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Cerecor, Inc. is a biopharmaceutical company, which engages in the development and commercialization of treatments for rare and orphan diseases. Its lead product candidates include CERC-801, CERC-802, and CERC-803, which are therapies for inherited metabolic disorders known as congenital disorders of glycosylation. The company was founded by Blake M. Paterson, Isaac Blech, Barbara S. Slusher, and Solomon H. Snyder on January 31, 2011 and is headquartered in Rockville, MD.

NeuBase Therapeutics, Inc., a preclinical-stage biopharmaceutical company, focuses on the development of therapies to treat rare genetic diseases and cancers caused by mutant genes. The company's proprietary modular peptide-nucleic acid antisense oligo (PATrOL) platform focuses on addressing Huntington's disease (HD) and myotonic dystrophy, as well as other genetic disorders, and cancer and oncology applications. Its pipeline includes NT0100 for Huntington's disease; NT0200 for myotonic dystrophy type 1; and NT0300 treatment targets the mutated KRAS gene. NeuBase Therapeutics, Inc. is headquartered in Pittsburgh, Pennsylvania.

NuCana plc, a clinical-stage biopharmaceutical company, engages in the development of medicines to treat patients with cancer. It applies its ProTide technology to transform prescribed chemotherapy agents and nucleoside analogs into medicines. The company, through its technology, is developing ProTides medicines to overcome the limitations of nucleoside analogs and generate much higher concentrations of anti-cancer metabolites in cancer cells. Its pipeline includes NUC-3373, a chemical entity derived from the nucleoside analog 5-fluorouracil that is in Phase 1b/2 study in patients with metastatic colorectal cancer, Phase 2 clinical trial for the second-line treatment of patients with advanced colorectal cancer, and Phase 1b/2 modular clinical trial for patients with advanced solid tumors and lung cancer. It is also developing NUC-7738, a ProTide transformation of 3'-deoxyadenosine, which is in the Phase 2 part of a Phase 1/2 clinical trial for patients with advanced solid tumors. The company has an assignment, license, and collaboration agreement with Cardiff ProTides Ltd to discover, drug design, and in vitro screen purine and pyrimidine-based nucleosides as potential drug candidates. The company was formerly known as NuCana BioMed Limited and changed its name to NuCana plc in August 2017. NuCana plc was incorporated in 1997 and is headquartered in Edinburgh, the United Kingdom.

OptiNose, Inc., a specialty pharmaceutical company, focuses on the development and commercialization of products for patients treated by ear, nose, throat, and allergy specialists in the United States. The company offers XHANCE, a therapeutic product utilizing its proprietary exhalation delivery system (EDS) that delivers a topically acting corticosteroid for the treatment of chronic rhinosinusitis with nasal polyps, as well as is in Phase IIIb clinical trial for treatment of chronic sinusitis; and Onzetra Xsail, a powder EDS device. It has a license agreement with Currax Pharmaceuticals LLC for the commercialization of Onzetra Xsail. The company was founded in 2000 and is headquartered in Yardley, Pennsylvania.

Taiwan Liposome Co., Ltd. engages in the research and development of lipid-based formulation drugs. Its products are used for pain management, eye disease treatment, and cancer treatment. The company was founded by Kee Lung Hong on November 10, 1997 and is headquartered in Taipei, Taiwan.