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AGIO vs IRD

Agios Pharmaceuticals, Inc. and Opus Genetics, Inc. Common Stock

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Agios Pharmaceuticals, Inc.

Agios Pharmaceuticals, Inc., a biopharmaceutical company, discovers and develops medicines in the field of cellular metabolism in the United States. Its lead product includes PYRUKYND (mitapivat), an activator of wild-type and mutant pyruvate kinase (PK), enzymes used for the treatment of hemolytic anemias in adults with PK deficiency. The company's PYRUKYND product also used for the treatment of sickle cell disease that is in phase 3 clinical trial; for the treatment of PK deficiency in pediatric patients; and for the treatment of adult patients with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia. In addition, it develops tebapivat, a PK activator for the treatment of lower-risk myelodysplastic syndrome and hemolytic anemias; AG-181, a phenylalanine hydroxylase stabilizer for the treatment of phenylketonuria (PKU); and AG-236, an siRNA in-licensed from Alnylam, targeting the transmembrane serine protease 6 (TMPRSS6) gene for the treatment of polycythemia vera. Agios Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.

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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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