ProQR Therapeutics

ProQR Therapeutics is a clinical-stage biotechnology company founded in 2012, dedicated to developing transformative RNA therapies for severe genetic diseases. The company's primary focus is on its proprietary Axiomer RNA editing technology platform, which uses a cell's own editing machinery called ADAR (Adenosine Deaminase Acting on RNA) to make specific single nucleotide edits in RNA. This approach aims to reverse mutations, modulate protein expression, or alter protein function to prevent or treat diseases. ProQR's lead programs are designed for the treatment of cholestatic diseases and cardiovascular disease. The company's technology has shown promise in preclinical studies, demonstrating robust editing efficiency and durability in non-human primates. In April 2024, ProQR presented data on its AX-0810 program targeting NTCP for cholestatic diseases, including hepatic editing in non-human primates with strong correlating impact on disease-relevant biomarkers. ProQR's pipeline includes programs spanning discovery and (pre-)clinical stages in both medical devices and drug discovery.

Key customers and partnerships

ProQR has established significant partnerships to advance its RNA editing technology. In September 2021, the company entered into a licensing and collaboration agreement with Eli Lilly and Company to discover, develop, and commercialize new genetic medicines. This partnership was expanded in December 2022, with Lilly gaining access to additional targets in the central nervous system and peripheral nervous system using ProQR's Axiomer platform. As part of this expanded agreement, ProQR received USD 75 million, consisting of an upfront payment and an equity investment. The collaboration with Lilly makes ProQR eligible to receive up to approximately USD 3.75 billion in research, development, and commercialization milestones, as well as tiered royalties of up to mid-single digit percentage on product sales. ProQR has also partnered with leading academic researchers through its Immunology Innovation Program (IIP) to translate immunology breakthroughs into novel antibody-based medicines.