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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.

BeiGene, Ltd., through its subsidiaries, engages in the development and commercialization of oncology medicines worldwide. Its products include BRUKINSA to treat various blood cancers; TEVIMBRA to treat various solid tumor and blood cancers; PARTRUVIX for the treatment of various solid tumor malignancies; XGEVA to treat bone metastases from solid tumors and multiple myeloma, as well as giant cell tumor of bone; BLINCYTO to treat acute lymphoblastic leukemia; KYPROLIS to treat R/R multiple myeloma; REVLIMID to treat multiple myeloma; VIDAZA to treat myelodysplastic syndromes, chronic myelomonocyte leukemia, and acute myeloid leukemia; SYLVANT to treat idiopathic multicentric castleman disease; QARZIBA to treat neuroblastoma; POBEVCY to treat metastatic colorectal cancer, liver cancer, and non-small cell lung cancer (NSCLC); BAITUOWEI, to treat breast and prostate cancers; TAFINLAR and MEKINIST to treat NSCLC and melanoma; VOTRIENT for advance renal cell carcinoma; AFINITOR for advance renal cell carcinoma, NET, SEGA, & breast cancers; and ZYKADIA to treat ALK + NSCLC. The company is also developing inhibitors comprising Sonrotoclax (BGB-11417), BGB-10188, BGB-21447, Ociperlimab (BGB-A1217), Surzebiclimab (BGB-A425), BGB-15025, BGB-26808, Lifirafenib (BGB-283), BGB-3245, BGB-30813, and BGB-43395; antibodies, including Zanidatamab, BGB-A445, and BGB-A3055, as well as BGB-16673, a BTK-targeted CDAC, and BGB-24714, a SMAC mimetic. It has collaborations with Shoreline Biosciences, Inc., Amgen Inc., Novartis AG, Bristol Myers Squibb company, Shandong Luye Pharmaceutical Co., Ltd., Nanjing Leads Biolabs, Inc., EUSA Pharma, Assembly Biosciences, Inc., Bio-Thera Solutions, Ltd., and Nanjing Leads Biolabs Co., Ltd. The company was incorporated in 2010 and is based in Grand Cayman, the Cayman Islands.

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.