… in mice in vivo via modulation of ANGPLT3 protein properties leading to favorable increase in LPL enzymatic … lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today … the potential of Axiomer EONs in preparation for clinical development and supporting discovery of potential new …
… Research Partnership and EB Medical Research Foundation to Develop QR-313 for the Treatment of Dystrophic Epidermolysis Bullosa The funding will be used for the clinical development of QR-313 Clinical trial in DEB patients to start …
… are no therapies commercially available or in clinical development for this disease. QR-110 has received fast track … granted orphan drug designation in the United States and European Union. Interim safety and efficacy trial results … that the first patient has been dosed in the Phase 1/2 open-label trial assessing the safety, tolerability, …
Advancing AX-0810 through ongoing Phase 1 dosing, with target engagement data expected 1H 2026Selected Development Candidates AX-2402 for Rett syndrome (MECP2, R270X) and AX-2911 for MASH (PNPLA3)Ended 2025 with € 92.4 million cash and cash equivalents and achie
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
