argenx SE American Depositary Shares
argenx SE, a commercial-stage biopharma company, develops various therapies for the treatment of autoimmune diseases in the United States, Japan, China, the Netherlands, and internationally. The company offers VYGART and VYGART HYTRULO to treat generalized myasthenia gravis (gMG), immune thrombocytopenia (ITP), and chronic inflammatory demyelinating polyneuropathy (CIDP). It also develops efgartigimod for the treatment of seronegative and ocular gMG, thyroid eye diseases, ITP, myositis, Sjögren's disease, lupus nephropathy, systemic sclerosis, antibody-mediated rejection, and autoimmune encephalitis; empasiprubart for multifocal motor neuropath, CIDP, delayed graft function, and dermatomyositis; and ARGX-119 for congenital myasthenic syndrome, amyotrophic lateral sclerosis, and spinal muscular atrophy. In addition, the company is also developing ARGX-119, a MuSK agonist to treat congenital myasthenic syndromes and amyotrophic lateral sclerosis; ARGX-109, which targets IL-6 to treat inflammation; ARGX-121 and ARGX 220 that targets the immune system; ARGX-213, which targets FcRn; and ARGX-118, which develops antibodies against Galectin-10, as well as cusatuzumab, ARGX-112, ARGX-114, and ARGX-115. It has strategic collaborations with OncoVerity, Inc, LEO Pharma A/S, Zai Lab Limited, AbbVie, Inc., Elektrofi, Genmab SE, Chugai Pharmaceutical Co., Ltd., Clayton Foundation, Halozyme Therapeutics, Inc., AgomAb Therapeutics NV, Broteio Pharma B.V., VIB vzw, University of Texas, BioWa, Inc., Lonza Sales AG, Université Catholique de Louvain, Sopartec S.A., NYU Langone Health, Leiden University Medical Center, and IQVIA Ltd. argenx SE was incorporated in 2008 and is based in Amsterdam, the Netherlands.
Open ARGX dashboard ↗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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