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.
Open BDRX dashboard ↗BioMarin Pharmaceuticals Inc
BioMarin Pharmaceutical Inc., a biotechnology company, engages in the development and commercialization of therapies for life-threatening rare diseases and medical conditions in the United States, Europe, Latin America, the Middle East, the Asia Pacific, and internationally. The company's products include VIMIZIM, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; VOXZOGO, a once daily injection analog of c-type natriuretic peptide (CNP) for the treatment of achondroplasia; NAGLAZYME, a recombinant form of N- acetylgalactosamine 4-sulfatase for patients with MPS VI; and PALYNZIQ, a PEGylated recombinant phenylalanine (Phe) ammonia lyase enzyme delivered through subcutaneous injection to reduce blood Phe concentrations. It also develops BRINEURA, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; ALDURAZYME, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase; KUVAN, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria, an inherited metabolic disease; and ROCTAVIAN, an adeno associated virus vector for the treatment of severe hemophilia A. The company's products under development include BMN 333, a longer-acting CNP for the treatment of multiple growth disorders, such as achondroplasia and hypochondroplasia; BMN 349, an oral therapeutic for the treatment of liver disease associated with alpha-1 antitrypsin deficiency; and BMN 351, an oligonucleotide for the treatment of duchenne muscular dystrophy. It serves specialty pharmacies, hospitals, non-U.S. government agencies, distributors, and pharmaceutical wholesalers. The company has license and collaboration agreements with Catalyst Pharmaceutical Partners, Inc., and Ares Trading S.A. The company was incorporated in 1996 and is based in San Rafael, California.
Open BMRN dashboard ↗