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.
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IDEAYA Biosciences, Inc., a precision medicine oncology company, discovers and develops targeted therapeutics for patient populations selected using molecular diagnostics in the United States. The company's products in development include IDE196, a protein kinase C inhibitor that is in Phase 2/3 clinical trials for genetically defined cancers having GNAQ or GNA11 gene mutations; IDE397, a methionine adenosyltransferase 2a inhibitor that is in Phase 2 clinical trials for patients with solid tumors having methylthioadenosine phosphorylase gene deletions, such as non-small cell lung (SCLC) and MTAP-deletion urothelial cancer; IDE849, a DLL3 antibody drug conjugate inhibitor that is in Phase 1 clinical trial for the treatment of SCLC and neuroendocrine tumors; IDE275, a Werner Helicase inhibitor that is in Phase 1 clinical trial for patients having tumors with high microsatellite instability; IDE161, a poly ADP-ribose glycohydrolase inhibitor that is in Phase 1/2 clinical trial to treat tumors with homologous recombination deficiency (HRD), and other genetic or molecular signatures; IDE705 (GSK101), a Poly Theta Helicase inhibitor that is in Phase 1 clinical trial for the treatment of tumors with BRCA or other mutations associated with HRD. It also develops preclinical programs, including IDE892, a MTA-cooperative PRMT5 inhibitor; IDE034, a B7H3/PTK7 topo-I-payload bispecific BsADC; and IDE251, a KAT6/7 inhibitor. In addition, the company offers preclinical research programs focused on pharmacological inhibition; DECIPHER dual CRISPR library for synthetic lethality target and biomarker discovery; and INQUIRE chemical library and HARMONY machine-learning engines to enhance its drug discovery platform. The company has strategic alliances with GlaxoSmithKline, Pfizer Inc., Gilead Sciences, Inc., Merck, Biocytogen, Cancer Research UK, and the University of Manchester. IDEAYA Biosciences, Inc. was incorporated in 2015 and is headquartered in South San Francisco, California.
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