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
Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1
Development of Axiomer® RNA base editing technology platform continues across multiple therapeutic areas
Stre
… 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 …