Duke CTSA Special Populations Core Spring Seminar Series Presents: Gene Therapy for Genetic Disorders
Date: Thursday, May 20, 2021
Time: 1:00-2:00 pm
Developing Rare Diseases Therapies in Collaboration with the NIH
Dr. Dwight Koeberl, MD PhD will describe the development of gene therapy for Pompe disease from proof of concept experiments to Phase I clinical trial initiation. This project was performed entirely in the academic environment at Duke University with federal resources, culminating in a partnership with an industry sponsor to support manufacturing and expansion of the clinical trial. It is one example of recent advances in the field of gene therapy for rare diseases.