RNARISE™: A Compact, Non-Viral Self-Amplifying RNA Platform for Therapeutics

  • Leverage AI-driven discovery to identify novel eukaryotic RNA replicases and engineer a 1.8 kb non-viral self-amplifying system
  • Demonstrate biochemical validation and cell data confirming functional RNA amplification
  • Translate platform innovation toward therapeutic application through early protein replacement programs in MPSIIIA/B