… targeting NTCP for cholestatic diseases to be first ProQR Axiomer™ RNA editing program to enter the clinic Company to … for cardiovascular diseases, and progress made across the Axiomer platform €89.4 million cash and cash equivalents as …
… transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced the … perspective in guiding the Company in the translation of Axiomer, our leading RNA editing platform technology, to the …
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
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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
… transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced the … seen firsthand the transformative potential of ProQR’s Axiomer RNA editing platform. I am excited to join the …
… it will present preclinical data for its proprietary Axiomer™ RNA editing technology platform and its AX-0810 … a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of …