Dr. Connor is Executive Director and Scientific Lead for the Pediatric Trials Consortium at Critical Path Institute, President of Clinical Research Alliance LLC, and Emeritus Professor of Pediatrics, Microbiology, Immunology and Tropical Medicine at George Washington University School of Medicine and Health Sciences and Children’s National Health System in Washington DC.

Lily Mulugeta is th Associate Director for Policy and Research in the Division of Pediatrics and Maternal Health at the FDA. She engages in scientific and regulatory review and research related to pediatric drug development. Dr. Mulugeta serves as a representative for the Division on the FDA Pediatric Review Committee (PeRC). Prior to joining the Division in 2017, Lily was the Scientific Lead for Pediatrics in the Division of Pharmacometrics at the FDA. Prior to joining the FDA, Dr. Mulugeta practiced as a Critical Care Specialist at Children’s National Medical Center (CNMC) in Washington D.C. She also served as a faculty member in the Department of Pediatrics at the George Washington School of Medicine and in the Department of Pharma

Christina is Global Head of Pediatric & Maternal Health Policy at Novartis. She graduated from the U Rochester Sch of Medicine & Dentistry and trained in Peds/Pediatric Critical Care Medicine at SUNY Buffalo. She has 20+ yrs of experience in Clinical Development and Regulatory Policy. Her activities advancing the environment for pediatric and maternal health include: ICH E11A Expert Work Group, C-Path International Neonatal Consortium, EFGCP Children's Medicines WP, IQ Consortia Clin Pharm Leadership Group Pediatric WG & NIH/NICHD National Advisory Council on Child Health & Human Development. In her career, Christina has over 40 peer-review published articles and 100 invited lectures.

Dr. Thomas F. Miller is Vice President & Global Head, Pediatric Development for Bayer’s Pharmaceutical Division, a role he assumed in 2017. In total, Dr. Miller’s career in the life science industry spans more than 23 years, with a primary focus on the development of therapeutics, medical devices and combination products for infirmed children. He is a respiratory physiologist with a primary interest in translational and clinical research for pediatric critical care patients. Prior to joining Bayer, Dr. Miller served in the capacity of Chief Executive Officer of Therabron Therapeutics, Inc. with oversight of all operational functions for the company (R&D, manufacturing, quality, regulatory affairs, etc.).

Andrew is currently Senior Vice President, Regulatory Affairs at Neurogene Inc, a company devoted to gene therapy approaches to management of neurodegenerative disorders in children and adults. Formerly, he served Senior Vice President, Global Regulatory Affairs at Amicus Therapeutics from 2016-2020. He served as Division Deputy Director of Gastroenterology and Inborn Errors Products, Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA) since 2010-2016. From 2000-2010 Andrew was Portfolio Leader in Established Products responsible for worldwide leadership of Internal Medicine products in Johnson and Johnson.Andrew is a graduate of Columbia College of Columbia University and Mt. Sinai School of Medicine.

Robert “Skip” Nelson, MD, MDiv, PhD. is currently Senior Director, Pediatric Product Development and Pediatric Strategy Lead in Immunology at Johnson & Johnson. Previously, he was the Deputy Director and Senior Pediatric Ethicist in the Office of Pediatric Therapeutics, Office of the Commissioner at the U.S. Food and Drug Administration. Prior to joining FDA full-time in 2009, he was Professor of Anesthesiology, Critical Care and Pediatrics at The Children’s Hospital of Philadelphia and University of Pennsylvania School of Medicine.

Dr. Portman joined BMS 2007 & Novartis 2014 from Univ of Texas Med Sch (Houston)Professor/Director, Div of Pediatric Nephrology Training: MD (Dartmouth), Pediatrics (Fitzsimons/Univ of Colo), Ped Neph (St Louis Children's). Numerous national committees (BPCA ad com, PAS, Amer Board of Pediatrics, FDA CV/Renal Drugs Ad Com, PhRMA & BIO (Chair) Pediatric Committee, Co-director, International Neonatal Consortium. >100 publications, editor of Pediatric Hypertension text & sub-editor (DIA’s TIRS).

James M. Wilson, MD, PhD, is a Professor in the Perelman School of Medicine at the University of Pennsylvania where he has led an effort to develop the field of gene therapy. His research career spanning over 40 years has focused on rare diseases and ways to treat them by gene therapy. The Wilson lab identified a new type of vector based on novel isolates of adeno-associated viruses which have become best in class for gene therapy being used by over 29 companies. More recently Dr. Wilson’s laboratory has focused on improved vectors for gene therapy and clinical applications of genome editing and mRNA therapy.

I'm a stay-at-home mom. (Simplest way to say I do a little bit of everything.) My husband and I are raising four boys. We're constantly fielding the comment/question everywhere we go, "Four boys! Are you going for a girl?" We have a daughter, too. Heaven is taking care of her for us. That's were Spinal Muscular Atrophy (SMA) enters our story - unique in that we've experienced both ends of the spectrum: a SMA Type 1 diagnosis before treatment was available (our daughter) and the same diagnosis with treatment (our now four year old son). I'm honored to share our journey and love to see the encouragement and hope that has been inspired through the telling.

Dr. Susan McCune is the Director of the Office of Pediatric Therapeutics at FDA. She joined the Agency in 2003, and from 2/10 to 1/17 was Deputy Director, Office of Translational Sciences, CDER. She completed her undergraduate degree at Harvard, her MD at GWU, and training at Children’s National Medical Center (CNMC). She is Board Certified in Pediatrics and Neonatal/Perinatal Medicine, and practiced academic medicine at Johns Hopkins and CNMC with research at NICHD prior to joining the FDA.

