Company: University of Florida
Job title: Professor & Associate Chairman
Dr. Barry Byrne is a clinician scientist who is studying a variety of rare diseases with the specific goal of developing therapies for inherited muscle disease. As a pediatric cardiologist, his focus is on conditions that lead to skeletal muscle weakness and abnormalities in heart and respiratory function. His group has made significant contributions to the understanding and treatment of Pompe disease, a type of neuromuscular disease due to glycogen storage in motor units. The research team has been developing new therapies using AAV-mediated gene therapy to restore cardiac and skeletal muscle function in DMD, Friedreich’s ataxia, Pompe, and other inherited neuromuscular diseases. His group at the Powell Center has also established a series of new methods for large-scale AAV clinical manufacturing. The work is supported by several NIH and foundation awards.
Dr. Byrne is the Associate Chair of Pediatrics and Director of the University of Florida Powell Center and Child Health Research Institute. He obtained his B.S. degree from Denison University; M.D. and Ph.D. from the University of Illinois; and he completed his Pediatrics residency and cardiology fellowship as well as post-doctoral training in Biological Chemistry at the Johns Hopkins Hospital. Following his early career at Hopkins, he joined the University of Florida and is now the Earl and Christy Powell University Chair in Genetics.
In addition to his academic appointments, he serves as the Chief Medical Advisor of the Muscular Dystrophy Association and a member of the TACT committee of TREAT-NMD. He is an advisor to the Pfizer Rare Disease Therapeutic Area Advisory Committee and co-founder of AavantiBio.
Live Q&A – Ask the speakers your questions 11:20 am
day: Day Two
Immune Management in AAV Gene Therapy to Enhance Safety and Improve Efficacy 9:30 am
• Understanding the mechanisms behind immune response to better inform an immunogenicity mitigation strategy • Optimizing the process of management formulation to better enhance safety and improve efficacy • An immune management AAV gene therapy strategy case studyRead more
day: Day Two