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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.

Gamida Cell Ltd., a clinical-stage biopharmaceutical company, develops cell therapies to cure blood cancers and serious blood diseases. The company's lead product candidate is omidubicel, a cell therapy that has completed Phase III clinical trial in patients with hematologic malignancies, as well as in Phase II clinical trials in patients with severe aplastic anemia. It is also developing GDA-201, an investigational NK cell-based cancer immunotherapy, which is in Phase I/II studies for the treatment of relapsed or refractory non-Hodgkin lymphoma and multiple myeloma. The company was incorporated in 1998 and is headquartered in Jerusalem, Israel.

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.

Voyager Therapeutics, Inc., a biotechnology company, focuses on the treatment of gene therapy and neurology diseases. The company's lead clinical candidate is VY-TAU01, an anti-tau antibody program for the treatment of alzheimer's disease. Its product pipeline includes superoxide dismutase 1 silencing gene therapy, which is in preclinical trial for the treatment of amyotrophic lateral sclerosis; tau silencing gene therapy, which is in preclinical trial for the treatment of alzheimer's disease; and vectorized anti-amyloid antibody, a gene therapy targeting anti-amyloid for the treatment of alzheimer's disease and is in preclinical trial. In addition, the company develops VY-FXN01, which is in preclinical trial to treat friedreich's ataxia; and GBA1 gene replacement to treat parkinson's disease and is in preclinical trial. Further, it provides research program for the treatment of Huntington's disease. The company has collaboration and license agreements with Alexion; AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc; and Sangamo Therapeutics, Inc. Voyager Therapeutics, Inc. was incorporated in 2013 and is headquartered in Lexington, Massachusetts.