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.
Ayala Pharmaceuticals
NASDAQ:AYLAAyala Pharmaceuticals, Inc., a clinical-stage oncology company, focuses on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers. The company's lead product candidate is AL101, a potent, selective, and injectable small molecule gamma secretase inhibitor (GSI) that is in Phase II clinical trial for the treatment of recurrent/metastatic adenoid cystic carcinoma for patients bearing Notch-activating mutations. It is also involved in developing AL102, a potent, selective, and oral small molecule GSI that is in Phase II/III pivotal study for the treatment of desmoid tumors. Ayala Pharmaceuticals, Inc. has a collaboration agreement with Novartis International Pharmaceutical Limited to develop AL102 for the treatment of multiple myeloma. The company was incorporated in 2017 and is headquartered in Rehovot, Israel.
Champions Oncology
NASDAQ:CSBRChampions Oncology, Inc. engages in the development and sale of technology solutions and products to personalize the development and use of oncology drugs. Its technology platform, TumorGraft, is a novel approach to personalizing cancer care based upon the implantation of human tumors in immune-deficient mice. It uses its technology to offer solutions to Translational Oncology Solutions, which includes pharmaceutical and biotechnology companies; and Personalized Oncology, which assists physicians in developing personalized treatment options for their cancer patients. The company was founded by James M. Martell and David Sidransky on June 4, 1985 and is headquartered in Hackensack, NJ.
LogicBio Therapeutics
NASDAQ:LOGCLogicBio Therapeutics, Inc., a genetic medicine company, focuses on developing and commercializing genome editing and gene therapy treatments using its GeneRide and sAAVy platforms. The company's GeneRide technology is a new approach to precise gene insertion harnessing a cell's natural deoxyribonucleic acid; and gene delivery platform, sAAVy is an adeno-associated virus, which is designed to optimize gene delivery for treatments in a range of indications and tissues. Its lead product candidate is LB-001 that is in Phase I/II clinical trials for the treatment of methylmalonic acidemia. The company has a collaboration with Children's Medical Research Institute to develop next-generation capsids for gene therapy and gene editing applications in the liver, as well as additional tissues; and a collaboration agreement with Takeda Pharmaceutical Company Limited to develop LB-301, an investigational therapy for the treatment of Crigler-Najjar syndrome. The company also has a research collaboration, license, and option agreement with CANbridge Care Pharma Hong Kong Limited; and collaboration agreement with Daiichi Sankyo Company. The company was incorporated in 2014 and is headquartered in Lexington, Massachusetts.
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.