C4 Therapeutics
NASDAQ:CCCCC4 Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops novel therapeutic candidates to degrade disease-causing proteins for the treatment of cancer, neurodegenerative conditions, and other diseases. Its lead product candidate is CFT7455, an orally bioavailable MonoDAC degrader of protein that is in Phase 1/2 trial targeting IKZF1 and IKZF3 for multiple myeloma and non-Hodgkin lymphomas, including peripheral T-cell lymphoma and mantle cell lymphoma, currently under Phase 1/2 clinical trials. The company is also developing CFT1946, an orally bioavailable BiDAC degrader targeting V600X mutant BRAF to treat melanoma, non-small cell lung cancer (NSCLC), colorectal cancer, and other solid malignancies, currently under Phase 1/2 clinical trials; and CFT8919, an orally bioavailable, allosteric, and mutant-selective BiDAC degrader of epidermal growth factor receptor, or EGFR, with an L858R mutation in NSCLC. It has strategic collaborations with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc.; Biogen MA, Inc.; Betta Pharmaceuticals, Co., Ltd.; and Merck Sharp & Dohme, LLC, as well as Calico Life Sciences LLC. The company was incorporated in 2015 and is headquartered in Watertown, 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.
Replimune Group
NASDAQ:REPLReplimune Group, Inc., a clinical-stage biotechnology company, focuses on the development of oncolytic immunotherapies to treat cancer. The company's proprietary tumor-directed oncolytic immunotherapy product candidates are designed and intended to activate the immune system against cancer. Its lead product candidate is RP1, a selectively replicating version of HSV-1 that expresses GALV-GP R(-) and human GM-CSF, which is in Phase I/II clinical trials for a range of solid tumors; and that has completed Phase II clinical trials for treating cutaneous squamous cell carcinoma. The company is also developing RP2, which is in Phase I clinical trials to express an anti-CTLA-4 antibody-like protein to block the inhibition of the immune response otherwise caused by CTLA-4; and RP3 that is in Phase I clinical trial to express immune-activating proteins that stimulate T cells. Replimune Group, Inc. was founded in 2015 and is headquartered in Woburn, Massachusetts.
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.