ProQR Therapeutics N.V. (PRQR)

Overview

ProQR Therapeutics N.V. is a biotechnology firm established in 2012, with its headquarters located in Leiden, the Netherlands. This company specializes in the discovery and development of innovative therapeutic medicines aimed at addressing a wide range of diseases, particularly focusing on genetic disorders affecting the liver and nervous system. ProQR Therapeutics engages in extensive research to create effective treatments, leveraging its partnerships with esteemed institutions and companies such as Radboud University Medical Center, Inserm Transfert SA, Ionis Pharmaceuticals, Inc., Vico Therapeutics B.V., University of Rochester, Leiden University Medical Center, and Eli Lilly and Company. These collaborations are centered on the discovery, development, and commercialization of potentially new medications for genetic disorders, showcasing the company's commitment to advancing medical science and improving patient outcomes.

Products and Services

• AX-0810 for Cholestatic Diseases

  Targeting the Na-taurocholate cotransporting polypeptide (NTCP), AX-0810 is a therapeutic candidate aimed at treating cholestatic diseases. This development demonstrates ProQR's venture into addressing liver-related diseases through innovative molecular targeting.

• AX-1412 for Cardiovascular Diseases (CVDs)

  With a focus on Beta-1,4-galactosyltransferase 1 (B4GALT1), AX-1412 represents ProQR's efforts in combating cardiovascular diseases. The targeting strategy of B4GALT1 indicates a novel approach in the therapeutic landscape of CVDs.

• AX-1005 for Undisclosed Targets in CVDs

  AX-1005 is another of ProQR's contributions to cardiovascular disease treatment, though its specific targets remain undisclosed. This project reflects the company's ongoing research and dedication to uncovering new paths in CVD therapy.

• AX-2402 for Rett Syndrome

  This early-stage research program focuses on developing a treatment for Rett Syndrome, a rare genetic neurological disorder, indicating ProQR's commitment to addressing rare diseases with high unmet medical needs.

• AX-2911 for Nonalcoholic Steatohepatitis (NASH)

  AX-2911 is aimed at treating Nonalcoholic Steatohepatitis (NASH), a liver disease that has been increasing globally. This aligns with ProQR's theme of targeting liver diseases through innovative therapeutic approaches.

• AX-0601 for Obesity and Type 2 Diabetes

  This program targets the growing global health issues of obesity and Type 2 diabetes, showcasing ProQR's venture into metabolic disease treatment with a focus on widespread conditions.

• AX-9115 for Rare Metabolic Condition

  Targeting a rare metabolic condition, AX-9115 embodies ProQR's dedication to developing treatments for rare diseases, reflecting the company's broader mission of addressing underserved medical areas.

• Axiomer RNA Base-Editing Platform Technology

  ProQR is also at the forefront of genetic editing with its Axiomer RNA base-editing platform technology. This innovative approach underpins the company's product pipeline, highlighting a commitment to cutting-edge science in the treatment of genetic disorders.