REGENXBIO Inc.
REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapies that deliver functional genes to cells with genetic defects in the United States. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's products in pipeline includes ABBV-RGX-314 for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases; and RGX-202, which is in Phase III clinical trial for the treatment of Duchenne muscular dystrophy, as well as RGX-121 for the treatment of mucopolysaccharidosis type II. It also develops RGX-111 for the treatment of mucopolysaccharidosis type I, RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2, and RGX-381 for the treatment of the ocular manifestations of CLN2 disease. In addition, the company licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies. Further, it has a collaboration and license agreement with AbbVie Global Enterprises Ltd. to develop ABBV-RGX-314 outside the United States. REGENXBIO Inc. was incorporated in 2008 and is headquartered in Rockville, Maryland.
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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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