Amicus Therapeutics
NASDAQ:FOLDAmicus 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.
Portola Pharmaceuticals
NASDAQ:PTLAPortola Pharmaceuticals, Inc., a biopharmaceutical company, develops and commercializes novel therapeutics in the areas of thrombosis and other hematologic disorders and inflammation in the United States. The company offers Andexxa, an antidote for patients treated with rivaroxaban and apixaban when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding; and Bevyxxa (betrixaban), an oral, once-daily Factor Xa inhibitor for the prevention of venous thromboembolism in adult patients hospitalized for an acute medical illness. It is also advancing cerdulatinib, a dual spleen tyrosine kinase and janus kinases inhibitor in development for the treatment of hematologic cancers. In addition, the company is developing PRT2761, a Syk inhibitor that has completed Phase II clinical trial for the treatment for allergic conjunctivitis. Portola Pharmaceuticals, Inc. has collaboration agreements with Bristol-Myers Squibb; Pfizer Inc.; Bayer Pharma, AG; Janssen Pharmaceuticals, Inc.; Daiichi Sankyo, Inc.; Millennium Pharmaceuticals, Inc.; SRX Cardio; LLC; Ora; and Astellas Pharma Inc. The company was founded in 2003 and is headquartered in South San Francisco, California.
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.
Ra Pharmaceuticals
NASDAQ:RARXRa Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops therapeutics for the treatment of diseases caused by excessive or uncontrolled activation of the complement system. The company's peptide chemistry platform enables the production of synthetic macrocyclic peptides that combine the diversity and specificity of antibodies with the pharmacological properties of small molecules. Its lead product candidate is Zilucoplan, an injection into the tissue under the skin that has completed Phase II clinical trial for the treatment of generalized myasthenia gravis (gMG); has completed Phase II clinical trial for treating paroxysmal nocturnal hemoglobinuria (PNH); and has completed Phase Ib clinical trial to treat patients with renal impairment. The company's pre-clinical programs include Factor D inhibition for treating C3 glomerulonephritis and dense deposit disease; and inhibitors of other complement factors for renal, autoimmune, and central nervous system diseases. It has collaboration and license agreement with Merck & Co., Inc. to identify orally available cyclic peptides for non-complement program targets, and provide research and development services. Ra Pharmaceuticals, Inc. was founded in 2008 and is headquartered in Cambridge, Massachusetts.
Zai Lab
NASDAQ:ZLABZai Lab Limited develops and commercializes therapies to treat oncology, autoimmune disorders, infectious diseases, and neuroscience. Its commercial products include Zejula, an orally administered poly polymerase 1/2 inhibitor; Optune, a cancer therapy that uses electric fields tuned to specific frequencies to kill tumor cells; NUZYRA for acute bacterial skin and skin structure infections, and community acquired bacterial pneumonia; Qinlock to treat gastrointestinal stromal tumors, and VYVGART, a human IgG1 antibody fragment for myesthenia gravis. The company also develops Tumor Treating Fields, a portable device for delivery of electric fields; Repotrectinib, a tyrosine kinase inhibitor (TKI) to target ROS1 and TRK A/B/C in TKI-naïve- or -pretreated cancer patients; Tisotumab vedotin, an antibody drug conjugate; Adagrasib for treating KRAS-G12C-mutated NSCLC, colorectal cancer, and pancreatic cancer; and Bemarituzumab to treat gastric and gastroesophageal junction cancer patients. In addition, it develops Sulbactam/durlobactam, a combination of a beta-lactam antibiotic and a beta-lactamase inhibitor for the treatment of serious infections caused by Acinetobacter; KarXT for the treatment of psychiatric and neurological conditions. It has license and collaboration agreement with Tesaro, Inc. to develop, manufacture, and commercialize niraparib; NovoCure to develop and commercialize Tumor Treating Fields; Deciphera to develop and commercialize ripretinib; Paratek Bermuda Ltd. to develop, manufacture, and commercialize omadacycline; argenx, to develop and commercialize efgartigimod; BMS to develop and commercialize tisotumab vedotin and repotrectinib; Mirati to research, develop, manufacture, and commercialize adagrasib; Amgen to develop and commercialize bemarituzumab; and Innoviva to develop and commercialize Sulbactam-Durlobactam; Karuna to develop and commercialize KarXT. The company was incorporated in 2013 and is headquartered in Shanghai, China.