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GKOS vs RARE

Glaukos Corporation and Ultragenyx Pharmaceutical Inc.

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Glaukos Corporation

Glaukos Corporation, an ophthalmic pharmaceutical and medical technology company, develops therapies for the treatment of glaucoma, corneal disorders, and retinal diseases in the United States and internationally. The company offers iStent and iStent inject W micro-bypass stents designed to treat mild-to-moderate open-angle glaucoma through the restoration of the natural physiologic outflow pathways for aqueous humor. It also provides iStent infinite indicated for use in the treatment of patients with glaucoma uncontrolled by prior medical and surgical therapy; iDose TR, an intracameral procedural pharmaceutical therapy indicated for the reduction of intraocular pressure in patients with open-angle glaucoma or ocular hypertension; iAccess, a precision blade; and iPRIME, a viscoelastic delivery system. In addition, the company develops iLink, a device used for the treatment of keratoconus without the removal of the epithelium; ILution, a platform of cream-based drug formulation applied to the outer surface of the eyelid for drop less transdermal delivery of pharmaceutically active compounds for the treatment of anterior segment eye disorders; and retinal XR platform to treat age-related macular degeneration, diabetic macular edema, retinal vein occlusion, and other posterior segment retinal diseases. It sells its products to ambulatory surgery centers, hospitals, and physician private practices through a direct sales organization, direct sales subsidiaries, and distributors. The company was formerly known as Transdx, Inc. Glaukos Corporation was incorporated in 1998 and is headquartered in Aliso Viejo, California.

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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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