Gene Replacement Therapy: Evolution from Bench to Bedside
Recorded on December 11, 2018
Sponsored by AveXis
Monogenic diseases represent a significant medical, economic and social burden worldwide and, until recently, have proven somewhat of an enigma to manage optimally. Recent advances in gene replacement therapy (GRT) offer the potential to treat single-gene diseases such as cystic fibrosis (CF) and spinal muscular atrophy (SMA).
A new era of GRT is upon us, and therefore, we are pleased to host a panel of experts on translational therapies who will discuss:
- The theory and practicalities of developing GRT for monogenic disease and taking it from the bench to the bedside
- Gene replacement approaches, such as adeno-associated virus (AAV) vectors, along with their advantages and limitations
- Topics related to the biosafety implications of GRT, including vector shedding and proper handling
This exciting webinar offers an overview of the rapidly evolving field of GRT and its therapeutic applications.
Dr. Wendy Chung, MD, PhD
Kennedy Family Professor of Pediatrics and Medicine
Columbia University Institute for Genomic Medicine
Dr. Chris Jenkins, PhD, MPH
Principal Partner and Chief Gene Therapy Biosafety Officer
Clinical Biosafety Sciences
This webinar is sponsored, developed and presented by the sponsor. The content of the Science & Innovation Theater Webinar and opinions expressed by the presenters are those of the sponsor or presenters and not of the Academy of Managed Care Pharmacy.