Ardelyx
NASDAQ:ARDXArdelyx, Inc., a biopharmaceutical company, discovers, develops, and commercializes medicines to treat gastrointestinal and cardiorenal therapeutic areas in the United States and internationally. The company's lead product candidate is tenapanor for the treatment of patients with irritable bowel syndrome with constipation. It also develops XPHOZAH, which is in Phase III clinical trial to reduce serum phosphorus in adults with chronic kidney disease (CKD)on dialysis, or hyperphosphatemia; RDX013, a potassium secretagogue, for the treatment of elevated serum potassium, or hyperkalemia, a problem among patients with kidney and/or heart failure; and RDX020, for adult patients with metabolic acidosis, a serious electrolyte disorder. The company has agreements with Kyowa Kirin, Fosun Pharmaceutical Industrial Development Co. Ltd., and Knight Therapeutics, Inc. for the development and commercialization of tenapanor in their respective territories. The company was formerly known as Nteryx, Inc. and changed its name to Ardelyx, Inc. in June 2008. Ardelyx, Inc. was incorporated in 2007 and is headquartered in Waltham, Massachusetts.
Athersys
NASDAQ:ATHXAthersys, Inc., a biotechnology company, focuses on the research and development activities in the field of regenerative medicine. Its clinical development programs are focused on treating neurological conditions, cardiovascular diseases, inflammatory and immune disorders, and pulmonary and other conditions. The company's lead platform product includes MultiStem cell therapy, an allogeneic stem cell product for the treatment of patients suffering from neurological damage from an ischemic stroke, as well as for acute respiratory distress syndrome, trauma complications, HSC transplant support, and other indications. It also develops MultiStem cell therapy to promote tissue repair and healing for animal patients. The company has license and collaboration agreements with Healios K.K. to develop and commercialize MultiStem cell therapy for ischemic stroke, acute respiratory distress syndrome, and ophthalmological indications, as well as for the treatment of liver, kidney, pancreas, and intestinal tissue diseases; and the University of Minnesota to develop MultiStem cell therapy platform. The company was founded in 1995 and is headquartered in Cleveland, Ohio.
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.
Tocagen
NASDAQ:TOCATocagen Inc., a clinical-stage cancer-selective gene therapy company, focuses on developing and commercializing product candidates designed to activate a patient's immune system against their cancer. Its cancer-selective gene therapy platform is built on retroviral replicating vectors (RRVs), which are designed to deliver therapeutic genes into the DNA of cancer cells. The company's lead product candidate is Toca 511 & Toca FC that is under Phase III clinical trial for recurrent high-grade glioma. It is also developing Toca 511 & Toca FC in a Phase Ib clinical trial for intravenous treatment of advanced cancers. In addition, the company is developing other RRVs to deliver genes to cancer cells against validated immunotherapy targets. The company has a license agreement with ApolloBio to develop and commercialize Toca 511 & Toca FC; and a collaboration agreement with NRG Oncology to develop a clinical trial utilizing Toca 511 & Toca FC for the treatment of patients with newly diagnosed glioblastoma. Tocagen Inc. was founded in 2007 and is based in San Diego, California.
UroGen Pharma
NASDAQ:URGNUroGen Pharma Ltd., a biotechnology company, engages in the development and commercialization of solutions for urothelial and specialty cancers. It offers RTGel, a novel proprietary polymeric biocompatible, reverse thermal gelation hydrogel technology to improve therapeutic profiles of existing drugs; and Jelmyto for pyelocalyceal solution. The company's lead product candidate is UGN-102 for the treatment of several forms of non-muscle invasive urothelial cancer that include low-grade upper tract urothelial cancer and low-grade intermediate risk non-muscle invasive bladder cancer (NMIBC). It is also developing UGN-301 for the treatment of high-grade NMIBC. The company has license agreement with Agenus Inc. to develop, make, use, sell, import, and commercialize products of Agenus for the treatment of cancers of the urinary tract via intravesical delivery; strategic research collaboration agreement with MD Anderson focusing on the sequential use of UGN-201 and UGN-301 for the treatment of NMIBC; and licensing and supply agreement with medac Gesellschaft für klinische Spezialpräparate m.b.H. to develop UGN-103 in low-grade intermediate risk NMIBC and UGN-104 in low-grade upper tract urothelial carcinoma. UroGen Pharma Ltd. was incorporated in 2004 and is based in Princeton, New Jersey.