Insmed Incorporated, a biopharmaceutical company, develops and commercializes therapies for patients with serious and rare diseases in the United States, Europe, Japan, and internationally. The company offers ARIKAYCE for the treatment of Mycobacterium avium complex lung disease as part of a combination antibacterial drug regimen for adult patients. It is also developing Brensocatib, an oral reversible inhibitor of dipeptidyl peptidase 1 for the treatment of patients with bronchiectasis and other neutrophil-mediated diseases; and Treprostinil Palmitil Inhalation Powder, an inhaled formulation of a treprostinil prodrug treprostinil palmitil for the treatment of pulmonary hypertension associated with interstitial lung disease and pulmonary arterial hypertension. The company was founded in 1988 and is headquartered in Bridgewater, New Jersey.

BioMarin Pharmaceutical Inc. engages in the development and commercialization of 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. It has license and collaboration agreements with Sarepta Therapeutics; Ares Trading S.A.; Catalyst Pharmaceutical Partners, Inc.; and CAMP4 Therapeutics Corporation. The company was incorporated in 1996 and is headquartered in San Rafael, California.

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Japan, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co., Ltd. The company was incorporated in 2010 and is headquartered in Novato, California.

Amicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, for the treatment of late onset. It has collaboration and license agreements with the University of Pennsylvania to research and develop parvovirus gene therapy products; and GlaxoSmithKline. Amicus Therapeutics, Inc. was incorporated in 2002 and is headquartered in Princeton, New Jersey.

Chimerix, Inc., a biopharmaceutical company, develops medicines to improve and extend the lives of patients facing deadly diseases. Its pipeline products include ONC201 a program that is in Phase 3 clinical trial for treating patients with H3 K27M-mutant diffuse glioma, as well as in Phase 2 clinical trial for the treatment of rare neuroendocrine tumors; and ONC206, an imipridone, Dopamine Receptor D2 (DRD2) antagonist, and caseinolytic protease P (ClpP) agonist, which is in Phase 1 clinical trial for adult and pediatric patients with primary central nervous system tumors. The company also develops ONC212, an imipridone agonist of the orphan G protein-coupled receptors (GPCR) tumor suppressor GPR132, as well as ClpP for oncology indications; and CMX521, a nucleoside analog antiviral drug candidate for the treatment of SARS-CoV-2. It has license agreement with SymBio Pharmaceuticals to develop and commercialize TEMBEXA for human diseases other than orthopoxviruses, including smallpox. Chimerix, Inc. was incorporated in 2000 and is headquartered in Durham, North Carolina.

Pharming Group N.V., a biopharmaceutical company, develops and commercializes protein replacement therapies and precision medicines for the treatment of rare diseases in the United States, Europe, and internationally. The company offers RUCONEST, a recombinant C1 esterase inhibitor for the treatment of acute attacks in adult and adolescent patients with acute hereditary angioedema (HAE); and Joenja (leniolisib), an oral small molecule PI3K? inhibitor for the treatment of activated phosphoinositide 3-kinase delta syndrome. It also develops OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy for the treatment of HAE. The company has a development collaboration and license agreement with Novartis; and a strategic collaboration agreement with Orchard Therapeutics plc for research, development, manufacturing, and commercialization of OTL-105. Pharming Group N.V. was incorporated in 1988 and is headquartered in Leiden, the Netherlands.

MediciNova, Inc., a biopharmaceutical company, focuses on developing novel and small molecule therapeutics for the treatment of serious diseases with unmet medical needs in the United States. It is developing MN-166 (ibudilast), an oral anti-inflammatory and neuroprotective agent for treating neurological and other disorders, such as primary and secondary progressive multiple sclerosis, amyotrophic lateral sclerosis, chemotherapy-induced peripheral neuropathy, degenerative cervical myelopathy, glioblastoma, and substance dependence and addiction, as well as prevention of acute respiratory distress syndrome, and long COVID. The company's product pipeline also includes MN-221 (bedoradrine), a selective beta-2-adrenergic receptor agonist for the treatment of acute exacerbations of asthma; MN-001 (tipelukast), an orally bioavailable small molecule compound to treat fibrotic and other diseases, including nonalcoholic fatty liver disease and idiopathic pulmonary fibrosis; and MN-029 (denibulin), a tubulin binding agent for treating solid tumor cancers. It has license agreements with Kyorin Pharmaceutical Co., Ltd; Angiogene Pharmaceuticals, Ltd.; and Meiji Seika Kaisha, Ltd. The company was incorporated in 2000 and is headquartered in La Jolla, California.

Cocrystal Pharma, Inc., a biotechnology company, focuses on the discovery and development of antiviral therapeutic treatments for serious and/or chronic viral diseases. It employs structure-based technologies to create antiviral drugs primarily to treat hepatitis C virus (HCV), influenza virus, coronavirus, norovirus, and respiratory virus infections. The company develops CC-31244, a HCV non-nucleoside polymerase inhibitor that has completed Phase II a clinical trial to treat HCV infection; and CC-42344, a PB2 inhibitor that has completed Phase I clinical trial for treating influenza infection. It is also involved in identifying and developing non-nucleoside polymerase inhibitors for norovirus infections. Cocrystal Pharma, Inc. has a license and research collaboration agreement with Merck Sharp & Dohme Corp. to discover and develop proprietary influenza A/B antiviral agents; and a license agreement with Kansas State University Research Foundation to develop antiviral compounds for the treatment of norovirus and coronavirus infections. The company is headquartered in Bothell, Washington.