Find all relevant presentations and publications for each of our programs. From review papers to preclinical posters and clinical data presentations at medical conferences.
… ProQR Announces Axiomer® RNA Editing Licensing and Research Collaboration … directed to up to five targets u sing ProQR’s proprietary Axiomer ® RNA editing platform ProQR to receive $ 5 0 million …
Partnership focused on utilizing Axiomer™ to develop editing oligonucleotides targeting an underlying genetic variant that causes Rett syndromeCollaboration expands ProQR’s commitment to advance the development of therapies for central nervous system diseases
Platform demonstrates robust in vivo editing capabilities reporting up to 70% editing of ACTB in the liver of non-human primates (NHPs) and miceFunctional effect demonstrated in mice in vivo via modulation of ANGPLT3 protein properties leading to favora
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
