepta Therapeutics, Inc., Genetic Therapies Center of Excellence (GTCOE)
Introduction
- Matthew McFarland, RPh, PhD
- Vice President, Commercialization and Industry Relations
- Nationwide Children’s Hospital
The Nationwide Innovation Center Endeavor; Innovation in Nursing Practice
Cory Criss and Timothy Landers will highlight Nationwide Children’s innovative models and discuss programming established to foster innovative ideas.
Presenters:
- Cory Criss, MD
- General Pediatric Surgery
- Nationwide Children’s Hospital
- Timothy Landers, PhD APRN-CNP FAAN
- Nurse Scientist, Center for Nursing Excellence
- Nationwide Children’s Hospital
Child-Resistant and Senior-Friendly Packaging Testing for Patented, Two-Stage Sprayer
Lara McKenzie will describe the development and proposed child-resistant and senior-friendly packaging testing of a two-stage trigger system to prevent accidental operation of a spray bottle containing household chemicals and dangerous solutions.
Presenter:
- Lara McKenzie, PhD, MA, FAAHB
- Principal Investigator and Director of Training and Education, Center for Injury Research and Policy
- Nationwide Children’s Hospital
Development and Commercialization of Organs on Chips
Gail Besner and Belgacem Mihi will discuss the development of a human intestinal organ-on-a-chip platform as a reliable alternative to pre-clinical animal testing, in order to improve the efficiency of bench-to-bedside investigational drug discovery.
- Gail E. Besner, MD
- Chief, H. William Clatworthy Jr. Professor of Surgery, Department of General Pediatric Surgery
- Principal Investigator, Center for Perinatal Research
- Nationwide Children’s Hospital
- Belgacem Mihi, DVM, PhD
- Research Scientist, Center for Perinatal Research
- Nationwide Children’s Hospital
Manipulating Mammalian Gene Expression with Bacterial Extracellular Vesicles
Christian Lauber will discuss how exposure to P. gingivalis outer membrane vesicles alters neurodevelopment of offspring in utero.
- Christian Lauber, PhD
- Microbial Ecologist
- Nationwide Children’s Hospital
Delivering on the Promise for Transformative Gene Medicine for Muscular Dystrophies
As a precision genetic medicine company focused on bringing a better life to patients with rare neuromuscular diseases, Sarepta Therapeutics is a valuable contributor to the Ohio biotechnology ecosystem. Groundbreaking work in Duchenne and limb-girdle muscular dystrophies is driven by multi-platform technologies in gene therapy, RNA and gene editing. Sarepta’s work in gene therapy, rooted in Ohio at its Genetic Therapies Center of Excellence (GTCOE), represents the most promising and clinically advanced in the world.
- Louise Rodino-Klapac, PhD
- Executive Vice President, Head of Research and Development and Chief Scientific Officer
- Sarepta Therapeutics, Inc., Genetic Therapies Center of Excellence (GTCOE)
