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ARWR vs IRD

Arrowhead Research Corporation and Opus Genetics, Inc. Common Stock

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Arrowhead Research Corporation

Arrowhead Pharmaceuticals, Inc. develops medicines for the treatment of intractable diseases in the United States. Its pipeline includes Plozasiran to reduce production of apolipoprotein C-III in Phase 3 studies; Zodasiran to reduce production of angiopoietin-like protein 3 in Phase 3 clinical trials; ARO-DIMER-PA, a dual functional RNAi molecule in a Phase 1/2a clinical trials; and ARO-PNPLA3, an investigational RNAi therapeutic in Phase 1 clinical trials. The company also develops ARO-INHBE, to reduce the hepatic expression of the INHBE gene and its secreted gene product, Activin E in Phase 1/2a clinical trials; ARO-ALK7 to silence adipocyte expression of the ACVR1C gene in Phase 1/2a clinical trials; ARO-RAGE to reduce production of the receptor for advanced glycation end products in Phase 1/2a clinical trials; and ARO-MAPT, an investigational RNAi-based therapy. In addition, it is developing ARO-C3 to reduce production of C3 in Phase 1/2a clinical trials; and ARO-CFB to reduce hepatic expression of CFB in a Phase 1/2a clinical trials. The company has collaboration and license Agreements with Glaxosmithkline Intellectual Property (No. 3) Limited; Takeda Pharmaceutical Company Limited; Amgen Inc.; and Sarepta Therapeutics, Inc. Arrowhead Pharmaceuticals, Inc. was founded in 2001 and is headquartered in Pasadena, California.

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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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