ProQR Announces Encouraging AX-0810 Phase 1 Safety and PK Data, Development Candidate Selections, and 2026 Outlook
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 cohortDevelopment Candidates selected for pipeline programs AX-2402 for Rett syndrome (MECP2, R270X) and AX-2911 for MASH (PNPLA3)Strategic collaboration with Eli Lilly achieved $4.5 million in milestones in 2025, contributing to strong financi ...