AX-0810 targeting NTCP for cholestatic diseases to be first ProQR Axiomer™ RNA editing program to enter the clinic Company to host virtual Investor and Analyst Event in December to highlight translational data and clinical plans for AX-0810, as well as AX-1412, targeting
First-in-human trial of AX-0810 will evaluate safety, tolerability, pharmacokinetics, and target engagement in healthy volunteers with initial data expected in Q4 2025Marks the first submitted Clinical Trial Application (CTA) advancing ProQR’s Axiomer™ ADAR-mediated RNA
Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1
Development of Axiomer® RNA base editing technology platform continues across multiple therapeutic areas
Stre
Post-hoc analyses from Illuminate trial of sepofarsen demonstrate an encouraging efficacy signal when comparing active treatment and sham eyes to their corresponding contralateral eyes across multiple endpoints – Company plans to discuss findings with regulators in Q3