Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Strengthened leadership with appointments of Chief Financial Officer and Chief
… technology was introduced at the Company’s second annual Research & Development Investor Day and in vivo proof of … F508del mutation were presented at the North American CF conference. A Phase 2 study is currently being designed and … QR-313 pre-clinical data were presented at the EB2017 ResearchConference and ESDR meeting. IND-enabling studies …