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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.

Cidara Therapeutics, Inc., a biotechnology company, focuses on the discovery, development, and commercialization of long-acting anti-infectives for the treatment and prevention of infectious diseases and oncology in the United States. The company's lead product candidate is rezafungin acetate, a novel molecule in the echinocandin class of antifungals for the treatment and prevention of invasive fungal infections, including candidemia and invasive candidiasis, which are fungal infections associated with high mortality rates. It also advances its Cloudbreak platform to develop conjugates for the prevention and treatment of influenza and other viral infections, such as RSV, HIV, and the SARS-CoV-2 strains causing COVID-19. The company was formerly known as K2 Therapeutics, Inc. and changed its name to Cidara Therapeutics, Inc. in July 2014. The company was incorporated in 2012 and is based in San Diego, California.

ERYTECH Pharma S.A., a biopharmaceutical company, focus on development of red blood cell-based therapeutics for cancer and orphan diseases in France and the United States. It develops eryaspase, which is in Phase 3 clinical development for the treatment of second-line pancreatic cancer, and in Phase 2 stage for the treatment of triple-negative breast cancer and second-line acute lymphoblastic leukemia patients. The company also engages in developing erymethionase, a preclinical product candidate that consists of methionine-?-lyase in red blood cells to target the amino acid metabolism of cancer cells and induce tumor starvation. ERYTECH Pharma S.A. was incorporated in 2004 and is headquartered in Lyon, France.

Solid Biosciences Inc., a life science company, develops therapies for neuromuscular and cardiac diseases in the United States. The company's lead product candidate is SGT-003, a gene transfer candidate for the treatment of Duchenne muscular dystrophy; and SGT-501 to treat Catecholaminergic polymorphic ventricular tachycardia. It is also developing AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy; SGT-601 to treat a rare cardiac disease characterized by mutations in the gene that codes for cardiac troponin T protein, which helps coordinate contraction of the heart muscle; SGT-701 for treatment of rare inherited disease characterized by mutations in the RBM20 gene, a cardiac splicing factor that regulates alternative splicing, and codes for RNA binding motif protein 20; and AVB-202-TT to treat Friedreich's ataxia, as well as other drugs for the treatment of cardiac and other diseases. In addition, the company is involved in developing platform technologies, including capsid libraries and dual gene expression, a technology for packaging multiple transgenes into one capsid. The company has collaboration and license agreement with Ultragenyx Pharmaceutical Inc. to develop and commercialize new gene therapies for Duchenne; and a license agreement with the University of Washington, University of Missouri, and University of Florida. Solid Biosciences Inc. was incorporated in 2013 and is headquartered in Charlestown, Massachusetts.

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, which is in Phase II clinical trial for the treatment of non-muscle invasive bladder cancer and lymphatic malformations. It is also developing intravenous choline chloride, an investigational phospholipid substrate replacement therapy that is in Phase II clinical trial for patients receiving parenteral nutrition. The company is headquartered in New York, New York.