Audentes Therapeutics
NASDAQ:BOLDAudentes Therapeutics, Inc., a clinical stage biotechnology company, focuses on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases caused by single gene defects. The company is developing AT132, which is in Phase I/II clinical studies for the treatment of X-linked myotubular myopathy (XLMTM); AT342 that is in Phase I/II clinical studies to treat crigler-najjar syndrome; AT845, which is in preclinical studies for the treatment of pompe disease; and AT307 to treat CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. It is also developing vectorized antisense treatments for the treatment of duchenne muscular dystrophy and myotonic dystrophy type 1. The company has a collaborative development agreement with Genethon to research, develop, manufacture, and commercialize products for the treatment of XLMTM; and a license and collaboration agreement with the University of Pennsylvania to research, develop, sell, and import licensed products for the treatment of crigler-najjar. Audentes Therapeutics, Inc. was founded in 2012 and is headquartered in San Francisco, California.
Coherus BioSciences
NASDAQ:CHRSCoherus BioSciences, Inc., a biopharmaceutical company, focuses on the research, development, and commercialization of cancer treatments primarily in the United States. The company develops UDENYCA, a biosimilar to Neulasta, a long-acting granulocyte-colony stimulating factor; LOQTORZI, a novel PD-1 inhibitor; and Casdozokitug, an investigational recombinant human immunoglobulin isotype G1 (IgG1) monoclonal antibody targeting interleukin 27. It is also developing CHS-114, an investigational highly specific human afucosylated IgG1 monoclonal antibody selectively targeting CCR8, a chemokine receptor highly expressed on Treg cells in the tumor microenvironment (TME); and CHS-1000, an antibody targeting human ILT4 designed to improve anti-PD-1 clinical benefit by transforming an unfavorable TME to a more favorable TME. In addition, the company's licensed immuno-oncology programs include NZV930, an antibody designed to inhibit cluster of differentiation 73; and GSK4381562, an antibody targeting CD112R for the treatment of solid tumors. Further, it offers YUSIMRY, a biosimilar to Humira for the treatment of patients with inflammatory diseases characterized by increased production of tumor necrosis factor (TNF) in the body, including rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn's disease, psoriasis, and ulcerative colitis. It collaboration agreement with Junshi Biosciences for the co-development and commercialization of toripalimab; agreement with Surface and Adimab LLC; license agreements with Bioeq AG and Genentech, Inc., as well as Vaccinex, Inc.; and out-licensing agreement with Novartis Institutes for Biomedical Research, Inc. and GlaxoSmithKline Intellectual Property No. 4 Limited. The company was formerly known as BioGenerics, Inc. and changed its name to Coherus BioSciences, Inc. in April 2012. Coherus BioSciences, Inc. was incorporated in 2010 and is headquartered in Redwood City, California.
Momenta Pharmaceuticals
NASDAQ:MNTAMomenta Pharmaceuticals, Inc., a biotechnology company, focuses on the discovery and development of novel biologic therapies for the treatment of rare immune-mediated diseases in the United States. Its novel therapeutic programs include M281, a fully-human anti-neonatal Fc receptor (FcRn), aglycosylated immunoglobulin G (IgG1), and monoclonal antibody to reduce circulating IgG antibodies by blocking endogenous IgG recycling via FcRn; M230, a recombinant trivalent human IgG1 Fc multimer containing three IgG Fc regions joined to maximize activity; and M254, a hyper-sialylated immunoglobulin to treat various inflammatory diseases, including idiopathic thrombocytopenic purpura and chronic inflammatory demyelinating polyneuropathy. The company's biosimilar programs comprise M923, a biosimilar of HUMIRA for the treatment of patients with rheumatoid arthritis, crohn's disease, ulcerative colitis, and psoriasis; and M710, a biosimilar of EYLEA for the treatment of neovascular age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema (DME), and diabetic retinopathy in patients with DME. Its complex generics programs include Enoxaparin sodium injection, a generic version of LOVENOX that is indicated for the prevention and treatment of deep vein thrombosis, as well as supports the treatment of acute coronary syndromes; GLATOPA, a generic version of once-daily COPAXONE for the treatment of patients with relapsing forms of multiple sclerosis; and GLATOPA, a generic version of three-times-weekly COPAXONE. The company has collaboration and license agreements with Sandoz AG, Mylan Ireland Limited, and CSL Behring Recombinant Facility AG. The company was formerly known as Mimeon, Inc. and changed its name to Momenta Pharmaceuticals, Inc. in September 2002. Momenta Pharmaceuticals, Inc. was founded in 2001 and is headquartered in Cambridge, Massachusetts.
Orchard Therapeutics
NASDAQ:ORTXOrchard Therapeutics plc, a gene therapy company, research, develops, and commercialize hematopoietic stem cell and gene therapies in the United Kingdom, Italy, France, and Germany. It offers OTL-200, an ex vivo autologous hematopoietic stem cell gene therapy for the treatment of patients with metachromatic leukodystrophy under the Libmeldy brand; and Strimvelis, a gammaretroviral vector-based gene therapy for the treatment of adenosine deaminase deficiency. The company's program for neurodegenerative disorders comprises clinical proof of concept-stage program, which includes OTL-203 for the treatment of mucopolysaccharidosis type I and OTL-201 for mucopolysaccharidosis type IIIA, or MPS-IIIA; and pre-clinical program, OTL-204 for the treatment of frontotemporal dementia with progranulin mutations. In addition, it develops program for immunological disorders consist of pre-clinical programs, which includes OTL-104 for Crohn's disease with mutations in the nucleotide-binding oligomerization domain-containing protein 2; and OTL-105 for the treatment of hereditary angioedema. The company was formerly known as Orchard Rx Limited. Orchard Therapeutics plc was founded in 2015 and is headquartered in London, the United Kingdom. As of January 24, 2024, Orchard Therapeutics plc operates as a subsidiary of Kyowa Kirin Co., Ltd..
REGENXBIO
NASDAQ:RGNXREGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapies that deliver functional genes to cells with genetic defects in the United States. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's products in pipeline includes ABBV-RGX-314 for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases; and RGX-202, which is in Phase I/II clinical trial for the treatment of Duchenne muscular dystrophy. It also develops RGX-121 for the treatment of mucopolysaccharidosis type II that is in Phase III clinical trial; RGX-111 for treating mucopolysaccharidosis type I; RGX-181 for the treatment of late infantile neuronal ceroid lipofuscinosis type II; and RGX-381 to treat the ocular manifestations of CLN2 disease. In addition, the company licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies. Further, it has a collaboration and license agreement with AbbVie Global Enterprises Ltd. to develop ABBV-RGX-314 outside the United States. REGENXBIO Inc. was incorporated in 2008 and is headquartered in Rockville, Maryland.