BridgeBio Pharma
NASDAQ:BBIOBridgeBio Pharma, Inc., a commercial-stage biopharmaceutical company, discovers, creates, tests, and delivers transformative medicines to treat patients who suffer from genetic diseases and cancers. Its products in development programs include AG10, a next-generation oral small molecule near-complete TTR stabilizer that is in Phase 3 clinical trial for the treatment of TTR amyloidosis, or transthyretin amyloid cardiomyopathy (ATTR-CM); low-dose infigratinib, an oral FGFR1-3 selective tyrosine kinase inhibitor, which is in Phase 3 double-blinded, placebo-controlled pivotal study for the treatment option for children with achondroplasia; and BBP-631, an AAV5 gene transfer product candidate that is in Phase 1/2 clinical trial for the treatment of congenital adrenal hyperplasia, or CAH, driven by 21-hydroxylase deficiency, or 21OHD. The company also develops Encaleret, a small molecule antagonist of the calcium sensing receptor, or CaSR, which is in phase 3 clinical trial for treating autosomal dominant hypocalcemia type 1, or ADH1; and BBP-418, a glycosylation substrate pro-drug that is in Phase 3 clinical trial for treating limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). In addition, it engages in developing products for mendelian, oncology, and gene therapy diseases. BridgeBio Pharma, Inc. has license and collaboration agreements with the Leland Stanford Junior University; and Leidos Biomedical Research, Inc. The company was founded in 2015 and is headquartered in Palo Alto, California.
Emergent BioSolutions
NYSE:EBSEmergent BioSolutions Inc., a life sciences company, focuses on the provision of preparedness and response solutions that address accidental, deliberate, and naturally occurring public health threats (PHTs) in the United States. Its products address PHTs, which include chemical, biological, radiological, nuclear, and explosives; emerging infectious diseases; travel health; public health crises; and acute, emergency, and community care. The company offers ACAM2000, a smallpox vaccine; Anthrasil to for inhalational anthrax; Botulism Antitoxin Heptavalent to treat botulinum disease; BioThrax, an anthrax vaccine; Ebanga, a monoclonal antibody to treat infection caused by Zaire ebolavirus; raxibacumab injection for the treatment of inhalational anthrax; reactive skin decontamination lotion kits; TEMBEXA for the treatment of human smallpox disease; Trobigard, an auto-injector atropine sulfate and obidoxime chloride auto-injector for the emergency treatment of known or suspected exposure to nerve agents; and vaccinia immune globulin intravenous that addresses complications from smallpox vaccine. It also provides NARCAN, a nasal spray for the emergency treatment of known or suspected opioid overdose; Vivotif, an oral vaccine for typhoid fever; and Vaxchora, a single-dose oral vaccine to treat cholera. In addition, the company is developing AV7909, an anthrax vaccine; CGRD-001, a pralidoxime chloride/atropine auto-injector; CHIKV VLP, a chikungunya virus VLP vaccine; EBS-LASV; EGRD-001, a diazepam auto-injector; SIAN, an antidote for the initial treatment of acute poisoning of cyanide; and UniFlu, a universal influenza vaccine. Further, it provides contract development and manufacturing services comprising drug substance and product manufacturing, and packaging, as well as technology transfer, process, and analytical development services. The company was incorporated in 1998 and is headquartered in Gaithersburg, Maryland.
FibroGen
NASDAQ:FGENFibroGen, Inc., a biopharmaceutical company, discovers, develops, and commercializes therapeutics to treat serious unmet medical needs. Its lead product candidates are Pamrevlumab, a human monoclonal antibody targeting connective tissue growth factor that is in Phase III clinical development for the treatment of locally advanced pancreatic cancer; and Roxadustat, an oral small molecule inhibitor of hypoxia-inducible factor prolyl hydroxylase activity, which has completed Phase III clinical development for the treatment of anemia in chronic kidney disease in China, Europe, Japan, and other countries, as well as in Phase III clinical development for anemia related with myelodysplastic syndromes. The company has collaboration agreements with Astellas Pharma Inc. and AstraZeneca AB. FibroGen, Inc. was incorporated in 1993 and is headquartered in San Francisco, California.
GW Pharmaceuticals
NASDAQ:GWPHGW Pharmaceuticals plc, a biopharmaceutical company, focuses on discovering, developing, and commercializing novel therapeutics from its proprietary cannabinoid product platform in various disease areas. Its lead product is Epidiolex, an oral medicine for the treatment of seizures associated with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex. The company also develops and markets Sativex for the treatment of spasticity due to multiple sclerosis. In addition, it develops various product candidates for the treatment of schizophrenia, autism spectrum disorder, neuropsychiatric symptom, and neonatal hypoxic ischemic encephalopathy. It primarily operates in Europe, the United Kingdom, the United States, and internationally. GW Pharmaceuticals plc was founded in 1998 and is based in Cambridge, the United Kingdom.
Ultragenyx Pharmaceutical
NASDAQ:RAREUltragenyx 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.