Novavax
NASDAQ:NVAXNovavax, Inc., a biotechnology company, that promotes improved health by discovering, developing, and commercializing vaccines to protect against serious infectious diseases. It offers vaccine platform that combines a recombinant protein approach, nanoparticle technology, and its patented Matrix-M adjuvant to enhance the immune response. It focuses on urgent health challenges, which is evaluating vaccines for COVID-19, influenza, and COVID-19 influenza combination. The company is commercializing a COVID-19 vaccine, NVX-CoV2373 under the brand names of Nuvaxovid, Covovax, and Novavax COVID-19 Vaccine, adjuvanted for adult and adolescent populations as a primary series and for both homologous and heterologous booster indications. It is also developing R21/Matrix-M adjuvant malaria vaccine. Novavax, Inc. was incorporated in 1987 and is headquartered in Gaithersburg, Maryland.
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..
Passage Bio
NASDAQ:PASGPassage Bio, Inc., a genetic medicines company, develops transformative therapies for central nervous system diseases. It develops PBGM01, a functional GLB1 gene encoding ß-galactosidase for infantile GM1; PBFT02, a functional granulin (GRN) and gene encoding progranulin (PGRN) for the treatment of FTD caused by progranulin deficiency; and PBKR03, a functional GALC gene encoding the hydrolytic enzyme galactosylceramidase for infantile Krabbe disease. The company also develops PBML04 for the treatment of metachromatic leukodystrophy; PBAL05 for the treatment of amyotrophic lateral sclerosis; and other program for huntington's disease. Passage Bio, Inc. has a strategic research collaboration with the Trustees of the University of Pennsylvania's Gene Therapy Program; and collaboration agreement, and a development services and clinical supply agreement with Catalent Maryland, Inc. The company was incorporated in 2017 and is headquartered in Philadelphia, Pennsylvania.
Sangamo Therapeutics
NASDAQ:SGMOSangamo Therapeutics, Inc., a clinical-stage genomic medicine company, focuses on translating science into medicines that transform the lives of patients and families afflicted with serious diseases in the United States. The company's clinical-stage product candidates are ST-920, a gene therapy product candidate, which is in Phase 1/2 clinical study for the treatment of Fabry disease; TX200, a chimeric antigen receptor engineered regulatory T cell (CAR-Treg) therapy product candidate that is in Phase 1/2 clinical study for the prevention of immune-mediated rejection in HLA-A2 mismatched kidney transplantation; SB-525, a gene therapy product candidate, which is in Phase 3 clinical trial for the treatment of moderately severe to severe hemophilia A; BIVV003, a zinc finger nuclease gene-edited cell therapy product candidate that is in Phase 1/2 PRECIZN-1 clinical study for the treatment of sickle cell disease. Its preclinical development products focus on CAR-Treg cell therapies for autoimmune disorders and genome engineering for neurological diseases. Sangamo Therapeutics, Inc. has collaborative and strategic partnerships with Biogen MA, Inc.; Kite Pharma, Inc.; Pfizer Inc.; Sanofi S.A.; Novartis Institutes for BioMedical Research, Inc.; Shire International GmbH; Dow AgroSciences LLC; Sigma-Aldrich Corporation; Genentech, Inc.; Open Monoclonal Technology, Inc.; F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc.; and California Institute for Regenerative Medicine. The company was formerly known as Sangamo BioSciences, Inc. and changed its name to Sangamo Therapeutics, Inc. in January 2017. Sangamo Therapeutics, Inc. was incorporated in 1995 and is headquartered in Brisbane, California.
Telesis Bio
NASDAQ:TBIOTelesis Bio, Inc., a synthetic biology company, manufactures and sells synthetic biology instruments, reagents, and associated products and related services, primarily to pharmaceutical and academic laboratories worldwide. Its solutions include BioXp system, including BioXp 3250 system, BioXp 9600 system, and BioXp DBC, which are empowers researchers to go from a digital DNA sequence to endpoint-ready synthetic DNA; BioXp portal, an online portal that offers an intuitive guided workflow and design tools for building new DNA sequences and assembling them into vectors of choice; BioXp kits, such as BioXp De Novo kits, BioXp select kits, BioXp next generation sequencing kits, and BioXp DBC kits, which contains building blocks and reagents, including its Gibson Assembly branded reagents, for specific synthetic biology workflow applications; Benchtop reagents that contain all the reagents necessary to proceed with a specific synthetic biology workflow on the benchtop using products generated on the BioXp system; and short oligo ligation assembly enzymatic DNA synthesis designs to significantly reduce timelines for constructing synthetic DNA, RNA, and proteins compared to traditional chemical synthesis, paving the way for more efficient and effective development of mRNA-based vaccines, diagnostics, therapeutics, and personalized medicines. It also serves government institutions, contract research organizations, and synthetic biology companies. The company was formerly known as Codex DNA, Inc. and changed its name to Telesis Bio Inc. in November 2022. Telesis Bio, Inc. was incorporated in 2011 and is headquartered in San Diego, California.