Cel-Sci Corporation
CEL-SCI Corporation, a clinical-stage biotechnology company, engages in the research and development to treat cancer and other diseases by using the immune system in the United States. The company's lead immunotherapy is Multikine, which has completed Phase III clinical trials for the potential treatment of certain head and neck cancers. It is also developing Ligand Epitope Antigen Presentation System (LEAPS) technology, a patented T-cell modulation process that stimulates the human immune system to fight bacterial, viral, parasitic infections, autoimmune conditions, allergies, transplantation rejections, and cancer, as well as to potentially treat rheumatoid arthritis. In addition, the company's LEAP product candidates include CEL-2000; CEL-4000; and CEL-5000 that are in preclinical trials for potential rheumatoid arthritis therapeutic treatments. CEL-SCI Corporation has a strategic partnership with Saudi Arabian Pharma Company for Multikine in the treatment of head and neck cancer. CEL-SCI Corporation was incorporated in 1983 and is headquartered in Vienna, Virginia.
Open CVM 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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