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Alnylam 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 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.

Catalent, Inc., together with its subsidiaries, develops and manufactures solutions for drugs, protein-based biologics, cell and gene therapies, and consumer health products worldwide. It operates in two segments, Biologics, and Pharma and Consumer Health. The Biologics segment provides formulation, development, and manufacturing for biologic proteins, cell gene, and other nucleic acid therapies; pDNA, iPSCs, oncolytic viruses, and vaccines; formulation, development, and manufacturing for parenteral dose forms, including vials, prefilled syringes, and cartridges; and analytical development and testing services for large molecules. The Pharma and Consumer Health segment offers formulation, development, and manufacturing services for soft capsules for use in a range of customer products, such as prescription drugs, over-the-counter medications, dietary supplements, unit-dose cosmetics, and animal health medicinal preparations; and oral, nasal, inhaled, and topical dose forms. This segment also provides clinical supply services through manufacturing, packaging, storage, distribution, and inventory management for small-molecule drugs, protein-based biologics, and cell and gene therapies in clinical trials; and pre-clinical screening, formulation, analytical development, and current good manufacturing practices manufacturing at clinical and commercial scale for softgel capsule, Zydis fast-dissolve tablets, oral solid-dose formats, dry powder inhalers, and nasal delivery devices. The company also offers FlexDirect direct-to-patient and FastChain demand-led clinical supply solutions; fill and finish operations for injectable products; and integrated development and product supply chain solutions. It serves pharmaceutical, biotechnology, and consumer health companies; and companies in other healthcare market segments, such as animal health and medical devices, as well as in cosmetics industries. The company was founded in 1933 and is headquartered in Somerset, New Jersey.

Ono Pharmaceutical Co., Ltd., together with its subsidiaries, produces, purchases, and sells pharmaceuticals and diagnostic reagents worldwide. It offers Opdivo Intravenous Infusion and Kyprolis Intravenous Injection for malignant tumors; Emend capsules/Proemend intravenous injections for chemotherapy-induced nausea and vomiting; Demser capsules for the symptoms in patients with pheochromocytoma; and Mektovi, Velexbru, and Braftovi capsules for malignant tumors, as well as ADLUMIZ tablets for cancer cachexia. The company also provides Glactiv tablets for type 2 diabetes; Forxiga tablets for diabetes; Onoact Intravenous Infusion for tachyarrhythmia; Opalmon tablets to treat peripheral circulatory disorder; Coralan for chronic heart failure; Orencia injections for rheumatoid arthritis; Rivastach patches for Alzheimer's disease; Ongentys tablets for Parkinson's disease; Parsabiv, an intravenous infusion for dialysis patients; Staybla tablets for overactive bladder; Onon capsules and Dry Syrups for bronchial asthma and allergic rhinitis; and JOYCLU intra-articular injection for the improvement of joint function, as well as Recalbon tablets for osteoporosis. In addition, it develops products for hepatocellular and urothelial carcinoma; ovarian, bladder, prostate, pancreatic, gastric, esophageal, colorectal, thyroid, cell lung, and breast cancer; acute myeloid leukemia; solid tumors; myelodysplastic syndrome; melanoma; T-cell lymphoma; tachyarrhythmia; pemphigus; scleroderma; seizures; diabetic polyneuropathy; neurodegenerative diseases; autoimmune diseases; narcolepsy; and thrombosis. The company has a collaboration with Adimab, LLC to discover novel antibody drugs; a research collaboration with Turbine Ltd. to identify and validate novel oncology targets; Harvard University for validating novel therapeutic targets; and a strategic drug discovery collaboration with Sibylla Biotech in central nervous system disorders. The company was founded in 1717 and is headquartered in Osaka, Japan.

Royalty Pharma plc operates as a buyer of biopharmaceutical royalties and a funder of innovations in the biopharmaceutical industry in the United States. It is also involved in the identification, evaluation, and acquisition of royalties on various biopharmaceutical therapies. In addition, the company collaborates with innovators from academic institutions, research hospitals and not-for-profits, small and mid-cap biotechnology companies, and pharmaceutical companies. Its portfolio consists of royalties on approximately 35 commercial products and 14 development-stage product candidates that address various therapeutic areas, such as rare disease, cancer, neuroscience, immunology, respiratory, infectious disease, hematology, and diabetes. The company was founded in 1996 and is based in New York, New York.