What is RNA and how can we use the body's own RNA editing machinery to treat disease? Learn more about the Axiomer® RNA editing technology and how we are creating a new class of medicines.
ProQR invented an entirely new way to treat genetic diseases. Axiomer enables RNA base editing by recruiting ADAR so your own body can treat disease. Learn more here.
Modification of a guide RNA can mimic the effect of hyperactive mutants and advance the approach of recruiting endogenous ADARs for site-directed RNA editing reported in Journal of the American Chemical Society (JACS) by Doherty EE et al.
… Lilly and ProQR to Expand RNAEditing Collaboration New agreement supports the discovery … in September 2021, applied ProQR’s proprietary Axiomer® RNAediting platform to target disorders of the liver and …
ProQR will focus exclusively on the development of the Axiomer® RNA editing technology platform across multiple therapeutic areas. This comes following feedback from the European Medicines Agency (EMA) related to ProQR’s sepofarsen for Leber Congenital Amaurosis type 10 (LCA10).
