RNA Therapeutics with LEAPER 2.0: Initial Proof of Concept in Preclinical Models including Human Primary Cells, Mice and Nonhuman Primates

Time: 9:10 am
day: Day Two


  • Understanding how therapeutically meaningful RNA editing rate was achieved in human primary cells in selected genetic diseases
  • Comparing to LEAPER 1.0, LEAPER 2.0 results in a significantly higher level of RNA editing in a mouse model
  • Highlighting the potent and durable RNA editing achieved in multiple target organs of non-human primates via AAV delivery of LEAPER 2.0