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Chimerix, Inc., a biopharmaceutical company, develops medicines to improve and extend the lives of patients facing deadly diseases. Its pipeline products include ONC201 a program that is in Phase 3 clinical trial for treating patients with H3 K27M-mutant diffuse glioma, as well as in Phase 2 clinical trial for the treatment of rare neuroendocrine tumors; and ONC206, an imipridone, Dopamine Receptor D2 (DRD2) antagonist, and caseinolytic protease P (ClpP) agonist, which is in Phase 1 clinical trial for adult and pediatric patients with primary central nervous system tumors. The company also develops ONC212, an imipridone agonist of the orphan G protein-coupled receptors (GPCR) tumor suppressor GPR132, as well as ClpP for oncology indications; and CMX521, a nucleoside analog antiviral drug candidate for the treatment of SARS-CoV-2. It has license agreement with SymBio Pharmaceuticals to develop and commercialize TEMBEXA for human diseases other than orthopoxviruses, including smallpox. Chimerix, Inc. was incorporated in 2000 and is headquartered in Durham, North Carolina.

Kezar Life Sciences, Inc., a clinical-stage biotechnology company, engages in the discovery and development of novel small molecule therapeutics to treat unmet needs in immune-mediated diseases and cancer in the United States. The company's lead product candidate is zetomipzomib (KZR-616), a selective immunoproteasome inhibitor that is in Phase 2b clinical trials for various indications, including lupus nephritis, dermatomyositis, and polymyositis; Phase 1b clinical trials in systemic lupus erythematosus; and completed Phase 2a clinical trials in lupus nephritis. Its preclinical products include KZR-261, a novel first-in-class protein secretion inhibitor for the treatment of tumors resistant to traditional chemotherapeutics. Kezar Life Sciences, Inc. was incorporated in 2015 and is headquartered in South San Francisco, California.

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.

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.

Strongbridge Biopharma Plc operates as a commercial-stage biopharmaceutical company, which focuses on the development and commercialization of therapies for rare diseases. Its pipeline is comprised of Keveyis, Macrilen, Recorlev, and Veldoreotide. The company was founded in 1996 and is headquartered in Trevose, PA.