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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Xencor, Inc., a clinical-stage biopharmaceutical company, focuses on the discovery and development of engineered monoclonal antibodies for the treatment of asthma and allergic diseases, autoimmune diseases, and cancer. The company provides Ultomiris to treat patients with atypical hemolytic uremic syndrome, generalized myasthenia gravis, neuromyelitis optica spectrum disororder; and Monjuvi for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma; and Sotrovimab that targets the SARS-CoV-2 virus. It develops XmAb819, a bispecific antibody to treat renal cell carcinoma; XmAb541, which is in Phase I for the treatment of ovarian cancer; XmAb808, a bispecific antibody that binds to the broadly expressed tumor antigen; Vudalimab, which is in Phase II clinical trial to treat metastatic castration-resistant prostate cancer; XmAb564, to treat autoimmune diseases; XmAb662, which is in Phase I clinical trial to treat patients with solid tumors; XmAb942, which is in Phase I clinical trial for patients with Crohn's disease and ulcerative colitis; and Plamotamab, a bispecific antibody, which is in Phase I clinical trial to treat lymphoma. Further, the company develops Xaluritamig, a bispecific T-cell engager that treats prostate cancer; Obexelimab, to treat patients with immunoglobulin G4-related disease; Teropavimab and zinlirvimab to treat human immunodeficiency virus; Tobevibart, a treatment for hepatitis Delta virus infection; Novartis, an antibody drug candidate that uses XmAb Fc technologies; Xpro1595 to treat patients with Alzheimer's disease and treatment-resistant depression; Zaltenibart to treat paroxysmal nocturnal hemoglobinuria and other alternative pathway disorders; ASP2138 to treat gastric, gastroesophageal junction, and pancreatic cancers; JNJ-9401 to treat prostate cancer; and JNJ-1493 to treat B-cell malignancies. The company was incorporated in 1997 and is headquartered in Pasadena, California.
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