ProQR Therapeutics N.V. Ordinary Shares
ProQR Therapeutics N.V., a biotechnology company, focuses on the discovery and development of novel therapeutic medicines. The company's products pipeline includes AX-0810 for cholestatic diseases targeting Na-taurocholate cotransporting polypeptide (NTCP); and AX-1412 for cardiovascular diseases (CVDs) targeting Beta-1,4-galactosyltransferase 1 (B4GALT1). It also develops various other early-stage research programs, including AX-1005 for undisclosed targets in CVDs; AX-2402, which focuses on Rett syndrome; AX-2911 for metabolic dysfunction-associated steatohepatitis (MASH) targeting patatin-like phospholipase domain-containing protein 3 (PNPLA3); AX-0601 for obesity and Type 2 diabetes; and AX-9115 for rare metabolic condition, as well as additional programs in Rett Syndrome, additional CNS programs, and multiple other unnamed targets and programs. In addition, the company develops Axiomer RNA base-editing platform technology. It has a license agreement with Radboud University Medical Center; Inserm Transfert SA; Ionis Pharmaceuticals, Inc.; Vico Therapeutics B.V.; University of Rochester; and Leiden University Medical Center, as well as license and research collaboration with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The company was incorporated in 2012 and is headquartered in Leiden, the Netherlands.
Open PRQR dashboard ↗Rocket Pharmaceuticals, Inc. Common Stock
Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases in the United States. It develops vivo adeno-associated viral (AAV) programs, which include programs for Danon disease (DD), a multi-organ lysosomal-associated disorder leading to early death due to heart failure (RP-A501), which is in Phase 2 trial; Plakophilin-2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), an inheritable cardiac disorder that is characterized by a progressive loss of cardiac muscle mass, severe right ventricular dilation, dysplasia, fibrofatty replacement of the myocardium and a high propensity to arrhythmias and sudden death (RP-A601), which is in phase 1; and BAG3 Dilated Cardiomyopathy (BAG3-DCM), which is a form of cardiomyopathy and is characterized by progressive thinning of the walls of the heart, which is in preclinical stage. It also develops vivo lentiviral (LV) programs, which include programs for Leukocyte Adhesion Deficiency-I (LAD-I), a genetic disorder that causes the immune system to malfunction (RP-L201); Fanconi Anemia (FA), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells (RP-L102); and Pyruvate Kinase Deficiency (PKD), a red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia (RP-L301). It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; REGENXBIO, Inc.; and Temple University. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.
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