Agenda Details | ASGCT Annual Meeting

Detailed Program

Wednesday, May 17 2023
08:00 AM – 09:45 AM PT

Advancing Novel Delivery Tools and Treatments for Neurodegenerative Disorders

There is an urgent need to investigate therapeutic strategies and novel treatments for genetic or acquired neurodegenerative disorders. To this end, important issues related to the effective delivery and biodistribution of the therapeutic product/s in target tissues, the durability of the beneficial effects as well as the potential toxicity of the procedure and/or products have to be carefully evaluated.

Session Details

08:00 AM – 09:45 AM - Room 411

Co-Chair

Angela Gritti, PhD. San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Italy

08:00 AM – 09:45 AM - Room 411

Co-Chair

Fredric Manfredsson. Barrow Neurological Institute, AZ

08:00 AM – 09:45 AM - Room 411

Co-Chair

Megan S. Keiser, PhD. Children's Hospital of Philadelphia, Philadelphia, PA

08:01 AM – 08:25 AM - Room 411

Discovery and Characterization of Novel Cross-Species BBB-Penetrant Capsids

Brett Hoffman, PhD. Voyager Therapeutics, Cambridge, MA

08:25 AM – 08:50 AM - Room 411

Exosomes for delivery to the CNS

Dhanu Gupta. University of Oxford, Oxford, United Kingdom

08:50 AM – 09:15 AM - Room 411

A Novel Delivery Approach to Support Development of Genetic Medicines for Human Inner Ear Conditions

Michelle D. Valero, PhD. Akouos, a wholly owned subsidiary of Eli Lilly, MA

09:15 AM – 09:40 AM - Room 411

Pushing the Envelope: Will Novel Gene Knockdown Strategies Get us to a Cure for Motor Neuron Disease?

Defne Amado, MD, PHD. University of Pennsylvania, PA