Applied Therapeutics
NASDAQ:APLTApplied Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the development of a pipeline of novel product candidates against validated molecular targets in indications of high unmet medical need in the United States. The company's lead product candidate is AT-007 (also called govorestat) that has completed phase 3 for the treatment of galactosemia in healthy volunteers and adults, in pediatric clinical study for the treatment of galactosemia in kids, for treating enzyme sorbitol dehydrogenase, and for the treatment of phosphomannomutase enzyme-CDG. It also develops AT-001 (also called caficrestat) that is in phase 3 clinical trials to treat diabetic cardiomyopathy, as well as for the treatment of diabetic peripheral neuropathy; and AT-003, which is in preclinical studies for the treatment diabetic retinopathy. The company has exclusive license and supply agreement with Mercury Pharma Group Limited to commercialize drug products containing AT-007. Applied Therapeutics, Inc. was incorporated in 2016 and is headquartered in New York, New York.
Aprea Therapeutics
NASDAQ:APREAprea Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing and commercializing synthetic lethality-based cancer therapeutics that targets DNA damage response pathways. Its lead product candidate is the ATRN-119 that is in Phase I clinical trials for treating advanced solid tumors, ovarian, breast, and prostate cancers. The company is also developing ATRN-1051, ATRN-354, and APRE-DDRi for the treatment of advanced solid tumors. The company is headquartered in Doylestown, Pennsylvania.
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.
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.