Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
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Zai Lab Limited, a biopharmaceutical company, focuses on discovering, developing, and commercializing products that address medical conditions in the areas of oncology, immunology, neuroscience, and infectious diseases. Its commercial products include Zejula, an orally administered poly (ADP-ribose) polymerase 1/2 inhibitor for treating ovarian cancer; VYVGART, a human IgG1 antibody fragment for generalized myasthenia gravis and chronic inflammatory demyelinating polyneuropathy; NUZYRA for community-acquired bacterial pneumonia and acute bacterial skin and skin structure infections; Optune for glioblastoma multiforme; Qinlock for gastrointestinal stromal tumors; Xacduro for treating hospital-acquired and ventilator-associated bacterial pneumonia caused by a cinetobacter baumannii-calcoaceticus complex; and Augtyro for the treatment of ROS proto-oncogene 1 and non-small cell lung cancer. The company's oncology pipeline includes bemarituzumab, tumor treating fields, tisotumab vedotin, and repotrectinib; and immunology, neuroscience, and infectious disease pipeline comprises efgartigimod and xanomeline and trospium chloride. Zai Lab Limited has license and collaboration agreements with Tesaro, Inc. to develop, manufacture, and commercialize niraparib; NovoCure Ltd. to develop and commercialize Tumor Treating Fields; Deciphera Pharmaceuticals, LLC to develop and commercialize ripretinib; Paratek Bermuda Ltd. to develop, manufacture, and commercialize omadacycline; argenx BV to develop and commercialize efgartigimod; Bristol-Myers Squibb Company to develop and commercialize tisotumab vedotin and repotrectinib; Amgen Inc. to develop and commercialize bemarituzumab; Innoviva, Inc. to develop and commercialize Sulbactam-Durlobactam; and Karuna Therapeutics, Inc. to develop and commercialize Xanomeline and trospium chloride, as well as strategic collaboration with Pfizer Inc. for sulbactam and durlobactam. The company was incorporated in 2013 and is headquartered in Pudong, China.
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