ProQR Advances RNA Editing Pipeline with AX-0810 Data on Horizon — ProQR Therapeutics N.V.

Event summary

ProQR ended 2025 with €92.4M in cash, down from €149.4M in 2024, supported by $4.5M in Lilly collaboration milestones.
AX-0810 Phase 1 trial for cholestatic diseases progressing with target engagement data expected 1H 2026.
Selected development candidates AX-2402 for Rett syndrome and AX-2911 for MASH, with first-in-human testing for AX-2402 planned for 1H 2027.
Board composition changes announced with co-founder Dinko Valerio and Alison Lawton rotating off at next AGM.

The big picture

ProQR's progress reflects the growing competition in RNA editing therapies, where precision and speed to clinical validation are critical. The company's strategic focus on cholestatic diseases and Rett syndrome positions it in high-unmet-need markets, but its ability to sustain momentum depends on successful data readouts and efficient capital management. The board changes signal a shift toward long-term clinical-stage strategy as ProQR navigates its next phase of growth.

What we're watching

Clinical Milestones: Whether AX-0810 target engagement data in 1H 2026 will validate ProQR's RNA editing approach for cholestatic diseases.
Pipeline Execution: The pace at which ProQR advances AX-2402 for Rett syndrome into first-in-human trials by 1H 2027.
Financial Runway: How ProQR manages its cash position through mid-2027 amid increasing R&D spending and potential Lilly collaboration milestones.