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JAZZ vs RGNX

Jazz Pharmaceuticals, Inc. and REGENXBIO Inc.

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Jazz Pharmaceuticals, Inc.

Jazz Pharmaceuticals plc identifies, develops, and commercializes pharmaceutical products in the United States, Europe, and internationally. The company offers Xywav to treat cataplexy or excessive daytime sleepiness (EDS) with narcolepsy and idiopathic hypersomnia (IH); Xyrem for the treatment of cataplexy or EDS with narcolepsy; Epidiolex for seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC); Rylaze for the treatment of acute lymphoblastic leukemia or lymphoblastic lymphoma; Enrylaze to treat acute lymphoblastic leukemia and lymphoblastic lymphoma; Zepzelca for the treatment of metastatic small cell lung cancer with disease progression on or after platinum-based chemotherapy; Ziihera to treat HER2-positive biliary tract cancers; Defitelio to treat severe veno-occlusive disease; and Vyxeos for the treatment of newly-diagnosed therapy-related acute myeloid leukemia. It also develops Zanidatamab in Phase 3 trial to treat HER2-expressing gastroesophageal adenocarcinoma (GEA), and patients with HER2-expressing metastatic GEA; Epidiolex in Phase 3 trial to treat LGS, DS, and TSC; Suvecaltamide in Phase 2 trial to treat Parkinson's disease tremor; JZP815, a pan-RAF kinase inhibitor in Phase 1 trial that targets components of the mitogen-activated protein kinase; JZP898, a conditionally activated interferon alpha molecule in Phase 1 trial; JZP441 in Phase 1 trial to treat narcolepsy, IH, and other sleep disorders; JZP324, an extended-release low sodium oxybate formulation in Phase 1 trial for patients with sleep disorders; and JZP150 to treat post-traumatic stress disorder. The company has licensing and collaboration agreements with Redx Pharma plc, Autifony Therapeutics Limited, Zymeworks Inc., Sumitomo Pharma Co., Ltd., and Werewolf Therapeutics, Inc. Jazz Pharmaceuticals plc was founded in 2003 and is headquartered in Dublin, Ireland.

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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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