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IRD vs RNAZ

Opus Genetics, Inc. Common Stock and TransCode Therapeutics, Inc. Common Stock

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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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TransCode Therapeutics, Inc. Common Stock

TransCode Therapeutics, Inc., a biopharmaceutical company, focuses on developing and commercializing drugs and diagnostics for treating and identifying cancer. The company's lead therapeutic candidate comprises TTX-MC138 that targets microRNA-10b, a master regulator of metastatic cell viability in various cancers comprising breast, pancreatic, ovarian, colon cancer, glioblastomas, and others. Its preclinical programs include TTX-siPDL1, an siRNA-based modulator of programmed death-ligand 1 and two indication agnostic programs; TTX-RIGA, an RNA- based agonist of the retinoic acid-inducible gene I targeting activation of innate immunity in the tumor microenvironment; TTX-CRISPR, a CRISPR/Cas9-based therapy platform for the repair or elimination of cancer-causing genes inside tumor cells; and TTX-mRNA, an mRNA-based platform for the development of cancer vaccines that activate cytotoxic immune responses against tumor cells. TransCode Therapeutics, Inc. has strategic collaboration agreement with The University of Texas M. D. Anderson Cancer Center. The company was incorporated in 2016 and is based in Boston, Massachusetts.

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