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Gemphire Therapeutics Inc., a clinical-stage biopharmaceutical company, focuses on developing and commercializing therapies for the treatment of dyslipidemia and nonalcoholic fatty liver disease (NAFLD/NASH). The company is developing gemcabene, a novel, once-daily, and oral therapy for dyslipidemia conditions where patients are unable to reach their lipid lowering goals, including patients already receiving maximally tolerated statin therapy. It also completed three Phase IIb clinical trials for gemcabene in homozygous familial hypercholesterolemia (HoFH) and hypercholesterolemia, including heterozygous familial hypercholesterolemic (HeFH) and atherosclerotic cardiovascular disease (ASCVD) patients on maximally tolerated statins, and severe hypertriglyceridemia (SHTG). The company was founded in 2008 and is headquartered in Livonia, Michigan.

IDEAYA Biosciences, Inc., a synthetic lethality-focused precision medicine oncology company, discovers and develops targeted therapeutics for patient populations selected using molecular diagnostics in the United States. The company's products in development include IDE196, a protein kinase C inhibitor that is in Phase 2/3 clinical trials for genetically defined cancers having GNAQ or GNA11 gene mutations; IDE397, a methionine adenosyltransferase 2a inhibitor that is in Phase 1/2 clinical trials for patients with solid tumors having methylthioadenosine phosphorylase gene deletions, such as non-small cell lung, bladder, gastric, and esophageal cancers; IDE161, a poly ADP-ribose glycohydrolase inhibitor that is in Phase 1 clinical trial to treat tumors with homologous recombination deficiency (HRD), and other genetic or molecular signatures; GSK101, a Pol Theta Helicase inhibitor that is in Phase 1 clinical trial for the treatment of tumors with BRCA or other homologous recombination, and HRD mutations; and Werner Helicase inhibitors for tumors with high microsatellite instability. It offers preclinical research programs focused on pharmacological inhibition; DECIPHER dual CRISPR library for synthetic lethality target and biomarker discovery; and INQUIRE chemical library and HARMONY machine-learning engines to enhance its drug discovery platform. The company has strategic alliances with GlaxoSmithKline, Pfizer Inc., Novartis International Pharmaceuticals Ltd., Amgen Inc., Gilead Sciences, Inc., Cancer Research UK, and the University of Manchester. IDEAYA Biosciences, Inc. was incorporated in 2015 and is headquartered in South San Francisco, California.

IVERIC bio, Inc., a biopharmaceutical company, focuses on the discovery and development of novel treatments for retinal diseases with unmet medical needs. The company develops Zimura, an inhibitor of complement factor C5 comprising GATHER1, which has completed Phase 2/3 clinical trial; GATHER2 that is in Phase 3 clinical trial for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD); and STAR or OPH2005 that is in Phase 2b clinical trial for the treatment of autosomal recessive Stargardt disease. Its preclinical product candidatures include IC-500, a high temperature requirement A serine peptidase 1 protein inhibitors for the treatment of GA and other age-related retinal diseases. The company also develops OPH2001 that has completed Phase 1/2a clinical trial of various doses of avacincaptad pegol (ACP) administered as a monotherapy for the treatment of GA; OPH2000, which has completed Phase 1/2a clinical trial of various doses of ACP administered in combination with Lucentis for the treatment of wet AMD; OPH2007 that has completed Phase 2a clinical trial for the treatment of wet AMD; and OPH2002, which has completed Phase 2a clinical trial for the treatment of idiopathic polypoidal choroidal vasculopathy. In addition, its minigene programs comprises miniCEP290 program for LCA10; miniABCA4 Program for STGD1; and miniUSH2A Program for usher syndrome type 2A-Related inherited retinal diseases (IRDs). The company was formerly known as Ophthotech Corporation and changed its name to IVERIC bio, Inc. in April 2019. The company was incorporated in 2007 and is based in Parsippany, New Jersey. As of July 11, 2023, IVERIC bio, Inc. operates as a subsidiary of Astellas US Holding, Inc.

KalVista Pharmaceuticals, Inc., a clinical stage pharmaceutical company, engages in the discovery, development, and commercialization of small molecule protease inhibitors for diseases with unmet needs in the United States. The company's product portfolio comprises small molecule plasma kallikrein inhibitors targeting hereditary angioedema (HAE) and diabetic macular edema (DME); and oral plasma kallikrein inhibitors. Its products include sebetralstat, which is initiation of the Phase 3 KONFIDENT trial as a potential oral, on-demand therapy for HAE attacks; and Factor XIIa, an oral inhibitor program which is in preclinical stage targets an enzyme in HAE. The company is headquartered in Cambridge, Massachusetts.

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.