VIRTUAL: Engineered AAVs with Targeted & efficient Noninvasive Gene Delivery to the Primate CNS

Time: 11:45 am
day: Day One: Track A AM


  • High-throughput screening of AAV capsid libraries directly in non-human primates yields multiple generations of improved AAVs for delivery to the CNS
    Combinatorial engineering of multiple surface-exposed loops confers organ and cell-type level tropism and transduction
  • The resulting engineered capsids show robust and uniform DNA, RNA, and protein expression throughout the primate CNS, and dramatically increased transduction and transcription in human neurons
  • The engineered capsids are highly scalable on our suspension culture GMP-manufacturing platform, with high productivity and product quality