Biodexa Pharmaceuticals plc American Depositary Shs
Biodexa Pharmaceuticals Plc, a clinical stage biopharmaceutical company, focuses on developing a pipeline of products for the treatment of familial adenomatous polyposis, non-muscle invasive bladder cancer, and Type 1 diabetes and rare/orphan cancers of the brain. Its lead product candidate Tolimidone, a selective activator of the lyn kinase enzyme, currently under Phase II studies for the treatment of Type 1 diabetes. The company is also developing MTX110, which is currently in Phase I studies for the treatment of diffuse intrinsic recurrent glioblastoma, diffuse midline glioma, and medulloblastoma. In addition, the company offers drug delivery platforms, such as Q-Sphera, a polymer microsphere microtechnology used for sustained release therapeutics; MidaSolve, an oligosaccharide nanotechnology used to solubilize drugs so that they can be administered in liquid form directly and locally into tumors; and MidaCore, a gold nanoparticle used for targeting sites of disease by using chemotherapeutic agents or immunotherapeutic agents. It has collaboration agreements with Janssen and Melior. The company was formerly known as Midatech Pharma plc and changed its name to Biodexa Pharmaceuticals Plc in March 2023. Biodexa Pharmaceuticals Plc was founded in 2000 and is headquartered in Cardiff, the United Kingdom.
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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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