What ProQR Therapeutics N.V. does.

We are a clinical-stage biotechnology company advancing a pipeline ( Figure 1 ) of RNA editing therapeutics based on our proprietary Axiomer RNA-editing platform. Our lead program, AX-0810 for cholestatic diseases targeting na-taurocholate cotransporting polypeptide (“NTCP”), is currently being evaluated in a Phase 1 clinical trial in healthy volunteers. With funding from Rett Syndrome Research Trust (“RSRT”), we are also advancing AX-2402 targeting methyl CpG binding protein 2 (“MECP2”) mutations for Rett syndrome, a severe neurodevelopmental disorder. Additional pipeline programs include AX-2911 targeting PNPLA3 for metabolic dysfunction-associated steatohepatitis (“MASH”), AX-1412 targeting the B4GALT1 gene for cardiovascular diseases (“CVDs”), as well as a number of additional discovery-stage programs.

Our robust pipeline is strategically focused on diseases originating in the liver and central nervous system (“CNS”), where human genetics and translational research support the potential for therapeutic benefit through RNA editing. We prioritize indications with high unmet medical need and biomarkers and well-defined clinical endpoints.

These programs are enabled by Axiomer, our proprietary RNA editing platform technology designed to harness endogenous adenosine deaminase acting on RNA (“ADAR”) enzymes to mediate precise, single nucleotide edits in RNA with high specificity and durability. Axiomer uses editing oligonucleotides (“EONs”) designed to recruit and direct ADARs to change an adenosine (A) to an inosine (I) in target RNA sequences where an inosine is translated as a guanosine (G). This approach can be used to correct a messenger RNA (“mRNA”) with a disease-causing mutation back to a normal (or wild type) mRNA, modulate protein expression, or alter a protein so that it will have a new function that helps prevent or treat disease.

Revenue and profit, by fiscal year.

More on PRQR.

• ProQR Therapeutics N.V. summary
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Figures from ProQR Therapeutics N.V.'s filings with the U.S. SEC, standardised automatically; prices are end-of-day closes. Not financial advice.