Eton Pharmaceutcials, Inc. Common Stock
Eton Pharmaceuticals, Inc., a pharmaceutical company, focuses on developing and commercializing treatments for rare diseases. Its commercial rare disease products include Increlex for the treatment of severe primary igf-1 deficiency; Alkindi Sprinkle for adrenal insufficiency; Galzin for Wilson disease; PKU Golike for phenylketonuria; Carglumic Acid for N-acetylglutamate synthase deficiency; Betaine Anhydrous for homocystinuria; and Nitisinone for tyrosinemia type 1. The company is also developing various product candidates, which are in late-stage development, including ET-400 for treating adrenal insufficiency; ET-600 for diabetes insipidus; Amglidia for neonatal diabetes mellitus; ET-700 for Wilson disease; ET-800 for adrenal insufficiency; and ZENEO hydrocortisone autoinjector for adrenal crisis. Eton Pharmaceuticals, Inc. was incorporated in 2017 and is based in Deer Park, Illinois.
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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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