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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.

Ayala Pharmaceuticals, Inc., a clinical-stage oncology company, focuses on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers. The company's lead product candidate is AL101, a potent, selective, and injectable small molecule gamma secretase inhibitor (GSI) that is in Phase II clinical trial for the treatment of recurrent/metastatic adenoid cystic carcinoma for patients bearing Notch-activating mutations. It is also involved in developing AL102, a potent, selective, and oral small molecule GSI that is in Phase II/III pivotal study for the treatment of desmoid tumors. Ayala Pharmaceuticals, Inc. has a collaboration agreement with Novartis International Pharmaceutical Limited to develop AL102 for the treatment of multiple myeloma. The company was incorporated in 2017 and is headquartered in Rehovot, Israel.

Protalix BioTherapeutics, Inc., a biopharmaceutical company, engages in the development, production, and commercialization of recombinant therapeutic proteins based on its proprietary ProCellEx plant cell-based protein expression system in the United States, Australia, Canada, Israel, Brazil, Russia, Turkey, and internationally. The company offers Elelyso for the treatment of Gaucher disease; and Elfabrio for the treatment of adult patients with a confirmed diagnosis of Fabry disease. It is also developing PRX-115, a plant cell-expressed recombinant PEGylated Uricase which is in Phase I trial for the treatment of gout; and PRX-119, a plant cell-expressed PEGylated recombinant human DNase I product candidate that is in preclinical phase for the treatment of NETs-related diseases. The company has agreements and partnerships with Pfizer; Fundação Oswaldo Cruz; and Chiesi Farmaceutici S.p.A. The company was incorporated in 1993 and is headquartered in Karmiel, Israel. Protalix BioTherapeutics, Inc. is a subsidiary of Protalix Ltd.

TRACON Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel targeted therapeutics for cancer in the United States. Its clinical stage products include envafolimab (KN035), a PD-L1 single-domain antibody for the treatment of refractory soft tissue sarcoma; and YH001, an investigational humanized cytotoxic T-lymphocyte-associated protein 4 IgG1 monoclonal antibody that is in Phase I clinical trial for the treatment of various cancer indications. The company's clinical stage products also include TRC102, a small molecule that is in Phase II clinical trial for the treatment of mesothelioma, and in Phase I clinical trial to treat solid tumors, lung cancer, and glioblastoma; and TJ004309, a CD73 antibody that is in Phase I clinical development for the treatment of solid tumors. In addition, it is developing bispecific antibodies, which are in preclinical stage. It has collaboration and license agreements with 3D Medicines Co., Ltd. and Jiangsu Alphamab Biopharmaceuticals Co., Ltd. for the development of envafolimab; I-Mab Biopharma for the development of CD73 antibody TJ004309 and bispecific antibodies; and cooperative research and development agreement with National Cancer Institute. The company was formerly known as Lexington Pharmaceuticals, Inc. and changed its name to TRACON Pharmaceuticals, Inc. in March 2005. TRACON Pharmaceuticals, Inc. was incorporated in 2004 and is headquartered in San Diego, California.