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DTIL vs IRD

Precision BioSciences, Inc. Common Stock and Opus Genetics, Inc. Common Stock

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Precision BioSciences, Inc. Common Stock

Precision BioSciences, Inc., a clinical stage gene editing company, develops in vivo gene editing therapies for gene edits, including gene insertion, excision, and elimination in the United States. The company offers ARCUS, a genome editing platform for DNA genome insertion, removal, and repair. It also provides PBGENE-HBV for the treatment of chronic hepatitis B virus (HBV) to eliminate covalently closed circular DNA with direct cuts and edits, as well as to inactivate integrated HBV DNA with the goal of long-lasting reductions in hepatitis B surface antigen; and PBGENE-3243 for the treatment of m.3243 associated mitochondrial myopathy. In addition, the company develops PBGENE-NVS (insertion) for sickle cell and beta thalassemia diseases; PBGENE-DMD (excision) for duchenne muscular dystrophy; PBGENE-LIVER (insertion), a liver directed target; PBGENE-CNS (excision), a central nervous system directed target; and iECURE-OTC (insertion) for ornithine transcarbamylase deficiency. It has collaboration agreements with Novartis Pharma AG to discover and develop in vivo gene editing products; TG Cell Therapy, Inc. to develop, manufacture, and commercialize azer-cel for autoimmune diseases and other indications outside of cancer; and iECURE, Inc. to develop ARCUS-based gene-insertion therapies. Precision BioSciences, Inc. was incorporated in 2006 and is headquartered in Durham, North Carolina.

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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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