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AZTR vs RARE

Azitra Inc and Ultragenyx Pharmaceutical Inc.

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Azitra Inc

Azitra, Inc., an early-stage clinical biopharmaceutical company, develops therapies for precision dermatology using engineered proteins and live biotherapeutic products to treat skin diseases. It develops ATR-12, a genetically modified strain of S. epidermidis, which is in Phase Ib clinical trial for treating Netherton syndrome, a skin disease. The company also develops ATR-04, a genetically modified strain of S. epidermidis that is in Phase 1/2 clinical trial for treating the papulopustular rash experienced by cancer patients undergoing epidermal growth factor receptor inhibitor; and ATR-01, an engineered recombinant human filaggrin protein for treating ichthyosis vulgaris, a skin disease. It has a collaboration with Bayer. The company was incorporated in 2014 and is based in Branford, Connecticut.

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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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