Revolutionizing AAV Delivery Platforms: Minimizing Immunogenicity & Maximizing Biodistribution for Enhanced Brain Targeting

Time: 2:05 pm
day: Pre-Conference 1 Track PM


Despite notable promising efforts to achieve brain-wide transduction in mice and monkeys, developing a BBB-crossing capsid remains an unsolved problem.

• How can we translate successful cases of AAV delivery in vivo into clinical programs and overcome challenges of cross species differences in capsid property requirements?

• Which features of capsid design should be improved to enhance BBB penetration efficiency and frequency of neuronal uptake?

• How do we establish a successful balance between sufficient dose levels and peripheral immunogenicity?