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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.

Axcella Health Inc. operates as a clinical-stage biotechnology company in the United States. The company treats complex diseases using endogenous metabolic modulators. Its product pipeline includes AXA1125 that is in Phase 2a clinical trial for long COVID therapy for patients, as well as Phase 2b clinical trial for treating non-alcoholic steatohepatitis; and AXA1665, which is in Phase 2 clinical trial for the reduction in risk of overt hepatic encephalopathy recurrence. The company was formerly known as Newco LS16, Inc. and changed its name to Axcella Health Inc. in June 2016. Axcella Health Inc. was incorporated in 2008 and is based in Cambridge, Massachusetts.

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 reprogramming the patient's innate immune system to treat cancer and neurodegenerative diseases. 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 focuses on treating women with relapse refractory ovarian carcinoma solid tumor and patients with high-risk myelodysplastic syndrome; INB03 to treat patients with cancers 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; and 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. The company was incorporated in 2015 and is headquartered in Boca Raton, Florida.

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.