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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.

Checkmate Pharmaceuticals, Inc., a clinical-stage biotechnology company, focuses on developing and commercializing novel therapeutics for the treatment of cancer. It develops CMP-001, which is in phase II clinical trial in combination with pembrolizumab to treat patients with PD-1 refractory melanoma; combination with nivolumab to treat patients with PD-1 naïve neoadjuvant melanoma; and treatment in patients with PD-1 refractory melanoma. Checkmate Pharmaceuticals, Inc. has strategic alliances with Merck KGaA and Pfizer. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Genfit S.A., a biopharmaceutical company, discovers and develops drug candidates and diagnostic solutions for metabolic and liver-related diseases. The company develops Elafibranor, which is in Phase III clinical trial to treat patients with primary biliary cholangitis. It also engages in the development of NIS4 technology for the diagnosis of nonalcoholic steatohepatitis (NASH) and fibrosis; VS-01 for the treatment of Urea Cycle Disorder (UCD) and Organic Acidemia Disorder (OAD); GNS561, which is in Phase 1b/2 trial to treat patients with cholangiocarcinoma (CCA); and VS-01-ACLF and Nitazoxanide (NTZ), which is in Phase 1 trial to treat acute-on-chronic liver failure, as well as VS-02-HE, which is in preclinical trial for the treatment of Reduction of Hyperammonemia and the Stabilization of Blood Ammonia. The company has a licensing agreement with Labcorp for the commercialization of NASHnext, a blood-based molecular diagnostic test; and Genoscience Pharma to develop and commercialize the investigational treatment GNS561 for CCA. The company was incorporated in 1999 and is headquartered in Loos, France.

Protara Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in advancing transformative therapies for the treatment of cancer and rare diseases. The company's lead program is TARA-002, an investigational cell therapy for the treatment of lymphatic malformations. It also develops intravenous choline chloride, an investigational phospholipid substrate replacement therapy for the treatment of intestinal failure associated liver disease. The company was formerly known as ArTara Therapeutics, Inc. and changed its name to Protara Therapeutics, Inc. in May 2020. The company is headquartered in New York, New York.

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.