Monte Rosa Therapeutics
NASDAQ:GLUEMonte Rosa Therapeutics, Inc., a clinical-stage biotechnology company, engages in the development of novel small molecule precision medicines that employ the body's natural mechanisms to selectively degrade therapeutically relevant proteins. The company develops MRT-2359, an orally bioavailable molecular glue degrader targeting the translation termination factor protein GSPT1 for the treatment of MYC-driven tumors; MRT-6160 for the treatment of systemic and central nervous system autoimmune diseases; and MRT-8102 for the treatment of IL-1?/NLRP3 driven inflammatory diseases. It also develops CDK2 to treat ovarian, uterine, and breast cancers. The company was incorporated in 2019 and is headquartered in Boston, Massachusetts.
Ocugen
NASDAQ:OCGNOcugen, Inc., a clinical-stage biopharmaceutical company, focuses on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve patients' health. The company's pipeline product includes OCU400, a novel gene therapy product candidate restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases, currently under Phase 3 trials for the treatment of retinitis pigmentosa and Phase 1/2 trials for the treatment of leber congenital amaurosis; OCU410, a gene therapy under phase 1/2 for the treatment of dry age-related macular degeneration (AMD); and OCU410ST, a gene therapy under phase 1/2 for the treatment of Stargardt disease. It is also involved in the development of OCU200, a novel fusion protein that is in preclinical development stage for the treatment of diabetic macular edema, diabetic retinopathy, and wet age-related macular degeneration; and NeoCart, an autologous chondrocyte-derived neocartilage, currently under Phase 3 studies indicated for the repair of knee cartilage injuries in adult. In addition, the company is developing OCU500, a COVID-19 vaccine; OCU510, a seasonal quadrivalent flu vaccine; and OCU520, a combination quadrivalent seasonal flu and COVID-19 vaccine. It has collaboration agreements with National Institute of Allergy and Infectious Diseases for early clinical studies for the OCU500 program; and a strategic partnership with CanSino Biologics Inc. for manufacturing its modifier gene therapy pipeline product candidates. The company was founded in 2013 and is headquartered in Malvern, Pennsylvania.
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.
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.; 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 Richmond, California.
Valneva
NASDAQ:VALNValneva SE, a specialty vaccine company, develops, manufactures, and commercializes prophylactic vaccines for infectious diseases with unmet needs. It offers IXIARO, an inactivated Vero cell culture-derived Japanese encephalitis vaccine indicated for active immunization against Japanese encephalitis; DUKORAL, an oral vaccine for the prevention of diarrhea caused by Vibrio cholera and/or heat-labile toxin producing enterotoxigenic Escherichia coli bacterium; IXCHIQ, a single-dose, live-attenuated vaccine for the prevention of disease caused by chikungunya virus; and VLA2001, an inactivated whole-virus COVID-19 vaccine. The company also develops VLA15, a vaccine candidate, which is in Phase III clinical trial against Borrelia, the bacterium that causes Lyme disease; VLA1553, a vaccine candidate, which is in Phase III clinical trial against the chikungunya virus; VLA1554, a vaccine candidate targeting human metapneumovirus; and VLA2112, a vaccine candidate to treat patients with epstein-barr virus. It sells its products in the United States, Canada, Germany, Austria, Nordics, the United Kingdom, France, rest of European countries, and internationally. Valneva SE has collaborations with Pfizer, Inc. to co-develop and commercialize its Lyme disease vaccine; and Instituto Butantan for the development, manufacturing, and marketing of single-shot chikungunya vaccine. The company was founded in 1998 and is based in Saint-Herblain, France.