Showcasing the Latest CNS Toxicity Knowledge After Direct Parenchymal Administration

Time: 5:30 pm
day: Day One


  • Assessing the delivery procedure: risks and improvements
  • Exploring local toxicity and AAV capsids, and transgene expression
  • MRI follow-up and evaluation of local tolerance: experience from large animals studies and patients
  • Evaluating the immune reaction and redosing strategies
  • Next steps: disease selection and protocol design