Alnylam Pharmaceuticals
NASDAQ:ALNYAlnylam Pharmaceuticals, Inc., a biopharmaceutical company, focuses on discovering, developing, and commercializing novel therapeutics based on ribonucleic acid interference. Its marketed products include ONPATTRO (patisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; AMVUTTRA for the treatment of hATTR amyloidosis with polyneuropathy in adults; GIVLAARI for the treatment of adults with acute hepatic porphyria; and OXLUMO for the treatment of primary hyperoxaluria type 1. In addition, the company develops patisiran for the treatment of transthyretin amyloidosis, or ATTR amyloidosis, with cardiomyopathy; cemdisiran to treat complement-mediated diseases; Belcesiran for the treatment of alpha-1 liver disease; Elebsiran to treat chronic HBV infection; Zilebesiran to treat hypertension; ALN-APP to treat Alzheimer's disease and cerebral amyloid angiopathy; and ALN-HSD to treat NASH. Further, it offers Fitusiran for the treatment of hemophilia, Inclisiran to treat hypercholesterolemia, lumasiran for the treatment of advanced PH1, and vutrisiran for the treatment of ATTR amyloidosis, which is in phase 3 clinical trial. Alnylam Pharmaceuticals, Inc. has strategic collaborations with Regeneron Pharmaceuticals, Inc. to discover, develop, and commercialize RNAi therapeutics for a range of diseases by addressing therapeutic targets expressed in the eye and CNS; and Roche to develop pharmaceutical products containing zilebesiran. It also has license and collaboration agreements with Novartis AG; Vir Biotechnology, Inc.; Dicerna Pharmaceuticals, Inc.; Ionis Pharmaceuticals, Inc.; and PeptiDream, Inc. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts.
BioMarin Pharmaceutical
NASDAQ:BMRNBioMarin Pharmaceutical Inc. develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops Roctavian, an adeno associated virus vector, for the treatment of patients with severe hemophilia A. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. BioMarin Pharmaceutical Inc. has license and collaboration agreements with Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc. The company was incorporated in 1996 and is headquartered in San Rafael, California.
Galapagos
NASDAQ:GLPGGalapagos NV, a biotechnology company, develops medicines focusing on oncology and immunology primarily in the United States and Europe. The company's pipeline products comprise GLPG3667 that has completed phase 1b trial; GLPG5101, a CD19 CAR-T product candidate manufactured at point-of-care, currently in Phase1/2 trial in relapsed/refractory non-hodgkin lymphoma; GLPG5201, a CD19 CAR-T product candidates manufactured at point-of-care, currently in phase 1/2 trial in replapsed/refractory chronic lymphocytic leukemia; and GLPG5301, a BCMA CAR-T product candidate manufactured at point-of-care, currently in phase 1/2 in relapsed/refractory multiple myeloma. The company has collaboration agreements with Gilead Sciences, Inc.; and AbbVie S.à r.l. Galapagos NV was incorporated in 1999 and is headquartered in Mechelen, Belgium.
Genmab A/S
OTCMKTS:GNMSFGenmab A/S develops antibody therapeutics for the treatment of cancer and other diseases primarily in Denmark. The company markets DARZALEX, a human monoclonal antibody for the treatment of patients with multiple myeloma (MM); teprotumumab for the treatment of thyroid eye disease; and Amivantamab for advanced or metastatic gastric or esophageal cancer and NSCLC. Its products include daratumumab to treat MM, non-MM blood cancers, and AL amyloidosis; GEN1047; tisotumab vedotin for treating cervical, ovarian, and solid cancers; DuoBody-PD-L1x4-1BB, and DuoBody-CD40x4-1BB for treating solid tumors; Epcoritamab for relapsed/refractory diffuse large B-cell lymphoma and chronic lymphocytic leukemia; and HexaBody-CD38 and GEN3017 for treating hematological malignancies. In addition, the company develops Inclacumab, which is in Phase 3 trial for vaso-occlusive crises; Camidanlumab tesirine to treat hodgkin lymphoma and solid tumors; JNJ-64007957 and JNJ-64407564 to treat MM; PRV-015 for treating celiac disease; Mim8 for treating haemophilia A; and Lu AF82422 for treating multiple system atrophy disease. It operates various active pre-clinical programs. The company has a commercial license and collaboration agreement with Seagen Inc. to co-develop tisotumab vedotin. It also has a collaboration agreement with argenx to discover, develop, and commercialize novel therapeutic antibodies with applications in immunology and oncology; and AbbVie for the development of epcoritamab, as well as collaborations with BioNTech, Janssen, and Novo Nordisk A/S. Genmab A/S was founded in 1999 and is headquartered in Copenhagen, Denmark.
Grifols
NASDAQ:GRFSGrifols, S.A. operates as a plasma therapeutic company in Spain, the United States, Canada, and internationally. The company provides immunoglobulin to treat immunodeficiencies; albumin used to restore circulatory volume and protein loss in pathophysiological conditions, such as liver cirrhosis, cardiocirculatory failure, trauma and severe burns; alpha-1 proteinase inhibitor, a plasma protein, used to treat a genetic disease known as alpha-1; factorVIII/von Willerbrand factor and factor IX, clotting factors for the treatment of hemophilia A and von Willebrand's disease, as well as hemophilia B; antithrombin III to treat hereditary antithrombin deficiency; Fostamatinib, a spleen tyrosine kinase inhibitor; combination of fibrinogen and enzyme thrombin that acts as a biological sealant to control surgical bleeding; and plasma exchange with albumin used to treat Alzheimer's disease. It markets diagnostic testing equipment, reagents, and other equipment; biological products; manufactures and sells plasma to third parties; and involves in research activities, as well as markets pharmaceutical products for hospital pharmacies. In addition, the company offers Yimmugo PID, an immunology drug; and Yimmugo ITP, a hematology drug. Further, it develops Xembify Pre-filled syringes, FlexBag, and Prolastin vials; Xembify Biweekly dosing, Prolastin-C, Fostamatinib2, and VISTASEAL which are in Phase IV development stage; Xembify, Albumin 20% and 5%, Fibrinogen, Trimodulin, Cytotec pregnancy, and AMBAR-Next in Phase III development stage; and AKST4290 that is in Phase II clinical development. Additionally, it offers recIG, Alpha-1 AT in non-cystic fibrosis bronchiectasis, ATIII, GIGA 2339, GIGA564, and OSIG. It has collaboration agreements with Canadian Blood Services for the processing of other plasma-derived products and with GIANT; and GigaGen to develop recombinant polyclonal immunoglobulin therapies. The company was founded in 1909 and is headquartered in Barcelona, Spain.