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GANX vs IRD

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

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Gain Therapeutics, Inc. Common Stock

Gain Therapeutics, Inc., a biotechnology company, develops novel small molecule therapeutics to treat diseases across various therapeutic areas in Switzerland, Spain, the United States, and Australia. Its drug discovery platform Magellan discovers novel allosteric binding sites in a disease; identifies proprietary small molecules that bind these sites to modulate protein function; and treats the underlying cause of the disease. The company's lead drug candidate is GT-02287, which is currently in a Phase 1b study for the treatment of Parkinson's disease with or without a GBA1 mutation. It has various small molecule drug candidates, which are in the discovery, research, and preclinical stages for the treatment of dementia with Lewy bodies, Alzheimer's disease, and Gaucher's disease, lysosomal storage disorders, metabolic disorders, and solid tumors. Gain Therapeutics, Inc. was founded in 2017 and is based in Bethesda, Maryland.

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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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