Pipeline ProQR Pipeline
ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Our initial discovery programs aim to explore the potential of our platform across diseases that originate in the liver and nervous system.
ProQR pipeline
Our pipeline programs share several key characteristics including a deep rooting in human genetics, the potential to have a major impact in a high unmet medical need, the ability to leverage the existing proven delivery technology for delivery to hepatocytes in liver, the opportunity to monitor early biomarkers to establish target engagement in Phase I trials for human proof of concept and availability of well-defined clinical endpoints.
Internal link ProQR pipeline
RNA therapy
An RNA therapy is designed to correct the mistake, or mutation, in the RNA of someone with a disease. By correcting the mistake, the RNA can then be used to create the protein that the cell needs, taking away the underlying cause of the disease.
Internal link RNA therapy
AX-0810 cholestatic diseases (NTCP)
At ProQR, we are dedicated to improving the lives of patients and families affected by rare and common disorders using Axiomer, our innovative RNA editing platform technology.
Internal link AX-0810 cholestatic diseases (NTCP)