Cabaletta Bio
NASDAQ:CABACabaletta Bio, Inc., a clinical-stage biotechnology company, focuses on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases. The company's lead product candidate is DSG3-CAART, which is in Phase I clinical trial for the treatment of mucosal pemphigus vulgaris, an autoimmune blistering skin disease. Its product candidate pipeline also includes MuSK-CAART, a preclinical stage product to treat a subset of patients with myasthenia gravis; CABA-201, a 4-1BB-containing CD19-CAR T investigational therapy, for the treatment of severe autoimmune diseases; PLA2R-CAART, a discovery stage product to treat patients with PLA2R-associated membranous nephropathy; and DSG3/1-CAART, a discovery stage product for the treatment of mucocutaneous pemphigus vulgaris. It has a collaboration with the University of Pennsylvania and the Children's Hospital of Philadelphia. The company was formerly known as Tycho Therapeutics, Inc. and changed its name to Cabaletta Bio, Inc. in August 2018. Cabaletta Bio, Inc. was incorporated in 2017 and is headquartered in Philadelphia, Pennsylvania.
Opthea
NASDAQ:OPTOpthea Limited, a clinical stage biopharmaceutical company, engages in the development and commercialization of therapies primarily for eye disease in Australia. The company's development activities are based on the intellectual property portfolio covering Vascular Endothelial Growth Factors (VEGF) VEGF-C, VEGF-D, and VEGF Receptor-3 for the treatment of diseases associated with blood and lymphatic vessel growth, as well as vascular leakage. Its lead product candidate is Sozinibercept (OPT 302), a soluble form of vascular endothelial growth factor receptor-3 VEGFR-3, currently under Phase 3 clinical development as a novel therapy for wet age-related macular degeneration and diabetic macular edema. The company was formerly known as Circadian Technologies Limited and changed its name to Opthea Limited in December 2015. Opthea Limited was incorporated in 1984 and is based in South Yarra, Australia.
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..
Oyster Point Pharma
NASDAQ:OYSTOyster Point Pharma, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of pharmaceutical therapies to treat ophthalmic diseases in the United States. The company's product candidate is TYRVAYA, a nicotinic acetylcholine receptor agonist for the treatment of signs and symptoms of dry eye disease. It is also developing TYRVAYA that is in Phase II clinical trial for the treatment of for neurotrophic keratopathy. The company was incorporated in 2015 and is headquartered in Princeton, New Jersey.
Solid Biosciences
NASDAQ:SLDBSolid Biosciences Inc., a life science company, engages in developing therapies for neuromuscular and cardiac diseases in the United States. The company's lead product candidate is SGT-003, a next-generation gene transfer candidate for the treatment of Duchenne muscular dystrophy (Duchenne); AVB-202-TT, a gene therapy program for the treatment of Friedreich's ataxia; AVB-401, a gene therapy program for the treatment of BAG3-mediated dilated cardiomyopathy; and other drugs for the treatment of undisclosed cardiac diseases. It also engages in developing platform technologies, including dual gene expression, a technology for packaging multiple transgenes into one vector, as well as novel capsids. The company has collaboration and license agreement with Ultragenyx Pharmaceutical Inc. to develop and commercialize new gene therapies for Duchenne. Solid Biosciences Inc. was incorporated in 2013 and is headquartered in Charlestown, Massachusetts.