ProQR Doses First Participant in Phase 1 Study of AX-0811
核心洞察
ProQR Therapeutics announced that the first participant has been dosed in the Phase 1 clinical trial of AX-0811 (搜索) in healthy volunteers.
AX-0811 (搜索) is an AI-discovered RNA editing oligonucleotide designed to modulate NTCP (搜索), with preclinical data showing greater potency and longer half-life than AX-0810.
The company will host a virtual investor and analyst event on September 30, 2026, on NTCP (搜索) modulation in biliary atresia (搜索) via liver-targeted RNA editing.
ProQR Therapeutics N.V. (Nasdaq: PRQR) has dosed the first participant in the Phase 1 clinical trial of AX-0811 (搜索) in healthy volunteers. AX-0811 is the company's next-generation, AI-discovered investigational RNA editing oligonucleotide (EON) designed to modulate NTCP (搜索), with preclinical data demonstrating greater potency and a longer half-life compared with AX-0810. It is being developed as a potential disease-modifying treatment for cholestatic liver diseases (搜索), including biliary atresia (搜索).
The Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic biomarkers of AX-0811 (搜索) in healthy volunteers, and builds on ProQR's clinical experience with AX-0810. Target engagement data for the first two cohorts are expected in early January 2027. The candidate is based on ProQR's proprietary ADAR (搜索)-mediated Axiomer RNA editing technology platform, in which editing oligonucleotides recruit endogenous ADAR to change adenosine to inosine in RNA.
ProQR will also host a virtual investor and analyst educational event, "NTCP (搜索) Modulation in Biliary Atresia (搜索) via Liver-Targeted RNA Editing," on September 30, 2026 at 11 am ET. The event will review the role of bile acids and NTCP modulation in cholestatic liver disease, the recently reported AX-0810 Phase 1 target engagement data, and the translational rationale and clinical development strategy for NTCP modulation in biliary atresia, including the framework for evaluating upcoming AX-0811 (搜索) Phase 1 data.
Source: ProQR Announces First Participant Dosed in Phase 1 Study of AX-0811