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KPRX vs RARE

Kiora Pharmaceuticals, Inc. Common Stock and Ultragenyx Pharmaceutical Inc.

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Kiora Pharmaceuticals, Inc. Common Stock

Kiora Pharmaceuticals, Inc., a clinical-stage specialty pharmaceutical company, engages in the development and commercialization of therapies for the treatment of ophthalmic diseases. Its lead product is KIO-301, a potential vision-restoring small molecule, which is in Phase 2 clinical trials for the treatment of retinitis pigmentosa, choroideremia, and Stargardt disease. The company is also developing KIO-104, a non-steroidal, immuno-modulatory, small-molecule inhibitor of dihydroorotate dehydrogenase, which is in phase 1b/2a study for the treatment of posterior non-infectious uveitis, as well as under pre-clinical development for the treatment of proliferative vitreoretinopathy; and KIO-101, an eye drop formulation for the treatment of ocular presentation of rheumatoid arthritis. It has strategic development and commercialization agreement with Théa Open Innovation for the development of KIO-301; and collaboration and development agreement with Global Genes for treating rare ocular disorders. The company operates in the United States, Austria, and Australia. The company was formerly known as Eyegate Pharmaceuticals, Inc. and changed its name to Kiora Pharmaceuticals, Inc. in November 2021. Kiora Pharmaceuticals, Inc. was incorporated in 1998 and is headquartered in Encinitas, 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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