PRQR

ProQR Therapeutics is a clinical-stage biotechnology company developing RNA editing therapeutics based on its proprietary Axiomer platform, which uses editing oligonucleotides to recruit endogenous ADAR enzymes for precise adenosine-to-inosine edits in target RNA. The company's lead program AX-0810 for cholestatic diseases is in Phase 1 trials, while additional pipeline candidates include AX-2402 for Rett syndrome, AX-2911 for metabolic dysfunction-associated steatohepatitis (MASH), and AX-1412 for cardiovascular disease, with development focused on liver and central nervous system indications. ProQR also collaborates with Eli Lilly on an expanded platform partnership covering up to fifteen ADAR editing targets.

Pipeline

Catalyst Calendar