ProQR Therapeutics N.V. (NASDAQ: PRQR)

ProQR Therapeutics N.V., a biotechnology company publicly traded under the symbol PRQR, is dedicated to developing innovative RNA therapies for severe rare and common diseases. The company's primary business activities revolve around advancing its proprietary Axiomer and Trident RNA-editing platform technologies to develop novel RNA editing therapeutics. ProQR's operations span across various countries, with a specific focus on liver-originating and central nervous system (CNS) diseases.

ProQR generates revenue by leveraging its Axiomer and Trident RNA editing platform technologies to develop Editing Oligonucleotides (EONs). These EONs are designed to recruit and direct endogenously expressed ADAR enzymes to make single adenosine-to-inosine (A-to-I) changes in the RNA in a highly specific and targeted manner. The company's product pipeline includes AX-0810 for Cholestatic Diseases targeting NTCP and AX-1412 for Cardiovascular Disease (CVD) targeting B4GALT1.

Within the industry, ProQR competes with large pharmaceutical, biotechnology, specialty pharmaceutical, and generic drug companies, academic institutions, government agencies, and research institutions. However, ProQR possesses a competitive advantage in its RNA editing technologies, Axiomer and Trident, which enable single nucleotide changes to RNA in a highly specific and targeted way. Additionally, ProQR's pipeline programs target a wide range of diseases, including cholestatic diseases, CVD, and Rett syndrome, setting it apart from competitors.

ProQR's customers include pharmaceutical companies, research institutions, and patients suffering from various diseases. In January 2024, ProQR announced a partnership with the Rett Syndrome Research Trust (RSRT) to develop EONs targeting an underlying genetic variant that causes Rett syndrome, a rare neurodevelopment disorder. Furthermore, ProQR has entered into a global licensing and research collaboration with Eli Lilly and Company, focusing on the discovery, development, and commercialization of potential new medicines for genetic disorders using its Axiomer RNA editing technology.

ProQR's product portfolio includes AX-0810 for Cholestatic Diseases targeting NTCP and AX-1412 for Cardiovascular Disease (CVD) targeting B4GALT1. AX-0810 aims to reduce the reuptake of bile acids in the liver by inhibiting NTCP function, potentially reducing the accumulation of toxic bile acids in the liver. Meanwhile, AX-1412 represents a potential targeted approach to RNA editing of B4GALT1, leading to a promising strategy for protecting against CVDs by simultaneously lowering levels of LDL-c and fibrinogen.

References:
ProQR Therapeutics N.V. (2023). ProQR Therapeutics N.V. Form 10-K for the fiscal year ended December 31, 2023. U.S. Securities and Exchange Commission.