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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.

Fennec Pharmaceuticals Inc., a biopharmaceutical company, develops product candidates for use in the treatment of cancer in the United States. Its lead product candidate is the Sodium Thiosulfate, which has completed the Phase III clinical trial for the prevention of cisplatin induced hearing loss or ototoxicity in children. The company was formerly known as Adherex Technologies Inc. and changed its name to Fennec Pharmaceuticals Inc. in September 2014. Fennec Pharmaceuticals Inc. was founded in 1996 and is based in Research Triangle Park, North Carolina.

INmune Bio, Inc., a clinical-stage immunology company, focuses on developing drugs to reprogram the patients innate immune system to treat disease in the United States. It intends to develop and commercialize product candidates to treat hematologic malignancies, solid tumors, and chronic inflammation. The company's development programs include INKmune, which is in Phase 1 for the treatment of patients with high-risk myelodysplastic syndrome; and INB03 develop pre-clinical data in cancer that express MUC4, a mucinous polyglucan on the surface of some epithelial cancer cells that appears to predict resistant to immunotherapy, including women with MUC4 expressing HER2+ breast cancer and other MUC4 resistant cancers. It also provides XPro1595 for the treatment of Alzheimer's disease and Treatment Resistant Depression. It has license agreements with Xencor, Inc.; Immune Ventures, LLC; and University of Pittsburg. INmune Bio, Inc. was incorporated in 2015 and is headquartered in Boca Raton, Florida.

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.