… molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer ™ EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA …
… | New Orleans, Louisiana, USA Oral Presentation Title: ADAR-Mediated RNA Editing of SLC10A1(NTCP) as a Therapeutic … Editing-Based Correction of PNPLA3 I148M Functionality to Address Hepatic Steatosis (abstract 1654) Presenter: Gerard …
… Quarter 2025 Operating and Financial Results Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases …
… Marks the first submitted Clinical Trial Application (CTA) advancing ProQR’s Axiomer™ ADAR-mediated RNA editing platform into clinical development … Agency (EMA) to initiate a Phase 1 clinical trial of its lead pipeline program AX-0810 targeting NTCP, a liver cell …
… we exclusively focused our strategy on RNA editing, ProQR made important progress with Axiomer, our ADAR-mediated RNA editing technology platform,” said Daniel … protein data with the liver target ANGPTL3 in mice. Additionally, we further strengthened our leading global IP …
Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1; initiation of clinical trials anticipated in late 2024/early 2025
Axiomer activity demonstrated across multiple preclinical in vitro
… the event, ProQR will highlight its proprietary Axiomer™ ADAR-mediated RNA editing platform, along with updates on its … join the analyst Q&A session with the Management Team. Additionally, the following speakers will present at the …
… Results Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and … areas Strength of intellectual property estate and leading IP position supported with successful defense of opposition against a key Axiomer patent protecting ADAR-mediated RNA editing Well-capitalized with €139 M, in …
