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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.

Morphic Holding, Inc., a biopharmaceutical company, discovers and develops oral small-molecule integrin therapeutics for the treatment of autoimmune, cardiovascular, and metabolic diseases, as well as fibrosis and cancer. Its lead product candidate MORF-057, an a4ß7-specific integrin inhibitor affecting inflammation is in Phase 2 clinical trials for the treatment of inflammatory bowel disease. The company's products in pipeline include Next Gen a4ß7 Inhibitors for additional potential GI indications, such as EGIDs, pouchitis, etc.; avß8 for the treatment of Myelofibrosis and solid tumor; and fibronectin integrin for pulmonary hypertensive. In addition, it has a license agreement with Janssen to discover and develop novel integrin therapeutics; collaboration agreement with Schrödinger for integrin targets; and license agreement with Children's Medical Center Corporation to develop and commercialize products worldwide for any therapeutic or diagnostic use in humans and veterinary applications. Morphic Holding, Inc. was incorporated in 2014 and is headquartered in Waltham, Massachusetts.

Cassava Sciences, Inc., a clinical stage biotechnology company, develops drugs for neurodegenerative diseases. Its lead therapeutic product candidate is simufilam, a small molecule drug, which is completed Phase 2 clinical trial; and investigational diagnostic product candidate is SavaDx, a blood-based biomarker/diagnostic to detect Alzheimer's disease. The company was formerly known as Pain Therapeutics, Inc. and changed its name to Cassava Sciences, Inc. in March 2019. Cassava Sciences, Inc. was incorporated in 1998 and is based in Austin, Texas.

Xenon Pharmaceuticals Inc., a clinical-stage biopharmaceutical company, engages in developing therapeutics to treat patients with neurological disorders in Canada. Its clinical development pipeline includes XEN496, a Kv7 potassium channel opener that is Phase III clinical trials for the treatment of KCNQ2 developmental and epileptic encephalopathy; and XEN1101, a Kv7 potassium channel opener, which is in Phase II clinical trial for the treatment of epilepsy and other neurological disorders. The company's product candidates also comprise NBI-921352, a selective Nav1.6 sodium channel inhibitor that is in Phase II clinical trials for the treatment of SCN8A developmental and epileptic encephalopathy, and other indications, including adult focal epilepsy. It has a license and collaboration agreement with the Neurocrine Biosciences, Inc. to develop treatments for epilepsy. The company was incorporated in 1996 and is headquartered in Burnaby, Canada.

Zogenix, Inc. is a pharmaceutical company. The firm engages in the development and commercialization of central nervous system (CNS) therapies and products for the treatment orphan diseases and other CNS disorders. Its products include Fintepla and MT1621. The company was founded by Stephen James Farr, Cam L. Garner, Roger L. Hawley, Bret E. Megargel, Jonathan M. N. Rigby, Scott L. Glenn, and John J. Turanin on May 11, 2006 and is headquartered in Emeryville, CA.