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, AskBio

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

11:00 am 

Exploring Advances in Devices for Direct Gene Therapy Injection

Krystof Bankiewicz, MD, President & Chief Executive Officer, Brain Neurotherapy Bio

Synopsis
• Explore the state-of-the-art technical improvements for gene therapy delivery
• Introducing the latest MRI-friendly cannulas and needles
• Common challenges with age of patient, tech transfer and scaling

11:20 am 

Q&A Panel Discussion: Delivery

Martin Marsala, Professor, University of California, San Diego
Krystof Bankiewicz, President & Chief Executive Officer, Brain Neurotherapy Bio
Olivier Danos, Chief Scientific Officer, REGENXBIO
R. Jude Samulski, Chief Scientific Officer, AskBio

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?