2nd Annual Gene Therapy for Neurological Disorders

Join us at the virtual 2nd Annual Gene Therapy for Neurological Disorders conference from December 8-10, 2020 to learn about translational drug development challenges encountered in the development of neurological gene therapies.

December 10, 2020

10:00am ET

Lessons from the Clinic & Why AAV Biology Still Matters

Jude Samulski, PhD, Chief Scientific Officer, RhiaPharmaceutical

Synopsis
• Follow the history of AAV administration for CNS disorders
• Discuss the value of AAV biology
• Review clinical data

11:20 am 

Q&A Panel Discussion: Delivery

Martin Marsala, Professor, University of California, San Diego
Krystof Bankiewicz, MD, PhD, Department of Neurological Surgery, The Ohio State University Wexner Medical Center
Olivier Danos, Chief Scientific Officer, REGENXBIO
R. Jude Samulski, Chief Scientific Officer, RhiaPharmaceutical

Synopsis
• Comparing different patient populations for different indications and their suitability for different delivery routes
• Discussing bio-distribution vs. invasive procedures for delivery to the brain
• Exploring the risk/benefit of different routes of administration, is the data translatable?
• Introducing technology advances for the delivery devices – where is there room for improvement?