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.
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 Biopharma segment provides plasma derivatives products, including Gamunex/Gamunex-C Immune Globulin Injection, Flebogamma Immune Globulin Intravenous, Xembify Immune Globulin Subcutaneous solution, HyperRAB Rabies Immune Globulin, Prolastin/Prolastin-C/Prolastin-C Liquid/Prolasplan/Prolastina/Pulmolast/Lynspad Alpha 1-Proteinase Inhibitor, Fanhdi and Alphanate, Antihemophilic Factor/von Willebrand factor Complex, Koate-DVI Antihemophilic Factor, Albutein/Albutein Flexbag/ Human Albumin Grifols/Plasbumin Albumin, and Vistaseal/Veraseal; and Tavlesse, a non-plasma derivative medicinal product. The Diagnostic segment offers transfusion medicine, which includes Procleix Panther systems/Procleix Panther with Automation Ready technology, Automated NAT blood screening systems, assays, software, WADiana/Erytra/Erytra Eflexys analyzers, BLOODchip ID/IDCore/IDHPA/IDRHD/IDCORE/BIDS, and antigens; and clinical and diagnostics products, such as Promonitor and Alpha1D. The Bio Supplies segment provides human biological materials for life sciences research and the development and manufacturing of pharmaceutical and diagnostic products. The Others segment offers healthcare solutions, which provides services and manufactures products, including parenteral solutions, robotics, and software used by hospitals, blood banks, plasma collection centers and other healthcare systems. The company's products and services are used by healthcare providers to diagnose and treat patients with hemophilia, immune deficiencies, infectious diseases, and other medical conditions. Grifols, S.A. 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 1940 and is headquartered in Barcelona, Spain.
Sage Therapeutics
NASDAQ:SAGESage Therapeutics, Inc., a biopharmaceutical company, develops and commercializes brain health medicines. Its product candidates include ZULRESSO, a CIV injection for the treatment of postpartum depression (PPD) in adults; and ZURZUVAE, a neuroactive steroid, a positive allosteric modulator of GABAA receptors, targeting both synaptic and extrasynaptic GABAA receptors, for the treatment of postpartum depression. Its product pipeline also comprises SAGE-324, a compound that is in Phase II clinical trial to treat essential tremors, as well as has completed Phase I clinical trial for epilepsy and Parkinson's diseases; and SAGE-718, an oxysterol-based positive allosteric modulator of the NMDA receptor, which is in Phase II clinical trial for the treatment of depression, Huntington's disease, Parkinson's diseases, Alzheimer's disease, attention deficit hyperactivity disorder, schizophrenia, and neuropathic pain. The company has a strategic collaboration with Shionogi & Co., Ltd. for the development and commercialization of zuranolone in Japan, Taiwan, and South Korea; and a collaboration and license agreement with Biogen MA Inc. to jointly develop and commercialize SAGE-217 and SAGE-324 products. The company was formerly known as Sterogen Biopharma, Inc. and changed its name to Sage Therapeutics, Inc. in September 2011. Sage Therapeutics, Inc. was incorporated in 2010 and is headquartered in Cambridge, Massachusetts.
Sarepta Therapeutics
NASDAQ:SRPTSarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases. It offers EXONDYS 51 injection to treat duchenne muscular dystrophy (duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping; AMONDYS 45 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene; and ELEVIDYS, an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with duchenne with a confirmed mutation in the duchenne gene. The company is also developing SRP-5051, a peptide conjugated PMO that binds exon 51 of dystrophin pre-mRNA; and SRP-9003, a limb-girdle muscular dystrophies gene therapy program. It has collaboration and license agreements with F. Hoffman-La Roche Ltd; Nationwide Children's Hospital; Genevant Sciences; University of Florida; Dyno Therapeutics; Hansa Biopharma; Duke University; Genethon; and StrideBio. The company was incorporated in 1980 and is headquartered in Cambridge, Massachusetts.