Applied Genetic Technologies
NASDAQ:AGTCApplied Genetic Technologies Corporation, a clinical-stage biotechnology company, develops transformational genetic therapies for patients suffering from rare and debilitating diseases. Its advanced product candidates include three ophthalmology development programs across two targets, including X-linked retinitis pigmentosa, which is in the Phase 1/2 clinical trials; and achromatopsia that is in Phase 1/2 clinical trials. It is also developing an optogenetic product candidate to treat advanced retinal disease. In addition, the company has initiated one preclinical program in otology; preclinical program in dry age-related macular degeneration; and two preclinical programs in targeting central nervous system disorders, including frontotemporal dementia and amyotrophic lateral sclerosis. It has collaboration agreements with University of Florida; Bionic Sight, Inc.; and Otonomy, Inc. The company was incorporated in 1999 and is headquartered in Alachua, Florida.
Cidara Therapeutics
NASDAQ:CDTXCidara Therapeutics, Inc., a biotechnology company, focuses on the discovery, development, and commercialization of long-acting anti-infectives for the treatment and prevention of infectious diseases and oncology in the United States. The company's lead product candidate is rezafungin acetate, a novel molecule in the echinocandin class of antifungals for the treatment and prevention of invasive fungal infections, including candidemia and invasive candidiasis, which are fungal infections associated with high mortality rates. It also advances its Cloudbreak platform to develop conjugates for the prevention and treatment of influenza and other viral infections, such as RSV, HIV, and the SARS-CoV-2 strains causing COVID-19. The company was formerly known as K2 Therapeutics, Inc. and changed its name to Cidara Therapeutics, Inc. in July 2014. The company was incorporated in 2012 and is based in San Diego, California.
ERYTECH Pharma
NASDAQ:ERYPERYTECH Pharma S.A., a biopharmaceutical company, focus on development of red blood cell-based therapeutics for cancer and orphan diseases in France and the United States. It develops eryaspase, which is in Phase 3 clinical development for the treatment of second-line pancreatic cancer, and in Phase 2 stage for the treatment of triple-negative breast cancer and second-line acute lymphoblastic leukemia patients. The company also engages in developing erymethionase, a preclinical product candidate that consists of methionine-?-lyase in red blood cells to target the amino acid metabolism of cancer cells and induce tumor starvation. ERYTECH Pharma S.A. was incorporated in 2004 and is headquartered in Lyon, France.
Protara Therapeutics
NASDAQ:TARAProtara Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in advancing transformative therapies for the treatment of cancer and rare diseases. The company's lead program is TARA-002, an investigational cell therapy for the treatment of lymphatic malformations. It also develops intravenous choline chloride, an investigational phospholipid substrate replacement therapy for the treatment of intestinal failure associated liver disease. The company was formerly known as ArTara Therapeutics, Inc. and changed its name to Protara Therapeutics, Inc. in May 2020. The company is headquartered in New York, New York.
X4 Pharmaceuticals
NASDAQ:XFORX4 Pharmaceuticals, Inc., a late-stage clinical biopharmaceutical company, focuses on the research, development, and commercialization of novel therapeutics for the treatment of rare diseases. Its lead product candidate is mavorixafor, a small molecule inhibitor of the chemokine receptor C-X-C chemokine receptor type 4 (CXCR4), which is in Phase III clinical trial for the treatment of patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome; and Phase II clinical trial to treat congenital, idiopathic, or cyclic neutropenia. The company is also developing X4P-003, a CXCR4 antagonist for the treatment of CXCR4 dependent disorders and primary immunodeficiencies; and X4P-002, a CXCR4 antagonist for the treatment of blood-brain barrier diseases. It has a license agreement with Abbisko Therapeutics Co Ltd. to develop, manufacture, and commercialize mavorixafor in combination with checkpoint inhibitors or other agents in oncology indications. The company is headquartered in Boston, Massachusetts.