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ProQR Highlights New Platform Data from Presentation on Axiomer RNA Editing Technology at Deaminet 2024

Author: Benzinga Newsdesk | January 19, 2024 08:05am

Platform demonstrates robust in vivo editing capabilities reporting up to 70% editing of ACTB in the liver of non-human primates (NHPs) and mice
Functional effect demonstrated in mice in vivo via modulation of ANGPLT3 protein properties leading to favorable increase in LPL enzymatic activity and meaningful impact of biomarkers
Additional data to be presented throughout 2024, including in vitro and in vivo data for pipeline programs AX-0810 for Cholestatic diseases targeting NTCP and AX-1412 for Cardiovascular diseases targeting B4GALT1

Posted In: PRQR