ProQR Therapeutics has dosed the first participant in its AX-0811 early-stage study of an RNA editing treatment candidate targeting NTCP. The milestone starts evaluation of safety, tolerability, pharmacokinetics and pharmacodynamic biomarkers, with initial target-engagement data expected in early January 2027.

Key Highlights

  • AX-0811 is designed to modulate NTCP and showed greater potency and a longer half-life than AX-0810 in preclinical studies.
  • ProQR Therapeutics has dosed the first participant in the AX-0811 clinical study.
  • The study will assess safety, tolerability, pharmacokinetics and pharmacodynamic biomarkers in healthy volunteers.
  • AX-0811 is an AI-discovered RNA editing oligonucleotide designed to modulate NTCP for potential treatment of cholestatic liver diseases, including biliary atresia.

ProQR Therapeutics N.V. (NASDAQ: PRQR) has dosed the first participant in its AX-0811 clinical study, advancing the RNA editing candidate into testing in healthy volunteers. The study will examine safety, tolerability, pharmacokinetics and pharmacodynamic biomarkers for a potential treatment targeting cholestatic liver diseases, including biliary atresia.

AX-0811 Moves Into Healthy-Volunteer Testing

AX-0811 is an AI-discovered investigational RNA editing oligonucleotide designed to modulate NTCP. ProQR said preclinical work showed greater potency and a longer half-life for AX-0811 compared with AX-0810.

The program will focus on NTCP modulation in biliary atresia through liver-targeted RNA editing and will include presentations from ProQR management and Gideon Hirschfield.