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AUTL vs RARE

Autolus Therapeutics plc American Depositary Share and Ultragenyx Pharmaceutical Inc.

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Autolus Therapeutics plc American Depositary Share

Autolus Therapeutics plc, a clinical-stage biopharmaceutical company, develops T cell therapies for the treatment of cancer and autoimmune diseases in United Kingdom and internationally. The company's clinical-stage programs include obecabtagene autoleucel (AUTO1), a CD19-targeting programmed T cell investigational therapy that is in Phase 1b/2 clinical trial for the treatment of adult ALL; AUTO1/22, which is in a Phase 1 clinical trial in pediatric patients with relapsed or refractory ALL; AUTO4, a programmed T cell investigational therapy for the treatment of peripheral T-cell lymphoma targeting TRBC1 and TRBC2; AUTO6NG, a programmed T cell investigational therapy targeting GD2 in development for the treatment of neuroblastoma; and AUTO8, a product candidate to treat multiple myeloma. It focuses on developing AUTO5, a preclinical TRBC2 programmed T cell product candidate for the treatment of peripheral T-cell lymphoma. The company was incorporated in 2014 and is headquartered in London, the United Kingdom.

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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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