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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.

Moleculin Biotech, Inc., a clinical stage pharmaceutical company, focuses on the development of drug candidates for the treatment of cancers and viruses. Its lead drug candidate is Annamycin, which is in Phase 1/2 studies for the treatment of relapsed or refractory acute myeloid leukemia (AML) and cancers metastasized to the lungs. The company's flagship immune/transcription modulator is WP1066 that is in Phase I clinical trial for the treatment of brain tumors and pediatric brain tumors, as well as pancreatic cancer and other malignancies. It is also developing WP1220, an analog of WP1066 for the topical treatment of cutaneous T-cell lymphoma; and WP1122 to treat glioblastoma multiforme and COVID-19. The company was incorporated in 2015 and is headquartered in Houston, Texas.

Proteostasis Therapeutics, Inc., a clinical stage biopharmaceutical company, engages in the discovery and development of novel therapeutics to treat cystic fibrosis. The company's lead product candidates for the treatment of CF include PTI-801, a cystic fibrosis transmembrane conductance regulator (CFTR) corrector agent; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. It has collaboration agreements with Cystic Fibrosis Foundation, Inc. to research, develop, and commercialize products for the treatment of CF, non-classical CF, and other pulmonary diseases in the United States or the European Union; and Genentech, Inc. for licensing the technology and materials relating to therapeutic small molecule modulators. The company was formerly known as Proteoguard, Inc. and changed its name to Proteostasis Therapeutics, Inc. in September 2007. Proteostasis Therapeutics, Inc. was founded in 2006 and is headquartered in Boston, Massachusetts.

Zafgen, Inc., a clinical-stage biopharmaceutical company, develops therapies for patients suffering from metabolic diseases comprising type 2 diabetes, Prader-Willi syndrome (PWS), and other metabolically related disorders. The company's lead product candidate is ZGN-1061, a fumagillin-class methionine aminopeptidase 2 (MetAP2) inhibitor administered by subcutaneous injection, which is in Phase 2 clinical trial and profiled for its utility in the treatment of type 2 diabetes and other related metabolic disorders. It also develops ZGN-1258, a MetAP2 inhibitor for treatment of PWS. Zafgen, Inc. was founded in 2005 and is headquartered in Boston, Massachusetts.

Zosano Pharma Corporation, a clinical stage biopharmaceutical company, focuses on providing therapeutics and other bioactive molecules to patients suffering from migraine using its transdermal microneedle system. Its lead product candidate is Qtrypta (M207), a proprietary formulation of zolmitriptan used for the acute treatment of migraine. The company was founded in 2006 and is headquartered in Fremont, California.