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IRD vs SLN

Opus Genetics, Inc. Common Stock and Silence Therapeutics Plc American Depository Share

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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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Silence Therapeutics Plc American Depository Share

Silence Therapeutics plc, a biotechnology company, engages in the discovery and development of novel molecules incorporating short interfering ribonucleic acid (siRNA) to inhibit the expression of specific target genes in hematology, cardiovascular, and rare diseases. The company develops Zerlasiran (SLN360), which is in Phase II clinical trial for cardiovascular disease associated with elevated lipoprotein(a); Divesiran (SLN124), an siRNA molecule that is in Phase I clinical trial for the treatment of genetic hematological conditions, including polycythemia vera; and SLN548 for complement-mediated disease. Silence Therapeutics plc has collaboration agreements with AstraZeneca PLC to discover, develop, and commercialize siRNA therapeutics for the treatment of cardiovascular, renal, metabolic, and respiratory diseases; and Hansoh Pharmaceutical Group Company Limited to develop siRNAs. The company was formerly known as SR Pharma plc and changed its name to Silence Therapeutics plc in May 2007. Silence Therapeutics plc was incorporated in 1994 and is headquartered in London, the United Kingdom.

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