Opus Genetics, Inc. Common Stock
Opus Genetics, Inc., a clinical-stage ophthalmic biopharmaceutical company, focuses on developing and commercializing therapies for the treatment of inherited retinal diseases (IRDs). It develops OPGx-LCA5, an early-onset retinal degeneration that is in Phase 1/2 clinical trial for the treatment of LCA5-associated IRD; and OPGx-BEST1 to treat BEST1-associated retinal disease. The company also develops various pre-clinical IRD programs, including OPGx-RHO, a gene therapy that targets autosomal dominant retinitis pigmentosa caused by RHO mutations; OPGx-RDH12 that is designed to restore protein expression and halt functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase gene; OPGx-MERTK, an AAV vector for the treatment of MERTK IRD; OPGx-NMNAT1, a gene augmentation therapy designed to halt functional deterioration in pediatric patients with retinal degenerative disease caused by mutations in the nicotinamide mononucleotide adenylyltransferase 1 gene; and OPGx-CNGB1, an AAV gene therapy for retinitis pigmentosa due to mutations in the CNGB1 gene. In addition, it develops Phentolamine Ophthalmic Solution for reversal of mydriasis, as well as for the treatment of presbyopia and dim light or night vision disturbances; and APX3330, a small-molecule inhibitor of Ref-1 for the treatment of diabetic retinopathy. The company was formerly known as Ocuphire Pharma, Inc. Opus Genetics, Inc. was founded in 2018 and is headquartered in Durham, North Carolina.
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Jazz Pharmaceuticals plc identifies, develops, and commercializes pharmaceutical products in the United States, Europe, and internationally. The company offers Xywav to treat cataplexy or excessive daytime sleepiness (EDS) with narcolepsy and idiopathic hypersomnia (IH); Xyrem for the treatment of cataplexy or EDS with narcolepsy; Epidiolex for seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC); Rylaze for the treatment of acute lymphoblastic leukemia or lymphoblastic lymphoma; Enrylaze to treat acute lymphoblastic leukemia and lymphoblastic lymphoma; Zepzelca for the treatment of metastatic small cell lung cancer with disease progression on or after platinum-based chemotherapy; Ziihera to treat HER2-positive biliary tract cancers; Defitelio to treat severe veno-occlusive disease; and Vyxeos for the treatment of newly-diagnosed therapy-related acute myeloid leukemia. It also develops Zanidatamab in Phase 3 trial to treat HER2-expressing gastroesophageal adenocarcinoma (GEA), and patients with HER2-expressing metastatic GEA; Epidiolex in Phase 3 trial to treat LGS, DS, and TSC; Suvecaltamide in Phase 2 trial to treat Parkinson's disease tremor; JZP815, a pan-RAF kinase inhibitor in Phase 1 trial that targets components of the mitogen-activated protein kinase; JZP898, a conditionally activated interferon alpha molecule in Phase 1 trial; JZP441 in Phase 1 trial to treat narcolepsy, IH, and other sleep disorders; JZP324, an extended-release low sodium oxybate formulation in Phase 1 trial for patients with sleep disorders; and JZP150 to treat post-traumatic stress disorder. The company has licensing and collaboration agreements with Redx Pharma plc, Autifony Therapeutics Limited, Zymeworks Inc., Sumitomo Pharma Co., Ltd., and Werewolf Therapeutics, Inc. Jazz Pharmaceuticals plc was founded in 2003 and is headquartered in Dublin, Ireland.
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