… RNA therapies based on its proprietary Axiomer ™ RNA editing technology platform, today reported its financial … the past year as we exclusively focused our strategy on RNA editing, ProQR made important progress with Axiomer, our …
… RNA therapies based on its proprietary Axiomer RNA editing technology platform, today reported its financial … the Clinical Trial Application for AX-0810, our lead RNA editing program targeting NTCP for cholestatic diseases, …
… strength of leading IP estate around ADAR-mediated RNA editing highlighted with successful defense of challenge … ADAR RNA editing field in vivo proof of target engagement (RNA editing) with meaningful changes in biomarkers in NHPs …
… RNA therapies for genetic eye diseases and our proprietary RNA-editing platform technology.” “I first met Daniel more … RNA medicines to patients and advance next-generation RNA editing approaches.” About John M. Maraganore, Ph.D. Dr. …
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
… RNA therapies based on its proprietary Axiomer ™ RNA editing technology platform, today reported its financial … ADAR RNA editing field in vivo proof of target engagement (RNA editing) with meaningful changes in biomarkers in NHPs …
… RNA therapies based on its proprietary Axiomer ® RNA editing technology platform, today reported its financial … Genetic Medicines Conference panel discussion on “ ADAR RNA Editing: Unlocking New Therapeutic Opportunities ”. In …
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
AX-0810 clinical target engagement data in healthy volunteers on track for this quarter; biliary atresia selected as initial Phase 2 indicationAdditional programs advancing toward the clinic, including AX-0811 and AX-0422Axiomer platform supporting multiple addi