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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.

CASI Pharmaceuticals, Inc., a biopharmaceutical company, develops and commercializes therapeutics and pharmaceutical products in the People's Republic of China, the United States, and internationally. It offers EVOMELA, an intravenous formulation of melphalan for use as a conditioning treatment prior to stem cell transplantation, and as a palliative treatment for patients with multiple myeloma. The company's other hematology/oncology assets in pipeline include CNCT 19, an autologous CD19 CAR-T investigative product; BI-1206, novel anti-Fc?RIIB antibody, for the treatment of solid tumors and relapsed/refractory non-hodgkin lymphoma; CB-5339, a novel VCP/p97 inhibitor that focuses on valosin-containing protein (VCP)/p97 as a novel target in protein homeostasis, DNA damage response, and other cellular stress pathways for therapeutic use in the treatment of patients with various malignancies; and CID-103, a human IgG1 anti-CD38 monoclonal antibody. The company has licensing agreements with Juventas Biotechnology (Tianjin) Co., Ltd.; BioInvent International AB; Black Belt Therapeutics Limited; Cleave Therapeutics, Inc.; and Acrotech Biopharma L.L.C. to develop and commercialize its commercial product EVOMELA. It also has distribution agreements with China Resources Pharmaceutical Commercial Group International Trading Co., Ltd; Pharmathen Global BV; and Riemser Pharma GmbH. CASI Pharmaceuticals, Inc. was incorporated in 1991 and is based in Beijing, the People's Republic of China.

Entera Bio Ltd., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of orally delivered peptide and protein therapeutics for unmet medical needs. The company's lead product candidates include the EB612, which is in Phase II clinical trials for the treatment of hypoparathyroidism; and EB613 that has completed Phase II clinical trials for the treatment of osteoporosis, as well as is in Phase I clinical trials for the treatment of non-union fractures. Its other product candidate is GLP-2, which is in preclinical trial for the treatment of short bowl syndrome; and hGh for the GH deficiency. The company has a research collaboration and license agreement with the Amgen Inc. for the development and discovery of clinical candidates in the field of inflammatory disease and other serious illnesses. Entera Bio Ltd. was incorporated in 2009 and is based in Jerusalem, Israel.

Aceragen, Inc. is a clinical stage biopharmaceutical company, which focuses on the clinical development, and commercialization of drug candidates for both oncology and rare disease indications. The company was founded by Paul C. Zamecnik, Sudhir A. Agrawal, and James B. Wyngaarden on May 25, 1989 and is headquartered in Exton, PA.