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AKBA vs RARE

Akebia Therapeutics, Inc. and Ultragenyx Pharmaceutical Inc.

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Akebia Therapeutics, Inc.

Akebia Therapeutics, Inc., a biopharmaceutical company, focuses on the development and commercialization of therapeutics for patients with kidney diseases. Its product portfolio includes Vafseo (vadadustat), an oral hypoxia-inducible factor prolyl hydroxylase, which is in Phase III development for the treatment of anemia due to chronic kidney disease (CKD) in dialysis-dependent (DD) and non-dialysis dependent (NDD) patients; and Auryxia, a ferric citrate that is used to control the serum phosphorus levels in adult patients with DD-CKD and the treatment of iron deficiency anemia in adult patients with NDD-CKD. The company is also developing AKB-9090 for treating cardiac surgery-related acute kidney injury and acute respiratory distress syndrome; and AKB-10108 for the treatment of retinopathy of prematurity in neonates. The company has license and collaboration agreements with Mitsubishi Tanabe Pharma Corporation for the development and commercialization of Vafseo in Japan and other Asian countries. Akebia Therapeutics, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.

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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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