Systemic Administration of Novel Engineered AAV Capsids Facilitates Enhanced Transgene Expression in the Macaque CNS

Time: 12:00 pm
day: Day Two: Track B AM


  • Utilizing an mRNA-based selection strategy to select novel capsids with enhanced tropism for the CNS after intravenous delivery
  • We identified the novel PAL family of capsids which have enhanced CNS targeting and reduced liver tropism in 2 year old cynomolgus macaques
  • We found that a diverse set of capsids potent in the murine CNS were universally ineffective in macaques