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IRD vs RNAC

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

Cartesian Therapeutics, Inc., a clinical-stage biotechnology company, provides mRNA cell therapies for the treatment of autoimmune diseases. The company's lead product candidate is Descartes-08, an autologous mRNA CAR-T directed against the B cell maturation antigen (BCMA), which is in Phase 2b clinical trials for the treatment of autoimmune diseases, generalized myasthenia gravis, and systemic lupus erythematosus, as well as rare pediatric disease designation for the treatment of juvenile dermatomyositis. It is also developing Descartes-15, an autologous anti-BCMA mRNA CAR-T to treat autoimmune diseases, such as relapsed/refractory multiple myeloma. The company is headquartered in Frederick, Maryland.

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