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ACRS vs RARE

Aclaris Therapeutics, Inc. and Ultragenyx Pharmaceutical Inc.

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Aclaris Therapeutics, Inc.

Aclaris Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the development of novel drug candidates for immune-inflammatory diseases in the United States. It operates through two segments, Therapeutics and Contract Research. The Therapeutics segment is involved in identifying and developing therapies to address significant unmet needs for immuno-inflammatory diseases. Its Contract Research segment provides laboratory services. The company also develops ATI-1777, a soft JAK 1/3 inhibitor in Phase 2b trial for the treatment of atopic dermatitis and other dermatologic conditions; Bosakitug (ATI-045), an anti-TSLP monoclonal antibody in Phase 2a trial to treat moderate to severe atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyps, and moderate to severe chronic obstructive pulmonary disease; ATI-2138, an oral covalent inhibitor of ITK and JAK3 inhibitor in Phase 2a trial for the treatment for T cell-mediated autoimmune diseases; Zunsemetinib (ATI-450), a mitogen-activated protein kinase-activated protein kinase 2 inhibitor under Phase 1b/2 trials for the treatment of metastatic breast cancer and pancreatic ductal adenocarcinoma; and ATI-052, a humanized anti-TSLP and anti-IL4R bispecific antibody in preclinical stage to treat various atopic, immunologic, and respiratory diseases, as well as an ITK selective inhibitor candidate for the treatment of autoimmune diseases. Aclaris Therapeutics, Inc. was incorporated in 2012 and is headquartered in Wayne, Pennsylvania.

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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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