AX-0810 target engagement data in healthy volunteers on track for Q2 2026; biliary atresia selected as initial Phase 2 indicationPipeline expansion with advancement of AX-0811 (NTCP) and AX-0422 (IDUA) toward the clinic, supporting multiple upcoming clinical cataly
First clinical validation of Axiomer™ RNA editing established through NTCP modulation with positive AX-0810 clinical target engagement data; full Phase 1 dataset to be presented at a medical or scientific conference later this yearMultiple clinical milestones, including
… Reports Fourth Quarter and Full Year 2018 Operating and Financial Results LEIDEN, The Netherlands & CAMBRIDGE, Mass., … of severe genetic rare diseases, today reported its financial results for the fourth quarter and full year ended …