Homology Medicines
NASDAQ:FIXXHomology Medicines, Inc., a genetic medicines company, focuses on transforming the lives of patients suffering from rare genetic diseases in the United States. The company offers proprietary platform is designed to utilize its human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs) to deliver genetic medicines in vivo either through a nuclease-free gene editing mobility, gene therapy or for various genetic disorders. Its earlier stage product candidates comprising HMI-202, an investigational gene therapy which completed IND-enabling studies for the treatment of metachromatic leukodystrophy; and HMI-104 which completed IND-enabling studies. Homology Medicines, Inc. was incorporated in 2015 and is headquartered in Bedford, Massachusetts.
Frequency Therapeutics
NASDAQ:FREQFrequency Therapeutics, Inc., a clinical-stage biotechnology company, focuses on developing therapeutics to activate a person's innate regenerative potential to restore function. Its Progenitor Cell Activation approach uses small molecules to activate progenitor cells within the body to create functional tissue. The company's lead product candidate is FX-322, which is in Phase IIb clinical trial to treat the underlying cause of sensorineural hearing loss. It is also developing medicines for patients across a range of degenerative conditions, including multiple sclerosis, and diseases of the muscle, gastrointestinal tract, skin, and bone. The company has a license and collaboration agreement with Astellas Pharma Inc. for the development and commercialization of FX-322, as well as collaboration and licensing agreements with Massachusetts Eye and Ear, the Massachusetts Institute of Technology, and The Scripps Research Institute. Frequency Therapeutics, Inc. was incorporated in 2014 and is headquartered in Lexington, Massachusetts.
Keros Therapeutics
NASDAQ:KROSKeros Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops and commercializes novel therapeutics for patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta family of proteins in the United States. The company's lead product candidate is KER-050, which is being developed for the treatment of low blood cell counts, or cytopenias, including anemia and thrombocytopenia in patients with myelodysplastic syndromes, as well as in patients with myelofibrosis. It also develops KER-012, which is in Phase II clinical trial to treat pulmonary arterial hypertension and cardiovascular disorders; and KER-065 that is in Phase I clinical trial for the treatment of obesity and neuromuscular diseases. In addition, the company has collaboration and license agreement with Hansoh (Shanghai) Healthtech Co., Ltd. to develop, manufacture, and commercialize KER-050 and licensed products containing KER-050. Keros Therapeutics, Inc. was incorporated in 2015 and is headquartered in Lexington, Massachusetts.
Provention Bio
NASDAQ:PRVBProvention Bio, Inc. is a clinical-stage biopharmaceutical company, which engages in the development and commercialization of novel therapeutics and solutions. Its products include PRV-031 for the interception of type 1 diabetes (T1D), PRV-015 for the treatment of gluten-free diet non-responding celiac disease, PRV-6527 for Crohn's disease, PRV-3279 for the treatment of lupus, and PRV-101 for the prevention of acute coxsackie virus B (CVB), and the prevention of type 1 diabetes (T1D) onset. The company was founded by Francisco Leon and Ashleigh Palmer on October 4, 2016 and is headquartered in Red Bank, NJ.
ZIOPHARM Oncology
NASDAQ:ZIOPZIOPHARM Oncology, Inc. is a biopharmaceutical company, which engages in the development, acquisition, and commercialization of immuno-oncology platforms that leverage cell- and gene-based therapies to treat patients with cancer. Its pipeline includes Sleeping Beauty TCR-T Targeting neoantigens; Ad-RTS-hlL-12 + veledimex; and Sleeping Beauty CAR-T. The company was founded on September 9, 2003 and is headquartered in Boston, MA.