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 are rooted in human genetics and address high unmet medical needs, leveraging proven delivery technologies. They also enable early biomarker assessment in Phase I trials to demonstrate target engagement, human proof of concept, and 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/AX-0811 cholestatic diseases (NTCP)
AX-0810 and AX-0811 are investigational RNA editing therapies targeting NTCP for the treatment of cholestatic diseases. These diseases are characterized by excess bile acids in liver cells. AX-0810 and AX-0811 are designed to create a version of the NTCP protein which allows less bile acids into liver cells.
Internal link AX-0810/AX-0811 cholestatic diseases (NTCP)
AX-0422 Hurler syndrome (IDUA)
AX-0422 is an investigational RNA editing oligonucleotide being developed as a potential therapy for MPS I Hurler syndrome (MPS IH, or Hurler syndrome). It is designed to correct a disease-causing mutation in the IDUA gene.
Internal link AX-0422 Hurler syndrome (IDUA)