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Omeros Corporation and Ultragenyx Pharmaceutical Inc.

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Omeros Corporation

Omeros Corporation, a clinical-stage biopharmaceutical company, discovers, develops, and commercializes small-molecule and protein therapeutics, and orphan indications targeting immunologic diseases. The company's lead products candidate is the Narsoplimab (OMS721/MASP-2) for the treatment of hematopoietic stem-cell transplant-associated thrombotic microangiopathy (TA-TMA); and in Phase II clinical trial to treat COVID-19 and acute respiratory distress syndrome. It also develops OMS1029 that has completed phase I clinical trials for mannan-binding lectin-associated serine protease 2 (MASP-2) and lectin pathway disorders; zaltenibart that is in phase 3 clinical trials for Paroxysmal nocturnal hemoglobinuria; in phase 2 clinical trial for the treatment complement 3 glomerulopathy, and other alternative pathway disorders; and OMS527 that is in phase I clinical trials for addictions and compulsive disorders, and movement disorders, as well as cocaine use disorder. In addition, the company's products under preclinical development comprise MASP-2, a pro-inflammatory protein target for the treatment of lectin pathway disorders; MASP-3 small-molecule inhibitors for alternative pathway disorders; and Adoptive T-Cell and Immunostimulator Combination Therapies, and Oncotoxins and Immunomodulators for the treatment of various cancers. Omeros Corporation was incorporated in 1994 and is headquartered in Seattle, Washington.

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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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