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IRD vs VOR

Opus Genetics, Inc. Common Stock and Vor Biopharma 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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Vor Biopharma Inc. Common Stock

Vor Biopharma Inc. operates as a clinical-stage cell and genome engineering company. The company offers the Vor Bio platform, a technology platform for the treatment of acute myeloid leukemia (AML) and other blood cancers. It develops tremtelectogene empogeditemcel (trem-cel), a genome-edited hematopoietic stem and progenitor allogeneic donor product candidate to treat acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and other blood cancers; trem-cel in combination with Mylotarg to treat patients in VBP101, which is in phase 1 / 2; and VCAR33, a CAR-T therapy designed to target CD33, which is under preclinical studies to treat AML post-transplant. The company also develops trem-cel + VCAR33 treatment system, CD33-directed CAR-T program, which is under pre-clinical studies for the treatment of AML; VADC45 for the treatment of hematologic malignancies, conditioning, and immune reset, which is under preclinical studies; and CD33-CLL1 treatment system made with CD33-CLL1 multiplex-edited HSC therapy and a CD33-CLL1 multi-specific CAR-T therapy, which is under preclinical studies for the treatment of AML. It has a license agreement with Columbia University and the National Institutes of Health for patents; and with Editas Medicine, Inc. for ex-vivo Cas9 gene-edited HSC therapies for the treatment and/or prevention of hematological malignancies. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

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