Challenges to Implementing & Translating Biomarker Strategies in Gene Therapy for Neurodegenerative Diseases

Time: 11:20 am
day: Day Two


  • Translating biomarker strategies for gene therapy for neurodegenerative diseases programs presents unique challenges
  • Efficacy studies in mice usually are not performed in translatable matrices
  • Safety studies in mice or NHP usually do not reflect response in humans
  • Snapshot of some possible ways to navigate issue