ProQR Therapeutics NV is a clinical stage biopharmaceutical company, which engages in the discovery and development of RNA therapies for the treatment of severe genetic rare diseases with a focus on inherited retinal diseases. The company is headquartered in Leiden, Zuid-Holland and currently employs 166 full-time employees. The company went IPO on 2014-09-18. The firm is engaged in the discovery and development of ribonucleic Acid (RNA)-based therapeutics for the treatment of severe genetic disorders. Main purpose of the Company is advancement of its Axiomer RNA-editing platform technology. That technology 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 unaddressed needs. The firm operates research pipeline that contains of RNA Therapy and AX-0810 Cholestatic Diseases (NTCP).
