Fulcrum Therapeutics, Inc. Common Stock
Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops small molecules to improve the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its clinical-stage product candidate is pociredir, a fetal hemoglobin inducer, which is in phase 1b clinical trial for the treatment of sickle cell disease and beta-thalassemia. The company also discovers drug targets for potential treatment inherited aplastic anemias, such as of diamond-blackfan anemia, shwachman-diamond syndrome, and fanconi anemia, as well as novel fetal hemoglobin inducers, fibrotic disorders, and cardiomyopathies which is in preclinical trial. It has collaboration and license agreement with MyoKardia, Inc. to identify biological targets that are capable of modulating genes of interest with relevance to cardiomyopathies; and licensce agreement with CAMP4 which includes certain small molecule compounds, composition of matter and method of use patent rights, and know-how to research, develop, manufacture, use, and commercialize; and Sanofi for the development and commercialization of losmapimod, an oral small molecule being investigated for the treatment of facioscapulohumeral muscular dystrophy. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
Open FULC dashboard ↗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 ↗