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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Tonix Pharmaceuticals Holding Corp., a biopharmaceutical company, focuses on developing and commercializing therapeutics to treat and prevent human disease. It markets Zembrace SymTouch and Tosymra for the treatment of acute migraine with or without aura in adults. Its portfolio focuses on central nervous system disorders, as well as immunology, infectious disease, and rare disease product candidates. The company's products pipeline include TNX-102 SL (cyclobenzaprine HCl sublingual tablet), for the management of fibromyalgia; and is being developed to prevent and treat acute stress reaction, posttraumatic stress disorder, Alzheimer's disease, alcohol use disorder, and multi-site pain associated with Long COVID. It also develops TNX-1300, a double-mutant cocaine esterase, which is in Phase 2 clinical trials for the treatment of cocaine intoxication; and TNX-1900, an intranasal potentiated oxytocin for the treatment of binge eating disorder, adolescent obesity, and bone health in pediatric autism, as well as for social anxiety disorder. In addition, the company develops TNX-1500, an Fc-modified humanized monoclonal antibody indicated to prevent organ transplant rejection, as well as to treat autoimmune conditions; TNX-1700, a recombinant Trefoil Factor Family 2 fusion protein for the treatment of gastric and colorectal cancers; TNX-801 that is in the pre-IND development stage for protection against smallpox and mpox; and TNX-1800, a live virus vaccine for SARS-CoV-2. Its rare disease portfolio consists of TNX-2900, an intranasal potentiated oxytocin to treat for Prader-Willi syndrome. The company has collaboration agreement with Bilthoven Biologicals to advance TNX-801 and Makana Therapeutics, Inc. to study TNX-1500 for human-compatible organs and cells for the treatment of organ failure. Tonix Pharmaceuticals Holding Corp. is headquartered in Chatham, New Jersey.
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