Gene Therapy Pioneers Discuss What's Next for Gene Therapy

3:00 PM - 4:00 PM (PDT), Wednesday, June 15, 2022

Recent successes in genetic medicine (regulatory approvals of Zolgensma and Luxturna, for example) have paved the path for a broader second wave of therapies and laid the foundation for next-generation technologies. But there are still obstacles for these therapies, for example safety signals, expression in target tissues, and longevity of effect. From the expert panel of gene therapy pioneers, we will hear about some of the challenges and how they are being tackled.

Dr. Goldsmith, CEO of a leading genetic medicines company, will moderate the interactive panel discussion. He will pose questions to get at how gene therapy will likely reshape the future of biomedical research and medicine.

Discussion topics likely will include: 
- Immune response to gene delivery vectors and products of foreign transgenes
- Gene-editing technologies
- The future of functional genomics, given that only approximately 6,000 of approx. 20,000 human genes are known

Director, Gene Therapy Program, Rose H. Weiss Professor and Director, Orphan Disease Center, Professor of Medicine and Pediatrics, Perelman School of Medicine, University of Pennsylvania, Chief Scientific Advisor, Passage Bio
Gene Therapy Program at the University of Pennsylvania
Universitat Autonoma Barcelona
University of California, Los Angeles