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BioMarin 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.

Incyte Corporation, a biopharmaceutical company, engages in the discovery, development, and commercialization of therapeutics for hematology/oncology, and inflammation and autoimmunity areas in the United States and internationally. The company offers JAKAFI (ruxolitinib) for treatment of intermediate or high-risk myelofibrosis, polycythemia vera, and steroid-refractory acute graft-versus-host disease; MONJUVI (tafasitamab-cxix)/MINJUVI (tafasitamab) for relapsed or refractory diffuse large B-cell lymphoma; PEMAZYRE (pemigatinib), a fibroblast growth factor receptor kinase inhibitor that act as oncogenic drivers in liquid and solid tumor types; ICLUSIG (ponatinib) to treat chronic myeloid leukemia and Philadelphia-chromosome positive acute lymphoblastic leukemia; and ZYNYZ (retifanlimab-dlwr) to treat adults with metastatic or recurrent locally advanced Merkel cell carcinoma, as well as OPZELURA cream for treatment of atopic dermatitis. Its clinical stage products include retifanlimab under Phase 3 clinical trials for squamous cell carcinoma of the anal canal and non-small cell lung cancer; axatilimab, an anti-CSF-1R monoclonal antibody under Phase 2 that is being developed as a therapy for patients with chronic GVHD; INCA033989 to inhibit oncogenesis; INCB160058, which is being developed as a disease-modifying therapeutic; and INCB99280 and INCB99318 for the treatment solid tumors. The company also develops INCB123667, INCA32459, and INCA33890, as well as Ruxolitinib cream, Povorcitinib, and INCA034460. It has collaboration out-license agreements with Novartis and Lilly; in-license agreements with Agenus, Merus, MacroGenics, and Syndax; and collaboration and license agreement with China Medical System Holdings Limited for the development and commercialization of povorcitinib. The company sells its products to specialty, retail, and hospital pharmacies, distributors, and wholesalers. The company was formerly known as Incyte Genomics Inc and changed its name to Incyte Corporation in March 2003. Incyte Corporation was incorporated in 1991 and is headquartered in Wilmington, Delaware.

Repligen Corporation develops and commercializes bioprocessing technologies and systems for use in biological drug manufacturing process in North America, Europe, the Asia Pacific, and internationally. It offers Protein A ligands that are the binding components of Protein A affinity chromatography resins; and cell culture growth factor products. The company's chromatography products include OPUS pre-packed chromatography columns, which are used in the purification of biologics; and OPUS smaller-scale columns that are used in the high throughput process development screening, viral clearance validation studies, and scale down validation of chromatography processes. It also offers ELISA test kits; and chromatography resins under the CaptivA brand. In addition, the company provides filtration products, such as XCell Alternating Tangential Flow systems that are filtration devices used in upstream perfusion and cell culture processing; TangenX flat sheet cassettes, which are used in downstream biologic drug concentration, buffer exchange, and formulation processes; KrosFlo tangential flow filtration and tangential flow depth filtration systems; Spectra/Por laboratory and process dialysis products, and ProConnex TFDF flow paths. Further, it provides process analytics products, such as slope spectroscopy systems under the SoloVPE, FlowVPE, and FlowVPX brands. The company sells its products to life sciences, biopharmaceutical, and diagnostics companies; laboratory researchers; and contract manufacturing organizations. Repligen Corporation has collaboration agreements with Navigo Proteins GmbH to develop multiple affinity ligands. The company was incorporated in 1981 and is headquartered in Waltham, Massachusetts.

United Therapeutics Corporation, a biotechnology company, engages in the development and commercialization of products to address the unmet medical needs of patients with chronic and life-threatening diseases in the United States and internationally. The company offers Tyvaso DPI, an inhaled dry powder via pre-filled and single-use cartridges; Tyvaso, an inhaled solution via ultrasonic nebulizer; Remodulin (treprostinil) injection to treat patients with pulmonary arterial hypertension (PAH) to diminish symptoms associated with exercise; Orenitram, a tablet dosage form of treprostinil, to delay disease progression and improve exercise capacity in PAH patients; and Adcirca, an oral PDE-5 inhibitor to enhance the exercise ability in PAH patients. It also markets and sells Unituxin (dinutuximab) injection, a monoclonal antibody for treating high-risk neuroblastoma; and Remunity Pump, which contains a pump and separate controller for Remodulin. In addition, the company engages in developing RemoPro and Ralinepag for the treatment of PAH; Aurora-GT, a gene therapy product to rebuild the blood vessels in the lungs; and Nebulized Tyvaso, for the treatment of idiopathic pulmonary fibrosis, as well as xenografts, which are development-stage organ products. It has licensing and collaboration agreements with DEKA Research & Development Corp. to develop a semi-disposable system for the subcutaneous delivery of treprostinil; MannKind Corporation to develop and license treprostinil inhalation powder and the Dreamboat device; and Arena Pharmaceuticals, Inc. to develop Ralinepag. The company was incorporated in 1996 and is headquartered in Silver Spring, Maryland.