Submitted CTA for lead program AX-0810, targeting NTCP for cholestatic diseasesAdvancing AX-2402 program toward clinical candidate selection, targeting MECP2 (R270X) for Rett SyndromeHosting fall Analyst and Investor Event (virtual), featuring detailed AX-0810 P
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
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 Company expects up to four clinical data readouts in 2025 and 2026 across multi
Phase 2/3 pivotal Illuminate trial of sepofarsen for LCA10 enrollment complete with top-line data anticipated in H1 2022
Positive data reported from the Phase 1/2 clinical trial of QR-421a for the treatment of Usher syndrome and non-syndromic retini
Preclinical proof of concept data for the AX-0810 program targeting NTCP for cholestatic diseases presented at ASGCT 27th Annual Meeting – Management to host webinar today at 8:00 am EDT to highlight dataMartin Maier, PhD nominated to Board with Annual General
Received CTA authorization for Phase 1 trial of lead program AX-0810, targeting NTCP for cholestatic diseasesInitiating Phase 1 study of AX-0810 in healthy volunteers, with initial safety, tolerability, and PK data from Cohort 1 expected by year-end, and target engagemen