Arrowhead Research Corporation
Arrowhead Pharmaceuticals, Inc. develops medicines for the treatment of intractable diseases in the United States. Its pipeline includes Plozasiran to reduce production of apolipoprotein C-III in Phase 3 studies; Zodasiran to reduce production of angiopoietin-like protein 3 in Phase 3 clinical trials; ARO-DIMER-PA, a dual functional RNAi molecule in a Phase 1/2a clinical trials; and ARO-PNPLA3, an investigational RNAi therapeutic in Phase 1 clinical trials. The company also develops ARO-INHBE, to reduce the hepatic expression of the INHBE gene and its secreted gene product, Activin E in Phase 1/2a clinical trials; ARO-ALK7 to silence adipocyte expression of the ACVR1C gene in Phase 1/2a clinical trials; ARO-RAGE to reduce production of the receptor for advanced glycation end products in Phase 1/2a clinical trials; and ARO-MAPT, an investigational RNAi-based therapy. In addition, it is developing ARO-C3 to reduce production of C3 in Phase 1/2a clinical trials; and ARO-CFB to reduce hepatic expression of CFB in a Phase 1/2a clinical trials. The company has collaboration and license Agreements with Glaxosmithkline Intellectual Property (No. 3) Limited; Takeda Pharmaceutical Company Limited; Amgen Inc.; and Sarepta Therapeutics, Inc. Arrowhead Pharmaceuticals, Inc. was founded in 2001 and is headquartered in Pasadena, California.
Open ARWR dashboard ↗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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