Development of Axiomer® RNA base editing technology platform continues across multiple therapeutic areas; initial pipeline targets to be disclosed in early 2023 with Analyst and Investor R&D event to be held in Q1 2023
Management Team appointments and p
Foundation Fighting Blindness and ProQR enter into a partnership to develop QR-421a for Usher syndrome type 2A, targeting mutations in exon 13 of the causative USH2A gene.
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
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
Initial AX-0810 data show no safety signals after 4 weeks of dosing and pharmacokinetics consistent with non-clinical data; Phase 1 enrollment and dosing in healthy volunteers ongoing with target engagement data expected in H1 2026, followed by inclusion of a patient cohort
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