CTSI Special Populations: Duke CTSA Special Populations Spring Seminar Series



Presenter: Dwight D. Koeberl, MD PhD

Dwight D. Koeberl, MD, PhD | Medical Genetics Specialist | Duke Health

Meeting Information

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. 

Registration: CLOSED 

Supplemental Materials: A recording of the session is now available online. Also, included are the main presentations slides.