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AZN vs RARE

AstraZeneca PLC and Ultragenyx Pharmaceutical Inc.

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AstraZeneca PLC

AstraZeneca PLC, a biopharmaceutical company, focuses on the discovery, development, manufacture, and commercialization of prescription medicines. The company offers Imjudo, Datroway, Iressa, Tagrisso, Imfinzi, Lynparza, Calquence, Enhertu, Orpathys, Truqap, Zoladex, Faslodex, Farxiga, Brilinta, Crestor, Seloken, Lokelma, Roxadustat, Andexxa, Wainua, Symbicort, Fasenra, Pulmicort, Breztri/Trixeo, Saphnelo, Tezspire, Airsupra, Ultomiris, Soliris, Strensiq, Koselugo, Kanuma, Atacand, Plendil, Modip, Splendil, Munobal, Flodil, Komboglyze, Qtern, Byetta, XIGDUO, Accolate, Accoleit, Vanticon, Beyfortus, Synagis, FluMist, tixagevimab and cilgavimab, and sipavibart. It also provides Bricanyl Respules, Eklira Genuair/Tudorza/Bretaris, Bricanyl Turbuhaler, Rhinocort, Bevespi Aerosphere, Daliresp/Daxas, Oxis Turbuhaler, Breztri Aerosphere, Duaklir Genuair, Kavigale, Evusheld, Fluenz/FluMist, and Voydeya. The company offers its products for ocology, cardiovascular, renal and metabolism, respiratory & immunology, vaccines and immune, and therapies rare diseases. It serves primary and specialty care physicians through distributors and local representative offices in the United Kingdom, the United States, Europe, and Asia. It has a strategic agreement with Tempus to develop the largest multimodal foundation model in oncology. The company has a collaboration agreement with IonQ, Inc. for the development of quantum-accelerated computational chemistry workflow for healthcare, life sciences, and chemistry; and a strategic research collaboration with CSPC Pharmaceutical Group Limited to advance the discovery and development of novel oral candidates, with the potential to treat diseases across multiple indications. AstraZeneca PLC was formerly known as Zeneca Group PLC and changed its name to AstraZeneca PLC in April 1999. AstraZeneca PLC was incorporated in 1992 and is headquartered in Cambridge, the United Kingdom.

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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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