Workflow
ProQR Highlights Upcoming Presentation at 20th Annual Meeting of the Oligonucleotide Therapeutics Society
PRQRProQR(PRQR) GlobeNewswire News Room·2024-10-07 12:00

Core Insights - ProQR Therapeutics announced the presentation of preclinical data for its Axiomer™ RNA editing technology and AX-0810 pipeline program targeting cholestatic diseases at the 20th Annual Meeting of the Oligonucleotide Therapeutics Society [1][2] Group 1: Axiomer™ Technology - Axiomer™ is a next-generation RNA base editing technology that allows for specific single nucleotide changes in RNA, potentially leading to new medicines for various diseases [3][4] - The technology utilizes the human cell's own ADAR machinery to convert Adenosine (A) to Inosine (I), which is then translated as Guanosine (G), effectively correcting disease-causing mutations [3] Group 2: Pipeline and Therapeutic Focus - The AX-0810 program specifically targets NTCP to lower bile acid reuptake in cholestatic diseases, with a presentation scheduled for October 8, 2024 [2] - ProQR aims to address both rare and prevalent diseases with unmet medical needs through its proprietary RNA repair platform [4]