Delving into the Strategies to Effectively Measure AAV Safety in the Liver when AAV Gene Therapies are Administered Systemically & Intra CSF

Time: 10:00 am
day: Day Two


• Assessing the importance of the correct patient population to avoid the risk of decompensation 

• Leveraging liver biopsies to understand which cells are infiltrating the liver to gain more mechanistic understanding 

• Integrating novel monitoring methods to explore where the liver damage has occurred 

• Exploring the analysis of red cell morphology to inform on the overall safety profile of AAV