ProQR Therapeutics Doses First Participant in Phase 1 Study of AI-Discovered RNA Editing Candidate AX-0811

The entry of an AI-discovered oligonucleotide into first-in-human dosing marks a measurable inflection point for RNA editing therapeutics, and for the CDMOs and specialty biologics facilities that will need to support them. ProQR Therapeutics announced on September 9, 2026 that the first healthy volunteer has been dosed in the Phase 1 clinical trial of AX-0811, an ADAR-mediated RNA editing oligonucleotide (EON) targeting NTCP modulation in cholestatic liver diseases, including biliary atresia.

AX-0811 is the successor to AX-0810, differentiated by greater potency and a longer half-life, both attributes identified through ProQR's AI-driven discovery process on its proprietary Axiomer™ platform. The Phase 1 study is evaluating safety, tolerability, pharmacokinetics, and pharmacodynamic biomarkers in healthy volunteers. Phase 1 target engagement data from the first two cohorts are expected in early January 2027, giving QA and clinical operations teams a defined readout window against which to align oversight activities.

For manufacturing and QA leads, the AX-0811 programme surfaces a set of process considerations that are still maturing across the industry. EONs occupy a distinct space between small-molecule oligonucleotides and larger biologics: they require sequence-specific synthesis controls, rigorous impurity profiling, and stability programmes calibrated to the longer half-life ProQR is reporting preclinically. Facilities handling similar modalities will be tracking how ProQR's clinical data package addresses ICH Q10-aligned process knowledge management as the programme scales from Phase 1 into later development.

The translational rationale for NTCP modulation in biliary atresia, a rare paediatric cholestatic disease with limited disease-modifying options, will be reviewed publicly at ProQR's virtual educational event on September 30, 2026, beginning at 11 am ET. The session will feature Gideon Hirschfield, Professor of Gastroenterology and Hepatology at the Toronto Centre for Liver Disease, alongside ProQR management. The agenda includes a review of AX-0810 Phase 1 target engagement data and the clinical development framework for evaluating AX-0811 data as it emerges.

Regulatory affairs leads monitoring the RNA editing space will note that ProQR's clinical experience with AX-0810 provides a prior safety and PK reference dataset, a meaningful asset when engaging agencies on first-in-human protocols for next-generation EONs under evolving guidance frameworks for novel modalities.

Phase 1 target engagement data from the first two AX-0811 cohorts, expected in January 2027, will serve as the first clinical signal against which the Axiomer™ platform's AI-driven potency predictions can be assessed in humans.

Source: ProQR Therapeutics via GlobeNewswire, September 9, 2026. Virtual educational event webcast scheduled September 30, 2026, 11 am–12 pm ET; registration available at lifescievents.com.