Quoin Pharmaceuticals, Ltd. American Depositary Shares
Quoin Pharmaceuticals, Ltd., a late stage specialty pharmaceutical company, focuses on the development and commercialization of therapeutic products for rare and orphan diseases in the United States. The company's lead product is QRX003, a topical lotion to treat Netherton Syndrome (NS). It also developing QRX004 for the treatment of recessive dystrophic epidermolysis bullosa; QRX007 to treat Netherton Syndrome; and QRX008 for the treatment of scleroderma. It has a research agreement with Queensland University of Technology; a license agreement with Skinvisible Inc.; consulting agreements with Axella Research LLC; and a Master Service Agreement with Therapeutics Inc. Quoin Pharmaceuticals, Ltd. was founded in 2018 and is based in Ashburn, Virginia. Quoin Pharmaceuticals, Ltd. is a subsidiary of Skinvisible, Inc.
Open QNRX 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.
Open RARE dashboard ↗