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Applied 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, 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.

Inhibikase Therapeutics, Inc., a clinical-stage pharmaceutical company, develops therapeutics for Parkinson's disease and related disorders. Its lead product candidate is IkT-148009, a small molecule Abelson tyrosine kinase inhibitor, which is in Phase 2a clinical trials to the treatment of Parkinson's disease and gastrointestinal tract. The company is also developing IkT-001Pro, a prodrug of the anticancer agent imatinib mesylate to treat stable phase chronic myelogenous leukemia. Inhibikase Therapeutics, Inc. has research and development collaborations with Johns Hopkins University, Arizona State University, University of Bordeaux, and Michigan State University, as well as University of California; and a collaborative research and development agreement with Sphaera Pharma Pte. Ltd. The company was founded in 2008 and is headquartered in Atlanta, Georgia.

LogicBio 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.