ProQR Announces First Participant Dosed in Phase 1 Study of AX-0811 and Virtual Investor and Analyst Educational Event on September 30, 2026
ProQR dosed the first participant in the Phase 1 clinical trial of AX-0811, a next-generation, AI-discovered RNA editing oligonucleotide designed to modulate NTCP, in healthy volunteers. AX-0811 is being developed as a potential disease-modifying treatment for cholestatic liver diseases, including biliary atresia, with preclinical data showing greater potency and a longer half-life than predecessor AX-0810. Target engagement data from the first two Phase 1 cohorts is expected in early January 2027, making that the next hard catalyst for the program.
Key figures
- Drug Name
- AX-0811
- Phase Of Trial
- Phase 1
- Target
- NTCP
- Indication
- cholestatic liver diseases, including biliary atresia
- Preclinical Claim
- greater potency and longer half-life vs AX-0810
- Data Readout Detail
- Phase 1 target engagement data for the first two cohorts of healthy volunteers
- Data Readout Timing
- early January 2027
- Investor Event Date
- September 30, 2026, 11am ET
AI analysis
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