Dr. Meyer performed her graduate studies at the Institute of Cell Biology in Berne, Switzerland focusing on molecular and cell biology and especially RNA metabolism. She developed splicing correction therapeutical approaches for Spinal Muscular Atrophy (SMA) before moving to Columbus Ohio to work with Dr. Brian Kaspar on SMA gene therapy. As a post-doctoral researcher and later independent Principal Investigator and Assistant Professor at The Ohio State University, she developed various gene therapy approaches for severe neurological and neurodegenerative disorders. 4 programs were successfully translated to clinical trials to date. Dr. Meyer also establishes in vitro modelling assays to study neurological diseases and perform drug testing.

Dr. Marc Gastonguay has dedicated more than 25 years to science, advocacy, innovation and education in the discipline of pharmacometrics. He is founder and CEO of Metrum Research Group, a provider of strategic biomedical modeling and simulation solutions. Dr. Gastonguay received a BS in Pharmacy from the University of Connecticut, a PhD in Pharmacology from Georgetown University, and postdoctoral Fellowship at the US FDA. His current research interests include Bayesian modeling methods, pediatric clinical pharmacology, and drug development in rare diseases. He is also a strong advocate and contributor to open-science initiatives, including open-source software, open disease models & data and open courseware.

Gavin Miyasato is Associate Director of Statistics at TRINITY, a leading global life sciences consulting firm. He has been a member of the TRINITY team for 14 years and has worked closely with clients to support strategic decision making across the product lifecycle. He currently leads the Real-World Evidence Analytics function, where a key element of his role focuses on generating evidence for pharma/biotech/medical device companies to enhance their product value messaging to payers, physicians and patients. Gavin holds a B.S. in Chemical Engineering from MIT and a M.S. in Biostatistics from Harvard University.

Dr. John Michael Sauer is a pharmacologist and toxicologist by training with over 20 years of experience in drug discovery and development. He has been responsible for leading multiple functional areas across several pharmaceutical companies. He received his Doctorate degree from The University of Arizona. Currently, John Michael is the Program Officer of Biomarkers Program and the Executive Director of the Predictive Safety Testing Consortium (PSTC), Inflammatory Bowel Disease (IBD) Group, and Polycystic Kidney Disease Outcomes Consortium (PKDOC) at the Critical Path Institute, as well as an Adjunct Research Professor in the Department of Pharmacology at the University of Arizona, College of Medicine.

Neonatologist with a special interest in early phase drug development and improving the availability of high quality medicines to children and babies. Has studied 20 medicines in neonates and pregnant women including antibiotics, excipients, treatments for in utero growth restriction, inotropes, postnatal steroids and surfactant. Current work is focused on developing research infrastructure for pediatric medicines research in Europe and beyond.

Abby Bronson is the Vice President of Patient Advocacy and External Innovation at Edgewise Therapeutics where she leads patient advocacy and external relationships within the Muscular Dystrophy community. Previously she was Senior Vice President of Research Strategy at PPMD, focusing on Duchenne Muscular Dystrophy. She has experience from a variety of areas, including NIH, Children's National Medical Center and MedImmune. She holds an MBA from the Wharton School of Business at the University of Pennsylvania and a BA from the University of Vermont.

Dan Fields is the Vice President, Business Innovation at Children’s Hospital of Philadelphia (CHOP). At CHOP, Dan has responsibility for advancing a number of programs focused on advancing the institution’s innovation and growth priorities. He oversees CHOP’s novel accelerator for breakthrough clinical and research, which provides significant strategic investments in CHOP-led programs that are making transformative advancements in pediatric care. Dan provides administrative leadership for our new strategy related to cell & gene therapy. He also leads national business development efforts for the organization, focused on provider outreach and education across the country.

Lisa is a member of the Statistical Methodology group at Novartis and is based in Basel. Prior to joining the pharmaceutical industry, Lisa was a Lecturer in Statistics at Lancaster University, and held a UK Medical Research Council Career Development Award in Biostatistics. Her research interests are in clinical trials, including group sequential tests, adaptive designs for treatment selection, and Bayesian approaches for early phase dose-escalation trials. More widely, she is interested in developing approaches for leveraging existing information in trials, extrapolation, and methods to evaluate the probability of success of a drug development program. Lisa holds a PhD in Statistics from the University of Bath.

Dr. Nikolov is a board-certified Internist and Rheumatologist who joined FDA in 2009 and is currently Associate Director for Rheumatology in the Division of Pulmonary, Allergy and Rheumatology Products, CDER, where he reviews immuno-modulatory therapies for the treatment of pediatric and adult rheumatologic diseases. He completed his residency at Lincoln Medical Center, Bronx, NY and then a fellowship in rheumatology at the NIH, where participated in clinical investigational protocols and studied cellular and molecular mechanisms of autoimmunity. In 2005, Dr. Nikolov joined the Sjögren's Syndrome Group at the NIH, as a clinical investigator where he did research in systemic lupus erythematosus and Sjögren's syndrome.

Vicki Seyfert-Margolis, PhD is the founder and CEO of My Own Med, Inc., a company specializing in using digital technologies to support real world evidence clinical research. Previously, Dr. Seyfert-Margolis was the Senior Advisor for Science Innovation and Policy in the Office of the Commissioner of the US Food and Drug Administration. Vicki also serves on Board of Directors for the EveryLife Foundation for Rare Diseases, and Eureka Institute for Translational Medicine.